Evren U. Azeloglu, PhD is an Associate Professor of Medicine and Pharmacological Sciences at the Icahn School of Medicine at Mount Sinai; member of the Black Family Stem Cell Institute; and the director of the NIDDK-funded MERRIT Fellowship Program that aims to recruit engineering students into kidney research. His research aims to understand the principles of mechanobiological cellular decision-making and to use this knowledge in therapeutics discovery. He is an inventor of a number of advanced microscopy methods as well as implantable devices. Dr. Azeloglu directs Systems Bioengineering Lab (https://azeloglulab.org), a multidisciplinary research group in the Division of Nephrology that has been funded by multiple NIH, DOD, foundation, and industry grants. His team employs multiscale experimental and computational techniques, such as machine learning-based high-content image analytics, microfabrication, integrative multiomics, and tissue engineering, to study cell signaling, biomechanics and tissue regeneration using renal and vascular model systems.

Current ongoing research interests include:

1. Understanding the role of mechanosensitive actin-associated proteins in podocyte cytoskeletal integrity
2. Development of iPSC-based kidney-on-chip platforms for kidney precision medicine
3. Investigating the role of cellular biomechanics in kidney tissue organization and pathophysiology
4. Role of cellular biophysics in metabolic syndrome
5. Cellular mechanisms of tyrosine kinase inhibitor associated nephrotoxicity

Profile

Margaret H. Baron, MD/PhD, is The Irene and Arthur M. Fishberg Professor of Medicine and Professor of Cell, Developmental, and Regenerative Biology and of Oncological Sciences at Mount Sinai. She recently took on a new leadership role as Senior Associate Dean for Basic and Clinical Research Training and Director of the Office of Physician-Scientist Research Training, after serving as Senior Associate Dean for MD/PhD Education and Director of Mount Sinai’s Medical Scientist Training Program (MSTP, MD/PhD Program) for nearly 8 years. Her lab focuses on basic mechanisms in mammalian hematopoietic (blood cell) development, with an emphasis on cells of the erythroid and myeloid (red and white blood cell) lineages. We have used molecular, cellular, and biochemical approaches to study the regulation of globin gene expression, activation of hematopoiesis in the mouse embryo, and regulation of erythroid development and differentiation. The lab has made extensive use of novel transgenic mouse reporter lines for for tagging, tracking, and sorting of erythroid, endothelial, and endodermal lineages. A patent based on our work on hedgehog was licensed to Curis, Inc. To extend our studies on primitive erythroid development to definitive erythropoiesis, we established culture systems for the expansion and differentiation of mouse and human definitive erythroid progenitors. This work led to our discovery of a role for signaling by the vitamin D receptor (VDR) nuclear receptor transcription factor in the growth and development of definitive erythroid progenitors. These studies have the potential to reveal new approaches for regulating hematopoiesis and to suggest options for the development of novel therapies, including more efficient production ex vivo of blood cells for transfusion.

Ongoing research interests include:

• Investigating the molecular mechanisms that regulate erythroid progenitor growth and development

• Examining a possible role for immune-related genes in erythropoiesis

• Understanding the function of canonical Wnt signaling in embryonic (primitive) erythropoiesis

Profile

Emily Bernstein, PhD, is a Professor of Oncological Sciences and Dermatology at the Icahn School of Medicine at Mount Sinai and co-leader of the Cancer Mechanisms Program of The Tisch Cancer Institute, an NCI-designated center. She also organizes numerous epigenetics events including Mount Sinai’s Chromatin Club. Her team studies epigenetic mechanisms underlying normal development and cancer with a focus on histone variant proteins that replace canonical histones within the nucleosome core particle. Histone variants are specialized in function and represent an important layer of regulation to diversify the structural characteristics and functional outputs of chromatin. Her laboratory discovered a key role for H2A histone variants such as macroH2A and H2A.Z in melanoma malignancy and reprogramming towards pluripotency.

Ongoing research interests include:

• Investigating histone variant-deficient mice in normal development, stem cell populations, and tumor initiation/progression
• Understanding the role of the epigenome and non-coding DNA elements in cancer progression and drug resistance
• Modeling pediatric cancer mutations in chromatin factors by directed differentiation protocols of human pluripotent stem cells

Lab

Profile

James J. Bieker, PhD, is a Professor in the Department of Cell, Developmental, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai. The laboratory's interest is in the transcriptional regulation of red blood cell-specific gene expression, and in identifying the molecular events that confer the ability to express lineage-specific genes in uncommitted, pluripotent hematopoietic stem cells. These issues are being addressed by the functional analysis of a novel, erythroid-specific gene that the Bieker Laboratory identified a number of years ago named erythroid Krüppel-like factor; EKLF. Biochemical, molecular, cellular, developmental, and genetic studies in mice and humans have established that EKLF is an essential component required for globin switching and completion of the definitive erythroid program.

Using our model system, we aim to disentangle genotype from phenotype and cell-type and identify disease relevant common and unique pathways that hold promise for future therapies.

Ongoing research interests include:

• Analyzing EKLF protein/protein interactions and how they result in altered transcriptional and epigenetic changes at target loci
• Determining how molecular controls converge to regulate late events in erythropoiesis, particularly enucleation
• Analyzing EKLF upstream regulators to explain its exquisite tissue-restricted expression pattern, and to possibly link alteration of its expression level to aberrant red cell biology
• Determining the mechanism by which a human mutation in EKLF leads to congenital dyserythropoietic anemia.

Lab

Profile

Joel Blanchard, PhD, is an Investigator in the Black Family Stem Cell Institute, The Ronald Loeb Center for Alzheimer’s Disease, and an Assistant Professor in the Departments of Neuroscience, and Cell, Developmental and Regenerative Biology. He trained in stem cell biology and neuroscience at Harvard University and The Scripps Research Institute and completed post-doctoral studies at MIT. The Blanchard lab aims to develop cutting-edge in vitro models of the human brain. We then apply these models in conjunction with studies on post-mortem human brain tissue to understand and therapeutically target genetic and environmental vulnerabilities to neurodegeneration.

Ongoing research interests include:

• Developing and applying complex physiological models of neurodegeneration using induced pluripotent stem cells
• Investigating how genetic and environmental factors cooperate to influence neurodegeneration.
• Understanding the role of sex in vulnerability to neurodegeneration
• Establishing the neurological impact of COVID-19.

Lab

Profile

Timothy A. Blenkinsop, PhD, is an Assistant Professor in the Department of Cell, Development, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai, as well as member of the Ophthalmology Department, the Black Family Stem Cell Institute, and Mount Sinai’s Eye and Vision Research Institute. His team is interested in understanding human retina development in normal and pathological situations, as well as in modeling in vitro various eye diseases. The lab’s primary interest lies in retina stem cell biology, cell replacement therapy, and endogenous retina regeneration potential. The lab uses adult human tissues and pluripotent stem cells-derived differentiated cells to probe retina physiology and model diseases such as age-related macular degeneration, proliferative vitreoretinopathy, and proliferative diabetic retinopathy. The main goal is to identify epigenetic regulation of cell plasticity for stimulating it with therapeutic benefit, while also modeling diseases where plasticity leads to disease.

Ongoing research interests include:

• Exploring signaling transduction pathways involved in retinal pigment epithelium plasticity
• Exploring cell replacement therapy for age-related macular degeneration
• Researching cell fate specification of developing human eye field

Lab

Profile

Allison Bond, Ph.D. is an Assistant Professor in the Departments of Neuroscience and Cell, Developmental and Regenerative Biology. Research in the Bond Lab is motivated by the great need for effective treatments for brain disorders and the promise of stem cells as a target for medical interventions. Neural stem cells possess the incredible capacity to generate new cells over long periods of time, but we do not understand how they are regulated inside the brain. Our lab works to uncover the endogenous programs that regulate neural stem cell capacity across the lifespan in vivo so that we can successfully target the brain’s innate plasticity for therapeutic purposes. The Bond Lab is focused on understanding how interactions between neural stem cells and the surrounding niche environment influence the capacity for neural plasticity across the lifespan. The lab uses single-cell omics, lineage tracing, viral and CRISPR genetic manipulations, and time-lapse imaging to uncover mechanisms regulating neural stem cell function in the mammalian hippocampus region of the brain. The goal of the lab’s research is to identify therapeutic interventions that promote and preserve the brain’s endogenous regenerative capacity.

Ongoing Research Interests:

• Investigate mechanisms underlying neural stem cell property changes that occur across development and the lifespan
• Identify cell-cell interactions that orchestrate neural stem cell development
• Investigating the role of cellular biomechanics in kidney tissue organization and pathophysiology
• Uncover the long-term impact of altered neural stem cell development on hippocampus-related learning and memory function

Joseph Castellano, PhD is an Assistant Professor of Neuroscience and Neurology and member of the Friedman Brain Institute and the Ronald M. Loeb Center for Alzheimer’s disease at the Icahn School of Medicine at Mount Sinai. Understanding the mechanism by which aging acts as a major risk factor for neurodegenerative disorders is critical as the aging population increases in the coming decades. Recent work has demonstrated that age-related changes in neural cells of the neurogenic niche within hippocampus are regulated by cues present in the systemic environment. Research in the Castellano laboratory is focused on understanding the molecular mechanisms underlying such blood-CNS communication, as well as the extent to which this communication shapes development of brain neuropathology in neurodegenerative diseases. Towards the goal of developing novel therapies that exploit this putative communication, the laboratory is interested in identifying and defining youth-associated activities that can rescue aspects of Alzheimer’s-related pathology. The Castellano laboratory specializes in a wide range of genetic tools in mouse models to answer complex physiological and compartmental questions related to brain function. Multiple levels of analysis are incorporated in the laboratory’s experimental aims, including editing in cell culture, human disease modeling in mice via viral-mediated and cell transfer tools, and cognitive behavioral assays. A focused goal of the group is to characterize the mechanism by which novel blood-borne factors mediate changes in function in the hippocampus.

Ongoing research interests include:

• Characterizing mechanism of action of blood-borne factors within hippocampus
• Understanding the role of genetic risk factors in regulating blood-brain communication in the context of disease.
• creation of novel humanized mouse models to understand neuroimmune function in the context of disease pathology.

Lab
Profile

Hina W. Chaudhry, MD is a Professor of Medicine and Cardiology, and Director of Cardiovascular Regenerative Medicine at the Icahn School of Medicine at Mount Sinai. She obtained Bachelor’s degrees in Chemistry and Biology at MIT, and completed her MD with Honors through the Health Sciences and Technology program at Harvard Medical School. She completed her training in Internal Medicine and Cardiovascular Medicine at Duke and University of Pennsylvania respectively. She trained in Genetics and Developmental Biology at Columbia University where she also became Florence Irving Assistant Professor of Medicine. She has pioneered the study of cell cycle biology in cardiac development and its application to the treatment of heart disease. Her laboratory was the first in the world to regenerate the porcine heart after myocardial infarction with 55% increase in cardiomyocyte numbers using cyclin A2 gene therapy, the most extensive regeneration seen to date in large animals. Her lab has also utilized the phenomenon of fetal-maternal crosstalk to discover the stem properties of placental CDX2 cells which they have shown to be poised for cardiogenesis.

Ongoing research interests include:

• Studies of the epigenetic control of cell cycle silencing in the mammalian heart
• Interactions of cell cycle proteins with cardiac contractile proteins
• Development of an allogeneic cell therapy strategy utilizing CDX2 cells for heart repair and other types of organ repair

Profile

Bo Chen, PhD, is an Associate Professor in Ophthalmology at the Icahn School of Medicine at Mount Sinai and Director of the Ocular Stem Cell Program in the Department of Ophthalmology at the Icahn School of Medicine at Mount Sinai and New York Eye and Ear Infirmary of Mount Sinai. The Chen Laboratory is focusing on mechanistic and therapeutic studies of retinal degenerative diseases typically characterized by the loss of photoreceptors and retinal ganglion cells. The diseases studied include age-related macular degeneration, retinitis pigmentosa, and glaucoma. In developing treatments for these degenerative conditions, Dr. Chen’s laboratory employs neural regenerative and protective research strategies to generate new retinal neurons and to save existing retinal neurons, respectively.

Ongoing research interests include:

• Defining intrinsic signaling pathways and transcription control in Müller glial cells (MGs) and reprogramming them in vivo to generate MG-derived retinal stem cells capable of differentiating to new photoreceptors
• Investigating the molecular mechanisms underlying histone deacetylase 4 (HDAC4)-mediated photoreceptor protection using animal models of retinal degenerative diseases
• Exploring the functional role of calcium signaling in damaged ganglion cells (the primary cell type affected in glaucoma) and their axons

Lab

Profile

Ya-Wen Chen, PhD, is an Assistant Professor in the Department of Otolaryngology, and Cell, Developmental, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai. She is also a member of the Institute for Airway Science at Mount Sinai. She pioneered the work in lung organoid development and directed differentiation from human pluripotent stem cells (hPSCs) to respiratory epithelial cells. The Chen lab focuses on applying hPSC-derived lung organoids as a model to study the mechanisms of lung injury repair and the applications of stem cell-derived lung progenitor or mature airway/alveolar epithelial cells to lung injury repair, stem cell-based therapy, and regenerative medicine. The ultimate goal is to reconstruct a functional trachea and lung in vitro or in situ for replacement. In addition, responding to infectious disease caused by coronavirus family and influenza virus, the lab is studying the role of TMPRSS2, a type II transmembrane serine protease, during viral infection. To achieve their goals, the lab integrates research from three different fields: developmental biology, stem cell therapy, and bioengineering.

Ongoing research interests include:

• Branching morphogenesis of the lung
• Cell fate decision of lung progenitors
• Infectious disease modeling
• Lung cancer modeling
• Trachea transplantation
• Drug screening using lung organoid as a platform

Lab

Profile

Nicole Dubois, PhD, received her PhD in Cell and Developmental Biology from the Swiss Institute for Experimental Cancer Research and the University of Lausanne with Andreas Trumpp, PhD. She did her postdoctoral training in the laboratory of Gordon Keller, PhD, at the University Health Network in Toronto with a focus on pluripotent stem cell biology and their differentiation to cardiovascular lineages. She is currently an Associate Professor in the Department of Cell, Developmental, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai, the Black Family Stem Cell Institute, and the Mindich Child Health and Development Institute. Dr. Dubois' group studies early heart development and congenital heart disease using pluripotent stem cell differentiations and the mouse embryo as model systems.

Ongoing research interests include:

• Understanding the molecular mechanisms of atrial-ventricular development
• Exploring pluripotent stem cell differentiation to cardiovascular lineages
• Looking at cardiac maturation and tissue engineering
• Investigating long non-coding RNAs during cardiac development
• Exploring cardiac toxicity of cancer drugs (LINCS project)

Lab

Profile

Fanny Elahi, MD, PhD is a physician-scientist and Assistant Professor in the Departments of Neurology, Neuroscience, and Pathology at the Icahn School of Medicine at Mount Sinai in New York City. She serves as Director of Fluid Biomarker Research at the Barbara and Maurice Deane Center for Wellness and Cognitive Health and is one of the leaders at the Alzheimer’s Disease Research Center, where she co-directs the Genetics and Genomics Core and oversees the development of novel blood biomarkers for neurodegenerative diseases. She also has an appointment at the James J. Peters Department of Veterans Affairs Medical Center.

Elahi’s research is making progress on therapeutic discoveries for vascular causes of dementia with a focus on genetic diseases, such as CADASIL. By combining molecular techniques with clinical data, and advanced data analytics, her multidisciplinary research program is studying the link between disease of blood vessels and neurodegeneration. A major focus is the development of blood biomarkers and the combination of markers with in vitro models of disease to advance understanding of disease mechanisms and identification of novel drug targets. She is passionate about translating laboratory findings into clinical applications and looks forward to the day she will administer disease-modifying treatments to her patients and stop dementia in its tracks.

Ongoing research interests:

1. Identification of blood molecular signatures of risk and resilience for brain degenerative disorders
2. Multi-modal modeling of brain degenerative disorders to better understand mechanisms and therapeutic opportunities
3. Modeling vasculature contributions to cognitive impairment and dementia from in vivo to in vitro, and in silico therapeutic target identification

Biosketch

Elena Ezhkova, PhD, is an Associate Professor in the Cell, Developmental, and Regenerative Biology Department. Her laboratory implements an array of powerful cellular and high-throughput molecular biology tools to dissect how epigenetic gene regulators, specifically the polycomb repressive complexes 1 and 2 (PRC1 and PRC2), play a role in cell fate determination, homeostasis, and regeneration. Her lab has recently showed that PRC1 functions both as a transcriptional repressor and as a transcriptional activator during skin development. Importantly, PRC1-mediated gene activating functions are critical for hair follicle development and for the establishment of the adult bulge stem cell compartment. Identification of these molecular mechanisms that control cell fate determination, commitment, and differentiation aids in expanding our understanding of tissue development and the progression of various tissue disorders, including cancer. 

Ongoing research interests include:

• Uncovering the molecular mechanism of PRC-mediated gene regulation in skin development, homeostasis, and regeneration
• Dissecting the canonical and non-canonical PRC function in different epithelial tissues, including skin and oral epithelia
• Uncovering the molecular mechanisms controlling Merkel cell development and maintenance and Merkel cell carcinoma formation

Lab

Profile

Fei Fang, PhD, is an Assistant Professor in the Leni and Peter W. May Department of Orthopaedics at the Icahn School of Medicine at Mount Sinai. She received her graduate training in musculoskeletal tissue biomechanics at Washington University in St. Louis and completed postdoctoral studies in tendon developmental biology and stem cells at Columbia University Irving Medical Center. The Fang lab focuses on deciphering cues of musculoskeletal mechanobiology and stem cell biology and aims to combine the research results with tissue engineering and stem cell therapies for promoting regeneration of musculoskeletal tissues.

Ongoing research interests include:

• Investigating how mechanical cues regulate stem cell dynamics during tendon development and aging
• Understanding the role of stem cell niche in driving cell fate commitment of tendon and ligament
• Developing cell therapies for promoting tendon, ligament, and meniscus regeneration

Lab

Profile

Bruce Gelb, MD, is the Gogel Family Professor of Child Health and Development and Professor of Pediatrics and Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai, and he directs the Mindich Child Health and Development Institute. He is a co-leader of a National Heart, Lung, and Blood Institute-funded training program in molecular and cellular cardiology. Dr. Gelb’s research group studies the genetic causes of cardiovascular diseases of childhood, particularly focusing on congenital heart defects and inherited disorders of the Ras/microtubule-associated protein (MAP) kinase signal transduction pathway (called RASopathies). His group, working with the then Black Family Stem Cell Institute Director, Ihor Lemischka, PhD, generated the first human induced pluripotent stem cell (hiPSC) model of a cardiovascular trait (hypertrophic cardiomyopathy for Noonan syndrome with multiple lentigines).

Ongoing research interests include:

• Understanding the pathogenesis of hypertrophic cardiomyopathy for RASopathies
• Discovering novel therapies for hypertrophic cardiomyopathy for RASopathies
• Understanding congenital heart defect pathogenesis due to histone modifier mutations

Lab

Profile

Saghi Ghaffari, MD, PhD, is a Professor of Cell, Developmental, and Regenerative Biology and a member of The Tisch Cancer Institute. Her lab studies mechanisms that sustain blood forming stem and progenitor cells (HSPCs) throughout life and that are perturbed in disease. Her team is particularly interested in programs that maintain quiescence of blood-forming stem cells, a property that determines the potency and overall regenerative capacity of adult stem cells and that is lost with age. Quiescence is also a mechanism by which malignant stem cells resist therapy. To attain this goal, the Ghaffari Laboratory has been investigating metabolic and mitochondrial-related programs and organelle communications in young and aged HSPCs. The Ghaffari lab uses a variety of approaches, including various omics, super resolution imaging, and gene modulation technology combined with genetically modified mouse models and human cells to address these questions.

Ongoing research interests include:

• Investigating mechanisms that control mitochondria-lysosome communication in normal mouse and human hematopoietic stem cells (HSCs) in aged HSCs and in leukemic stem cells
• Exploring transcriptional and epigenetic programs that regulate organelle biogenesis in HSPCs and their alteration with age
• Investigating metabolic and redox regulation of erythroid cell maturation and identifying mechanisms and components of mitochondria-nucleus communication during this process
• Elucidating mechanisms of apoptosis in beta-thalassemia

Lab

Profile

Alison M. Goate DPhil, is a molecular geneticist with 34 years of experience in the application of molecular genetics and genomic approaches to the identification and characterization of the molecular mechanisms underlying genetic risk factors. I have a well-funded research program that has had continuous funding for the last twenty-eight years and has resulted in over 550 peer-reviewed publications. My laboratory has been central to the identification of several genes causing inherited neurological disorders including amyloid precursor protein and presenilins in Alzheimer's disease, microtubule associated protein and progranulin in frontotemporal dementia and TDP43 in ALS. For the last 10 years my lab has focused on the genetics of late onset AD. We were involved in the initial discovery of TREM2 as an AD risk factor (Guerreiro et al., 2013). For the last 6 years we have focused on the role of myeloid cells in AD risk.  In Huang et al., 2017 we linked a network of AD risk genes to a novel myeloid expressed AD risk gene, SPI1. More recently we have taken a genome-wide approach to integrating genomic and epigenomic data with genetic data to identify novel genetic causes of AD. This has resulted in the identification of novel AD risk genes coding for endolysosomal proteins (Novikova et al., Nat. Comm.). We have also used induced pluripotent stem cell models to study the role of AD risk variants in AD & FTD (Bowles et al., submitted). We have also focused on the development of induced pluripotent stem cell models to study the role of AD risk variants in neurodegeneration (TCW et al., in revision).  I have a proven track record of mentorship. All of the research in my lab is carried out by scientists in training-junior faculty, postdoctoral researchers and graduate students. In the last twenty-eight years I have been the primary mentor for 18 junior faculty/K awardees, 24 post-doctoral fellows and 18 graduate students. These trainees include both women and under-represented minorities. Many of my former trainees are now independent investigators at top ranked academic institutions including Karen Duff (UCL, UK), Carlos Cruchaga, Celeste Karch, (Washington U), Jim Ray (MD Anderson), Amanda Myers (U of Miami) and John Kauwe (BYU).

Lab

Profile

Ernesto Guccione, PhD, is an Associate Professor of Oncological Sciences, and Pharmacological Sciences at the Icahn School of Medicine at Mount Sinai. His team studies transcriptional and post-transcriptional mechanisms regulating development and cancer with a focus on mammalian protein methyltransferases and the function of alternative splicing. The team uses biochemistry, mouse models, next generation sequencing and splice switching antisense oligonucleotide (AON)-based approaches to understand the molecular mechanisms of action of candidate PMTs or specific isoforms. The range of techniques and approaches used in the lab has allowed researchers to characterize the mechanism of action of specific PMTs (e.g., PRMT5 and PRDM15) or oncogenic isoforms (e.g., MDM4l/s), which are of great interest for their clinical applications.

Ongoing research interests include:

• Investigating the role of PRMTs and PRDMs in normal development, stem cell populations, and tumor initiation/progression
• Using AON-based approaches to uncover the function of specific alternative splicing isoforms
• Exploring use of pharmacogenomic approaches to identify new therapies for hematological and solid tumors

Lab

Profile

Woojin M. Han, PhD, is an Assistant Professor in the Department of Orthopaedics at the Icahn School of Medicine at Mount Sinai. The Han Laboratory develops cell-instructive biomaterials to systematically study how stem cells interact with their niche in regulating tissue pathophysiology, with a primary focus on skeletal muscle stem (satellite) cells and fibro/adipogenic progenitors. His team also establishes regenerative therapies for treating injuries and diseases of the musculoskeletal system by harnessing cell-matrix interactions. Dr. Han has previously engineered a synthetic cell-instructive matrix for facilitating the transplantation and engraftment of muscle stem cells in the context of muscle injury and Duchenne muscular dystrophy. By independently tuning multiple material properties through parametric design, Dr. Han and his team have successfully developed a synthetic cell delivery platform conducive to muscle stem cell survival, proliferation, migration, and differentiation for targeting limb muscle trauma and dystrophic diaphragms. His research program will continue to integrate multidisciplinary bioengineering concepts to pioneer new technologies for dissecting stem cell-niche interactions and advancing regenerative therapies for musculoskeletal injuries and disorders.

Ongoing research interests include:

• Understanding how niche-derived physicochemical cues regulate muscle stem cell polarity, fate determination, and mechanotransduction in development, aging, and diseases.
• Engineering niche-mimetic platforms to direct muscle stem cell manufacturing.
• Developing cell-instructive biomaterials to treat skeletal muscle injuries and disorders.

Lab

Profile

Dan Hasson, PhD, is an Assistant Professor in the department of Oncological Sciences at the Icahn School of Medicine at Mount Sinai, and the co-Director of the TCI/BFSCI Bioinformatics for Next Generation Sequencing core (BiNGS). My main research interest is focused on understanding the functional roles of chromatin in cancer progression.

Research interest include:

• Histone variants
• cis regulatory elements
• Transcription factor networks
• Chromatin folding

Ronald Hoffman, MD, is the Albert A. and Vera G. List Professor of Medicine at the Icahn School of Medicine at Mount Sinai. He is a member of The Tisch Cancer Institute. His team has two areas of interest. One deals with the expansion of the number of marrow repopulating cells present in cord blood for transplantation into humans undergoing allogeneic stem cell transplantation for refractory hematological malignancies. This expansion strategy uses histone deacetylase inhibitors. The team is attempting to understand the mechanism underlying this ex vivo stem cell expansion technology, and is actively evaluating the clinical potential of this expanded cell product in a phase I clinical trial. The second area of interest deals with the blood cancer myelofibrosis. Dr. Hoffman and his team are characterizing the malignant myelofibrosis stem cells and their progeny and the consequence of their interactions with the marrow and splenic microenvironments. With this growing body of information the group is identifying novel agents to deplete myelofibrosis stem cells. These agents are currently being evaluated in a series of phase I/II clinical trials in a clinical consortium including 11 institutions in the United States and Canada, headed by Dr. Hoffman.

Lab

Profile

Nilsson Holguin, Ph.D, is an Assistant Professor of Orthopaedics at the Icahn School of Medicine at Mount Sinai. The focus of his research is the clarification of the adaptation of the spine on a molecular-, cellular- and tissue-level to aging, injury, genetics and biological sex in order to develop therapeutics for spinal degeneration and subsequent back pain.  They have expanded the role of mechanical forces in intervertebral disc degeneration and in bone formation. From independent research in his lab, he finds that therapeutics for osteoporosis may potentially be repurposed to tackle intervertebral disc degeneration, which does not have any FDA-approved therapeutics. The most recent publication is titled “Suppression of sost/sclerostin and dickkopf-1 augment the structure of the intervertebral disc in mice” (Cover of JBMR). Further, work along this line of research has uncovered a new role of an inflammatory-related protein in intervertebral disc degeneration and in osteoblast/osteoclast-related regulation of bone mass. He is the recipient of the Harold Frost Young Investigator Award and the NRSA Postdoctoral Award, among others.

Ongoing research interests include:

• Kroon, T., Bhadouria, N., Niziolek, P., Edwards, D., Clinkenbeard, E.L., Robling, A., Holguin, N., (2022). Suppression of sost/sclerostin and dikkopf-1 augment intervertebral disc structure in mice. J Bone and Miner Res. 37 (6): 1156-1169, DOI: doi.org/10.1002/jbmr.4546.

• Bhadouria, N., Niziolek, P., Berman, A., El Jordi, O., McKinzie, D., Wallace, J., Holguin, N., (2022). Raloxifene stimulates estrogen signaling to reduce sex- and age-related intervertebral disc degeneration in mice. Front. Biotech and Biotech, doi.org/10.3389/fbioe.2022.924918.

Profile

James Iatridis, PhD, is a Professor and Vice Chair for Research in Orthopaedics at the Icahn School of Medicine at Mount Sinai. His team studies the development of early and minimally invasive treatments to repair and regenerate injured and degenerated intervertebral discs. Intervertebral disc degeneration is a critical factor in chronic back pain that is exacerbated with poor loading conditions. Dr. Iatridis is a pioneer in understanding the mechanobiology of intervertebral disc pathophysiology and regeneration. His lab is developing tissue engineering strategies to promote intervertebral disc repair, and is actively identifying progenitors and niche factors that promote its regeneration. Insights from these studies reveal mechanisms on how stem cell niches function in musculoskeletal tissues, and provide a platform for developing intervertebral disc repair and regeneration strategies.

Ongoing research interests include:

• Identifying intervertebral disc progenitor populations that promote annulus fibrosus repair.

• Understanding mechanical niche factors modulating progenitor fate and function.

• Developing therapies that overcome pro-inflammatory conditions in intervertebral disc degeneration.

Profile

Clonal evolution and epigenetic plasticity represent significant challenges in achieving a cure for cancer. As tumors develop, they undergo genetic changes that lead to clonal diversity, allowing the emergence of resistant malignant cells, which can survive treatment. Concurrently, epigenetic modifications can alter gene expression without changing the DNA sequence, enabling cancer cells to evade therapeutic interventions. We believe that by measuring these phenomena directly in cancer patients through advanced single-cell sequencing technologies, we can gain valuable insights into therapy resistance mechanisms. This understanding can pave the way for more effective, personalized treatment strategies aimed at overcoming the dynamic nature of cancer.

Current projects in the laboratory include:

• Resistance to Hypomethylating Agents in MDS: This project aims to define the clonal evolution trajectories together with the changes in epigenetic profiles of malignant cells contributing to therapy resistance in HMA-treated patients. By doing so, we aim at setting the foundation for the development of new therapeutic approaches for the treatment of MDS patients showing resistance to HMAs
• Consequences of Disruption of DNA methylation in Hematopoiesis: By leveraging simultaneous capture of genotypes and transcriptional profiles directly from human samples, we aim to define the consequences ofdisruption of DNA methylation, to identify its functional consequences driving clonal expansions in the blood and reshaping the cell differentiation landscape.
• How DNA Methylation Modulates Transcription Factor Binding in vivo: Previous studies have demonstrated that transcription factors (TFs) exhibit variable binding preferences for methylated sites, with 5mC either enhancing or reducing a TF's affinity for its binding site. This groundbreaking work provided the foundation for considering TFs as readers of DNA methylation. This project will aim to define what is the in vivo sensitivity of TFs to the presence of 5mC at their binding sites.

Biosketch

Andrew Ji, MD, is a physician-scientist and Assistant Professor of Dermatology at the Icahn School of Medicine at Mount Sinai. His lab seeks to understand how intercellular communication contributes to cell subpopulation heterogeneity in diverse tissue states, including normal homeostasis and cancer. His work was among the first to integrate an array of multimodal techniques, including single-cell RNA-sequencing (scRNA-seq), spatial transcriptomics (ST), and multiplexed ion beam imaging (MIBI) on a common cohort of patient tissue. His lab further employs CRISPR genetics, including high-throughput screens, on organoid and xenograft mouse models to assign functional significance to novel subpopulation markers. Through use of these cutting-edge tools, his goals are to identify highly pathogenic subpopulations within complex tissues, how they arise, and exploit knowledge of cell-intrinsic and extrinsic crosstalk pathways to develop novel treatment strategies across cancer and other skin diseases.

Ongoing research interests include:

• Dissecting gene regulatory networks governing invasive tumor subpopulation behavior to identify therapeutic vulnerabilities

• Understanding cancer cell subpopulation signaling involved in tumor microenvironment remodeling

• Defining pathways governing fibroblast plasticity in tissue regeneration and cancer

Lab

Profile

Deepak Kaji, MD, PhD is an Instructor and research-track resident in the Department of Psychiatry at the Icahn School of Medicine at Mount Sinai. He uses human induced pluripotent stem cell models of the brain to study the impact of ultra-rare variants on psychotic illness. Using multi-comics technologies and electrophysiological assays, his team works to understand how ultra-rare variants drive developmental disruptions in the transcriptome , neuronal firing, and neural network dynamics. While schizophrenia undoubtedly has a complex architecture, we hope that studying these monogenic cases will illuminate conserved pathways, and therefore molecular targets, that drive the pathogenesis of schizophrenia.

Ongoing research interests include:

• Understanding the impact of ultra-rare variants on neurodevelopment and brain function
• Investigating the signal that drives maturation of ventricular radial glia towards the plethora of cells in the human brain.

Jaymin Kathiriya, PhD, is an Assistant Professor of Medicine and Cell, Developmental, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai. He is also a member of the Black Family Stem Cell Institute, the Institute of Airway Sciences, and the Mount Sinai National Jewish Health Respiratory Institute. The Kathiriya lab aims to determine the cellular and molecular basis of homeostatic maintenance of lung epithelium and injury response in the context of distal interstitial lung diseases. The lab is also interested in understanding cytoskeletal rearrangements of keratins during adult lung stem cell differentiation during regeneration. The lab employs a combination of in silico, in vitro, and in vivo techniques to understand both plastic and metaplastic regenerative responses of the adult lung epithelium.

Ongoing research interests include:

• Cellular and molecular identity of distal lung epithelial progenitors

• Mechanisms of early activation of stem cells

• Cytoskeletal remodeling of differentiation cell states of lung epithelial progenitors

• Developing novel in silico approaches to understand cells of origin in human diseases

As a professor of Genetics and Genomic Sciences at Icahn School of Medicine at Mount Sinai, I serve as one of the core PIs on the NIH-funded U54 grant, "Frontiers of Congenital Disorders of Glycosylation Consortium." The FCDGC's mission is to conduct one of the largest natural history studies on congenital disorders of glycosylation and identify novel therapies to improve the quality of life for individuals and families affected by CDG.

My laboratory specializes in studying the metabolic aspects of cellular plasticity in CDG using various in vitro and in vivo model systems. We have established a transdisciplinary pre-clinical research program focusing on the significance of cellular glycosylation and its relationship to neuronal structure and function. Specifically, in collaboration with Dr. Sloan at Emory University, we will lead Project 3 with the overarching hypothesis that impaired N-glycosylation in the context of PMM2 and ALG13 pathogenic variants

Profile

Robert Krauss, PhD, is a Professor of Cell, Developmental, and Regenerative Biology at the Icahn School of Medicine at Mount Sinai and Co-director of the Development, Regeneration, and Stem Cells training area for graduate students. His lab is interested in how niche-derived signals regulate stem cell function, using the skeletal muscle lineage as a model system. His lab showed that cadherins are niche factors that promote quiescence of skeletal muscle stem cells under homeostatic conditions, and regulate activation of these cells in response to injury, thereby facilitating tissue regeneration. His lab uses a variety of approaches, including mouse genetics and molecular cell biology, to address questions about how the muscle stem cell niche is constructed, maintained, and functions. The Krauss Laboratory also studies the Nodal and Hedgehog signaling pathways and how deficiency in these pathways results in the common birth defect, holoprosencephaly.

Ongoing research interests include:

• Exploring how cadherins regulate cytoskeletal architecture in muscle stem cells to provide structural integrity, mechanosensation, and cell polarity
• Identifying and analyzing the components of the adherens junction formed between muscle stem cells and their major niche cell, the myofiber
• Modeling the complex etiology of holoprosencephaly in mice
• Assessing functional consequences of human mutations involved in holoprosencephaly

Lab

Profile

Dr. Sai Ma is an Assistant Professor of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai, a member of Icahn Genomics Institute, a member of Skin Biology and Diseases Resource-based Center (SBDRC), Institute for Regenerative Medicine (IRM), Black Family Stem Cell Institute (BFSCI), and an affiliate member of the New York Genome Center (NYGC). His laboratory primarily focuses on innovating experimental and computational tools to investigate epigenetic mechanisms of gene regulation in single-cells across dynamic cell fate transitions. He is keen on developing sequencing tools to map the epigenomes and transcriptomes within rare populations, single-cells or in situ. He also builds workflows and apply these tools to investigate cell fate determination and perturbed cell states, such as hair follicle regeneration, brain development, and tumor progression.

Ongoing research interests include:

• Develop massive-scale single-cell multi-omic technologies
• Dissect epigenetic regulatory principles underlying cell differentiation
• Understand cell-cell communications in situ leveraging spatial-omic technologies

Lab

Profile

Eva Morava M.D., PhD is pediatrician, geneticist and metabolic specialist. Her major clinical expertise is inborn errors of metabolism (IEM). She is the director of the Inherited Metabolic Diseases Program at the Department of genetic and Genomic Sciences, at the Icahn School of Medicine. She has decades of experience in the diagnostics, follow-up and treatment in IEM, especially in congenital disorders of glycosylation (CDG) and mitochondrial disorders. She is actively involved in developing novel therapies in genetic disorders. Currently she focusses on translational research and clinical trials readiness in IEM and rare disease. She is the main PI of the U54 FCDGC consortium studying congenital disorders of glycosylation. Dr Morava and her team established a nationwide network of regional centers to develop treatment in these individually ultra-rare conditions, and meet currently unmet patient needs. FCDGC's mission is to improve clinical symptoms as well as improve quality of life and life expectancy of individuals with congenital disorders of glycosylation through advancing and sharing knowledge, developing and validating new diagnostic tools, and develop therapies to restore appropriate glycosylation. The Morava-Kozicz lab uses iPSC derived models (brain organoids, neurons, iCardiomocytes etc) to study rare disorders and find novel treatments. We use a multiomics approach with a strong metabolomics and glycoproteomics expertise for biomarker discovery and treatment monitoring in our models, translating our findings to human clinical trials.

Ongoing research interests include:

• Understanding the pathomechanism of Congenital disorders of glycosylation using brain organoids in rare CDG
• Dissecting the molecular, metabolic and genetic factors underlying cardiomyopathy in phosphoglucomutase deficiency in iPSC derived model systems
• Investigating drug targets in disorders of energy metabolism and mitochondrial disease

Bio Sketch

Florence Marlow, PhD, is an Associate Professor of Cell, Developmental, and Regenerative Biology, Co-director of the Development, Regeneration, and Stem Cells training area for graduate students, and an Associate Director for the Medical Scientist Training Program, all at the Icahn School of Medicine at Mount Sinai. Her team uses genetic, molecular, cell biological, and embryological approaches to investigate the molecular pathways and cell biological events that regulate specification and maintenance of the germline and that maintain polarity and function in oocytes and in neurons. Her lab identified RNAbps that interact with a key regulator of oocyte polarity and identified novel factors required for sex-specific differentiation of germline cells.

Ongoing research interests include:

• Determining how RNAbps control sex-specific differentiation of germline stem cells
• Performing genetic and cell biological analyses of the specialized cell divisions of germline stem cells and their immediate daughters
• Exploring how RNAbps regulate buckyball and oocyte polarity
• Analyzing mitochondrial bottlenecks in the germline
• Studying microglia involvement in shaping neural circuits underlying complex behaviors in juveniles and adults

Lab

Profile

Samuele G. Marro, PhD, is an Assistant Professor in the Nash Family Department of Neuroscience and co-Director of the Stem Cell Engineering Core at the Icahn School of Medicine at Mount Sinai. His research team focuses on the regulation of synaptic plasticity and its dysfunction in Fragile X syndrome, the number one genetic cause of autism. To accomplish this, the group studies human neurons directly differentiated from pluripotent stem cells that are genetically modified using CRISPR/Cas9 tools. The institutional Stem Cell Engineering Core itself provides services and resources at a reduced cost to the Icahn School of Medicine community that include the derivation of iPS from patient blood samples; iPS differentiation into different cell types; and gene-editing to create or repair putative disease mutations.

Ongoing research interests include:

• Investigating protein homeostasis in human neurons
• Exploring epigenetic regulation of synaptic plasticity
• Understanding and correcting the epigenetic silencing in Fragile X syndrome

Labs

Profile

Alison May, PhD, is an Assistant Professor of Cell, Developmental, and Regenerative Biology and Otolaryngology at the Icahn School of Medicine at Mount Sinai, and a member of the Black Family Stem Cell Institute and the Institute of Airway Sciences. The May lab aims to define causative mechanisms of disease through defining alterations in the structure and function of epithelial organs. The lab’s primary interest lies in understanding the molecular basis of exocrine gland development, regeneration, and remodeling in the upper airway, including the nose, sinuses and proximal trachea, and how perturbations to glandular systems contribute to Cystic Fibrosis and Chronic Rhinosinusitis. Her team uses a combination of in vivo, in vitro and in silico approaches to identify mechanisms of organ patterning, intercellular signaling and stem cell regulation with the aim of identifying therapeutic avenues for the treatment of airway disease.

Ongoing research interests include:

• Airway epithelial stem cell differentiation and lineage commitment
• Cell-to-cell interactions that govern exocrine morphogenesis
• Molecular mechanisms of tubulogenesis
• Role of CFTR in early organ development and epithelial patterning
• Genetic drivers of tissue-based alterations that contribute to Cystic Fibrosis and Chronic Rhinosinusitis

Lab

Profile

Sarah E. Millar, PhD, is Director of the Black Family Stem Cell Institute, and Lillian and Henry M. Stratton Professorial Chair in the Departments of Cell, Developmental, and Regenerative Biology, and Dermatology at the Icahn School of Medicine at Mount Sinai. Understanding the molecular and cellular mechanisms regulating the development, patterning, and postnatal renewal of the skin and ectodermal appendage organs such as hair follicles, teeth, and taste papillae, and identifying stem and progenitor cell populations in these organs, is critical for developing new therapies to accelerate wound healing, treat hair loss diseases, repair or replace diseased teeth, and ameliorate taste dysfunction. Research in the Millar Laboratory focuses on cell-cell signaling and epigenetic mechanisms that underlie these processes. In published research, researchers identified Wnt/beta-catenin signaling as a key pathway required for initiating the formation of ectodermal appendages from multipotent cells in mammalian embryos, and in controlling development and patterning of haired versus hairy skin. By analyzing genetic mouse models and tissues from human patients carrying mutations in the WNT10A gene, we showed that Wnt signaling plays a key role in regulating the functions of a wide variety of adult epithelial stem cells, as well as in controlling specialized differentiation programs in palmoplantar skin. We have also identified critical functions for epigenetic regulators including micro-RNAs and chromatin modifiers in skin development and regeneration.

Ongoing research interests include:

• Investigating mechanisms that cause ectodermal dysplasia in patients with mutations in the WNT10A gene, and testing potential therapeutic strategies
• Determining the mechanisms that underlie the formation and maintenance of hairy versus hairless skin and regulate hair patterning
• Delineating the functions of histone deacetylase chromatin modifiers in skin development, stem cells, and cancer
• Identifying pioneer transcription factors that control development and stem cell activity in skin and oral epithelia

Lab

Profile

Marek Mlodzik, PhD is Professor and Chair of Cell, Developmental, and Regenerative Biology, and Professor in the Departments of Oncological Sciences and Ophthalmology.  He is also on the IAB of the Tisch Cancer Institute, and organizes several cell signaling events in NYC. His team studies the mechanisms of the Wnt and Notch signaling pathways with specific focus on the role of Wnt-signaling in the planar cell polarity (PCP) pathway in normal organogenesis and patterning and disease contexts, including cancer, neural tube closure defects, and ciliopathies.  The Wnt/PCP pathway and Wnt signaling in general are critical in many stem cell niche interactions and stem cell maintenance. The lab uses primarily the Drosophila model for in vivo studies and mammalian cell based work for functional biochemical assays. His lab is the leader in the Wnt-PCP field, both in normal patterning as well as in functional disease dissections.

Ongoing research interests include:

• Investigating the regulatory interactions among the core PCP signaling factors and associated cell adhesion behavior
• Dissecting the process of nuclear translocation of beta-catenin in Wnt-signaling
• Modeling the complex functional behavior of neural tube closure defect patients in PCP establishment in Drosophila
• Understanding novel regulatory inputs to Notch signaling and associated read-outs

Lab

Profile

Dr. Shruti Naik, Ph.D., is an international leader in immunology. She is an Associate Professor in Immunology and Dermatology and the Director of the Tissue Repair Program at Icahn School of Medicine at Mt. Sinai. Dr. Naik's research uses advanced technologies to study how immune cells communicate with tissues, aiming to develop therapies that stop inflammatory damage and rejuvenate organs at the cellular and molecular levels. She has made groundbreaking discoveries on the microbiota's role in immunity, epigenetic memory of inflammation in stem cells, and the mechanisms of immune-tissue interactions in repair and inflammation. A strong advocate for diversity in science, Dr. Naik has received numerous accolades, including the Regeneron Award for Creative Innovation, L'Oréal For Women in Science Award, Damon Runyon Dale F. Frey Award for Breakthrough Scientist, Blavatnik Award for Young Scientists, Takeda Innovators in Science Award, Pew-Stewart Scholar, NIH Directors Innovator Award DP2, Packard Fellow, Burrows Welcome PATH Award, and is a NYSCF Robertson Stem Cell Investigator.

Ongoing research interests include:

• Tissue repair and regeneration in non-healing wounds and inflammatory diseases

• Microbe-tissue stem cell crosstalk

• Inflammatory memory in stem cells

Bio Sketch

Georgia Panagiotakos is a molecular and developmental neuroscientist focused on understanding mechanisms that regulate the adoption of cellular identity, and how disruptions of these mechanisms underlie neurodevelopmental disorders. I have approached this question by studying how immature neural cells regulate their intracellular calcium levels as a means of integrating intrinsic genetic programs with extracellular environmental cues. We aim to define how different modes of calcium entry and downstream intracellular calcium signaling pathways in the developing cerebral cortex set differentiation programs into motion.

Eirini Papapetrou, MD, PhD, is an Associate Professor of Oncological Sciences, Hematology, and Medical Oncology at the Icahn School of Medicine at Mount Sinai; and Associate Director of the Pluripotent Stem Cell Engineering Core. Her laboratory pioneered the modeling of blood cancers with human-induced pluripotent stem cell (iPSC). Specifically, her lab developed the first iPSC models of myeloid malignancies, including myelodysplastic syndromes and acute myeloid leukemia by reprogramming patient cells and by correcting and introducing mutations using CRISPR/Cas9-mediated gene editing. Dr. Papapetrou’s research program combines techniques and principles from stem cell research, cancer biology, and hematopoiesis with the goal of understanding disease mechanisms and identifying new therapeutic targets for hematologic malignancies. The unifying theme of the lab’s projects is the creation of genetically precise isogenic iPSC models of myeloid cancers and exploitation of the unique capabilities they offer for genotype-to-phenotype studies, interrogation of the effects of oncogenic mutations with integrative genomics analyses in a faithful cellular and genomic environment and genetic (CRISPR), and small molecule screens for drug repurposing or drug discovery. Dr. Papapetrou is the recipient of several awards, including the American Society of Gene and Cell Therapy Outstanding New Investigator Award, Damon Runyon-Rachleff Innovation Award, American Society of Hematology Scholar Award, Pershing Square Sohn Prize, and Leukemia and Lymphoma Society Scholar award, among others. She is an elected member of the American Society for Clinical Investigation.

Lab

Profile

Prashanth Rangan, Ph.D, is an Associate Professor of Cell, Developmental, and Regenerative Biology and Pediatrics at the Icahn School of Medicine at Mount Sinai. His team studies the genetic basis of female fertility using fruit fly oogenesis as a model system. Germ cells differentiate by undergoing meiosis to produce an oocyte. Once an oocyte fate is specified, the oocyte synthesizes mRNAs and proteins called the maternal contribution that are critical for initiating development of the early embryo. The Rangan lab research program asks the following questions pertaining to how germ cells transition into an oocyte:

1. How is germ cell entry into meiosis controlled?
2. How is the germ cell program terminated upon initiation of oocyte fate?
3. How are maternal mRNAs synthesized, selected, and regulated to initiate the next generation?
4. How does the surrounding soma regulate oocyte development?

The Rangan lab has discovered a programmatic transition that is required for germ cells to transition into an oocyte that we have termed germ cell to maternal transition (GMT).  We find that GMT includes a broad transcriptional reprogramming, specialized translation, and RNA degradation programs. We believe that defects in GMT could result in infertility. Thus, understanding this transition could underpin potential therapies for infertility.

Ongoing research interests include:

• Determining transcriptional and post-transcriptional control of germ cell to maternal transition
• Identifying the cue that initiates germ cell to maternal transition
• Ascertaining the conservation of germ cell to maternal transition invertebrates

Labs

Michael Rendl, MD, is a Professor of Cell, Developmental, and Regenerative Biology and Dermatology at the Icahn School of Medicine at Mount Sinai; Director of the NYSTEM Training Program in Stem Cell Biology; and Associate Director of the Black Family Stem Cell Institute. His team studies the formation and function of stem cell niches, using hair follicle formation during skin development and adult hair growth and regeneration as a model system. Dermal papilla (DP) cells are specialized mesenchymal niche cells that instruct hair follicle stem cells and progenitors. His laboratory discovered embryonic DP precursors, defined molecular signatures of embryonic and mature DP cells, and established the essential roles of Wnt signaling and the transcription factor Sox2 for DP functions. The lab has uncovered the DP-related dermal sheath (DS) as a key niche component for hair cycle progression and progenitor pruning, essential for the next wave of stem cell activation. Overall, insights from these studies reveal mechanisms on how stem cell niches function and provide a platform for developing hair regenerative therapies.

Ongoing research interests include:

• Understanding the molecular controls that regulate DP niche fate and function
• Investigating DP signals that regulate stem cell and progenitor functions
• Dissecting DS cellular and molecular mechanisms of hair cycle regulation

Lab

Profile

Christoph Schaniel, PhD, is an Assistant Professor in the Department of Pharmacological Sciences, the Department of Cell, Developmental and Regenerative Biology, and a member of the Mount Sinai Institute for System Biomedicine. His research is centered on understanding the cellular and molecular nature of hematopoietic stem cells (HSCs) in health and disease. He helped develop a clinical ex vivo expansion and cryopreservation process of umbilical cord blood HSCs for allogenic stem cell transplantation in refractory hematological malignancies that is being evaluated in a clinical trial (in collaboration with Ronald Hoffman, MD, and Camelia Iancu-Rubin, PhD). Additionally, he is investigating the mechanism and clinical applicability of direct reprogramming of HSCs from somatic cells for cell replacement therapies (together with Kateri A. Moore, DVM). Other research focuses on the effect of valproic acid on normal vs. malignant HSCs, the underlying pathologies of hematological malignancies and cancers using human primary stem cells, and induced pluripotent stem cells (PSCs). He is also invested in various projects aimed at understanding the mechanisms of cardiovascular diseases and drug action, responses, and toxicity in individuals using induced PSCs with the goal of advancing and developing precise and personalized therapeutic treatments.

Lab

Profile

Robert Sebra, PhD, is an Associate Professor in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai. His research lab centers on creating and applying bulk and single cell molecular methods to generate highly resolved data using genomics technology for variant discovery and annotation. This research integrates single molecular and cellular genomics data with assays to assess chromatin accessibility, proteomics, and epigenetics data to decipher factors of pathogenesis versus normal cellular functions. Determining which cellular niches and mechanisms in complex primary tissues drive disease or resilience facilitates translation of functional genomics data using model systems for screening possible downstream regenerative and therapeutic potential using high throughput methods.

Ongoing research interests include:

• Developing novel molecular methods and genome sequencing technologies
• Advancing high-throughput functional genomics
• Exploring whole genome and repeat content characterization for disease association
• Analyzing surveillance and genomic characterization of pathogen transmission
• Using genomics to temporally characterize development and pathogenesis
• Translating genomic data in various oncologic diseases including gynecologic and breast cancers

Lab

Profile

An unprecedented amount of information has been gathered from the analysis of cancer genomes. However, our ability to use this information to identify mechanisms that are critical for cancer cells’ homeostasis and proliferation is still limited. Our lab has pioneered the integration of state-of-the-art genomics, functional studies, and systems biology methods to generate functional maps, allowing us to pinpoint key regulatory hubs that, when perturbed, compromise cancer cell viability. Thus, our research aims to use this data to elucidate novel mechanisms crucial to cancer cells’ survival that represent potential therapeutic opportunities.

The overall interest of our group is to understand the molecular mechanisms that regulate mammary gland development and breast cancer tumor progression with the ultimate goal of rationally developing effective and safe patient-oriented therapies.

Ongoing research interests include:

• Identification of cancer-relevant functions
• In-depth mechanistic characterization of these functions
• Translation of these findings to the clinic

Lab

Profile

Christopher M. Sturgeon is an Associate Professor at the Icahn School of Medicine at Mount Sinai. Chris’s lab studies the development of the human hematopoietic system, using the in vitro differentiation of human pluripotent stem cells (hPSC) as a model system. The ability to differentiate hPSC towards a bona fide hematopoietic stem cell (HSC) would be a major step forward for the treatment of patients in need of a suitable donor match. Similarly, hPSCs offer unprecedented access to early embryonic hematopoietic lineages, which may have untapped clinical potential. To harness these possibilities, it is essential to be able to direct the differentiation of hPSCs in a controlled fashion. To that end, Chris’s research has developed defined media approaches, coupled with staged addition of recombinant morphogens such as BMP, WNT, and RA, to recapitulate these early embryonic developmental stages.

Ongoing research interests include:

• Investigating the molecular mechanisms of hematopoietic development and the immediate precursor to the HSC, hemogenic endothelium
• Characterizing the translational potential of HSC-independent immune lineages
• Identifying the developmental trajectory of nascent mesoderm as it differentiates towards blood
• Understanding the role of RNA splicing in embryonic hematopoiesis

Labs

Profile

Tianliang Sun, PhD, is an Assistant Professor in Liver division, Department of Medicine and the Department of Cell, Developmental and Regenerative Biology at the Icahn School of Medicine at Mount Sinai. He pioneered the work in identifying key signalling pathways during ductular reaction by using loss of function CRISPR screen in biliary epithelial cell derived liver organoids.  His recent study led to the understanding of fine tuning WNT/beta-catenin signalling pathway in regulation of hepatocytes metabolic zonation and proliferation. Combining state-of-art single cell sequencing, spatial transcriptomics, lineage tracing and CRISPR screening technologies, his team try to understand the mechanisms of liver regeneration, and how the imbalanced liver regeneration lead to many liver diseases.

Ongoing research interests include:

• Understanding the plasticity of liver epithelial cells during regeneration and tumor formation.
• Dissecting the mechanisms underlying the balancing of liver regeneration.
• Defining networks that integrate various signaling pathways in the multicellular dynamics during liver regeneration

Lab

Profile

The Trivieri Lab focuses on investigating key molecular mechanisms responsible for the onset and progression of Heart Failure and Pulmonary Hypertension (PH) using state-of-the-art techniques of stem cell biology and tissue engineering.

Ongoing research interests include:

1. Elucidating the role of Phospholamban (PLN) in the pathophysiology of inherited cardiomyopathy.

2. Modeling PH using adult stem cells derived smooth muscle and endothelial cells

Bio Sketch

Profile

Nadia (Nadejda) Tsankova, MD/PhD, is an Associate Professor in Pathology and Neuroscience, member of the Tisch Cancer Institute, and the Program Director of the Neuropathology fellowship at the Icahn School of Medicine at Mount Sinai, where she has been on Faculty since 2014. Her team studies the molecular mechanisms of growth and invasion in glioblastoma and their therapeutic vulnerability in preclinical models, focusing on the role of epigenetic and transcriptional regulators for maintaining developmentally-coopted cancer stem-cell and migration states, and emphasizing analyses of tumor biology in its nascent state and within its microenvironment through the use of primary patient samples and human-based models. The dismal prognosis in glioblastoma, the most common primary brain tumor in adults, is attributed to its diffusely infiltrative biology, which remains poorly understood and without effective therapy. Epigenetics maintains developmental plasticity in glioma stem cell states, and deeper understanding of its regulation may expose convergent therapeutic targets. The Tsankova lab previously uncovered an epigenetic mechanism driving EGFR re-expression in gliomas and has characterized novel strategies to prospectively isolate neural and glioma stem cell populations from human tissue. Through a series of epigenetic and functional studies, the lab recently discovered the Hippo pathway effector, TEAD1, to be an essential driver of glioblastoma migration and is now actively investigating the therapeutic efficacy of specific YAP-TEAD inhibitors in pre-clinical models. In parallel, the lab employs single cell transcriptomics and epigenomics to resolve glial lineage choice during late prenatal human neurodevelopment and to define developmental pathways co-opted in glioma growth and migration.

The ultimate goal of this research is to expose tumor-intrinsic vulnerabilities in glioblastoma malignancy that can be harnessed towards effective treatment options for patients.

Ongoing research interests include:

• Mechanistic analysis of EGFR and Hippo pathway crosstalk in glioblastoma driving tumor invasion and progression (R01NS106229-funded)

• Characterization genome-wide TEAD1 occupancy and YAP/TEAD-mediated chromatin remodeling in glioblastoma progression and chemoresistance (partially R01NS106229-funded)

• Resolving glial cell types and lineage trajectories during late prenatal and early postnatal human neurodevelopment and their recapitulation at the tumor core and infiltrative edge in glioblastoma (RF1DA048810-funded)

Lab

Profile

Alexander M. Tsankov, PhD, is an Assistant Professor in Genetics and Genomic Sciences at Icahn School of Medicine at Mount Sinai. He completed his PhD in electrical engineering and computer science at MIT and his postdoctoral training at the Broad Institute and Harvard University (Alex Meissner and Aviv Regev’s lab). The Tsankov lab overall vision is to use genomics to build data-driven, predictive models that improve diagnosis, find new drug-able pathways, and personalize treatment of patients with lung cancer and respiratory diseases. The lab specializes in next generation sequencing (NGS) technologies (e.g. single-cell transcriptomic, epigenomic, and spatial data) and computational analysis with the goal of unraveling how the underlying regulatory mechanisms, cell-cell interactions, and regenerative lineages have changed in lung disease compared to normal lung tissue homeostasis.

Ongoing research interests include:

• Using single-cell technologies to reconstruct the regenerative lineages in the human lung and to understand how these have been hijacked in cancer or altered in lung disease
• Investigating cell-cell interactions and their role on lung regeneration, disease progression, and immunosuppression.
• Dissecting the cell-type specific regulatory mechanisms underlying normal lung homeostasis and changes that arise in respiratory disease and lung cancer

Lab

Profile

Matheus Victor, PhD, is an investigator of the Institute for Regenerative Medicine, the Ronald M. Loeb Center for Alzheimer’s Disease and the Friedman Brain Institute. Dr. Victor is an assistant professor in the Department of Neuroscience, and in the Department of Cell, Developmental and Regenerative Biology. He joins the faculty of the Icahn School of Medicine at Mount Sinai as an NIH FIRST Faculty Scholar and a Howard Hughes Medical Institute Hanna Gray Faculty Fellow.

Dr. Victor earned his Ph.D. in Neuroscience from Washington University in St. Louis where he pioneered a novel cellular reprogramming approach to study the contribution of aging to neurodegeneration. Dr. Victor conducted his postdoctoral training at MIT where he investigated how genetic susceptibility in microglia, the brain-resident immune cells, contributes to the pathobiology of Alzheimer’s disease.

At Mount Sinai, the Victor lab leverages 3D stem cell technologies, CRISPR-based tools, post-mortem human brain samples, and mouse models to elucidate the molecular, cellular, and systems-level consequences of glial perturbations associated with neurodegenerative diseases to the function of neural circuits.

Ongoing research interests include:

• Neuron-glia interactions in the pathophysiology of neurodegenerative diseases.
• Mechanisms of neuronal network assembly, remodeling, and degeneration with 3D cell culture models of the human brain.
• Molecular, cellular and circuit level mechanisms governing neuropsychiatric symptoms of Alzheimer’s disease.

Elvin Wagenblast, PhD, is an Assistant Professor of Oncological Sciences and Pediatrics at the Icahn School of Medicine at Mount Sinai. The central question of his team is to understand how a normal blood stem cell can become cancerous. In leukemia, the initiating genetic mutations occur as early as during fetal development and generate preleukemic cells, which are the evolutionary ancestors of leukemia that arises after birth. The lab uses human primary blood stem cells and CRISPR/Cas9-mediated gene editing to model the preleukemic and leukemic phases of acute myeloid and lymphoblastic leukemia in order to uncover insights into the genetic, cellular and developmental mechanisms of the disease. The overall goal of the lab is to identify genetic dependencies of leukemia and guide the rational development of effective cancer therapeutics.

Ongoing research interests include:

• Understanding how the developmental stage of the cell of origin drives differences in the ability to initiate childhood and adult leukemia
• Investigating how oncogenic mutations and gene fusions lead to therapy resistance in leukemia
• Identifying and characterizing genetic vulnerabilities in leukemia using functional genomic screens

Lab

Profile

Mingang Xu, Ph.D., is a Research Assistant Professor in the Department of Cell, Developmental and Regenerative Biology. His current research focuses on molecular mechanisms regulating development and regeneration of the epidermis and its appendages, including hair follicles, fungiform papillae, filiform papillae, sweat glands and teeth. He is particularly interested in delineating the functions of Wnt/beta-catenin signaling in these processes, and in understanding how this pathway is dysregulated in skin diseases. He generated a genetic mouse model for the ectodermal dysplasia syndromes Odonto-onycho-dermal dysplasia (OMIM #257980) and Schöpf–Schulz–Passarge syndrome (OMIM #224750). Human patients with these diseases display skin and dental disorders. Dr. Xu uncovered the mechanisms underlying these diseases. His research has identified potential therapeutic approaches for affected individuals.

Ongoing research interests include:

• Mechanisms underlying skin heterogeneity and regeneration
• Molecular controls of ectodermal appendage patterning
• Mechanisms controlling Merkel cell maintenance and regeneration
• Roles of canonical and non-canonical Wnt signaling in craniofacial morphogenesis

Nan Yang, PhD, is an Assistant Professor of Neuroscience at the Icahn School of Medicine at Mount Sinai. She is also a member of The Friedman Brain Institute, and the Ronald M. Loeb Center for Alzheimer's Disease at Mount Sinai. She pioneered the work in direct lineage reprogramming from somatic cells to neural cells and its application in neuropsychiatric and neurological disease modeling. Her team studies how disease-associated risk variants contribute to pathogenesis of multiple neuropsychiatric disorders with a particular focus on understanding how patient mutations in chromatin modifying proteins and functional non-coding elements impact gene expression regulation and neuronal function and contribute to autism spectrum disorder (ASD).

Ongoing research interests include:

• Modeling ASD mutations in chromatin factors by directed differentiation protocols of human pluripotent stem cells into neurons
• Understanding the role of the epigenome and non-coding DNA elements in neuronal signaling dependent gene regulation in different human neuronal subtypes
• Exploring the mechanisms underlying transcription factors directed differentiation of human stem cells to neuronal subtypes.
• Investigating autophage in human neurons and assessing functional outcome of mutations in Alzheimer's disease and Parkinson’s disease that affect autophage pathways.

Lab

Profile

Zhe Ying, PhD, is an assistant professor in the Department of Cell, Developmental, and Regenerative Biology. His laboratory focuses on establishing the causal relationship between patient-derived genetic lesions and behaviors of epithelial cells during tumor initiation and progression. These include, but not limited to, cell fate choices, proliferation, migration and colonization of distant organ, which collectively contribute to the transition from homeostasis to malignant growth. In the past few years, he build up the know-how to rapidly test single or pooled gene function using embryonic transduction of oral and breast epithelia, and directly observe cell fate choice using intra-vital as well as whole mount 3D imaging. With these cutting-edge technologies, he discovered that PI3K induced differentiation creates a barrier to clonal expansion that may act as a dominate tumor suppressive mechanism in vivo. As a natural extension of this discovery, the lab is currently testing a hypothesis that patient-derived genetic lesions and secreted niche factors drive stratified epithelial cells to overcome PI3K induced differentiation and promote HNSCC initiation. In mammary epithelium, he characterized the lineage dynamics of its early progenitors using barcoded lentivirus, and established that every mouse mammary gland originates form a defined number of ectodermal progenitors that will give rise to epithelial clones of equal expansion potential. Based on this insight, he developed a genetic screen platform that identified both drivers of breast cancer initiation and commitment to disease-associated cell fates. Overall, these discoveries provide unique but fundamental insights of oncogenic growth from the perspectives of developmental biology and may lay the foundations of protentional cancer therapies.

Ongoing research interests include:

• How do key pro-renewal driver lesions and niche factors expand the epithelial progenitor pool in HNSCCs.
• Understand the cellular and molecular basis of linage-specific tumor suppressive function of p53.
• Functionally identify high-dimensional driver combinations of basal like breast cancer at single cell resolution.

Profile

Dr. Guo-Cheng Yuan is a Professor at Icahn School of Medicine at Mount Sinai. Prior to his current appointment, he was an Associate Professor at Dana-Farber Cancer Institute/Harvard Medical School and a Principal Faculty member at Harvard Stem Cell Institute. He obtained B.S and M.A degrees in Mathematics from Peking University, and a PhD degree in Mathematics from University of Maryland, followed by postdoctoral training at Brown University. Dr. Yuan transitioned into a computational biologist by taking up a postdoctoral fellow position at Harvard University before joining Dana-Farber as a faculty member. Dr. Yuan’s group has developed a number of computational methods for analyzing and interpreting large-scale biological data. Notable examples include methods for epigenomics (Haystack, diHMM, CUT&RUNTools), for single-cell analysis (SCUBA, GiniClust, Giotto), and for genome-editing (CRISPResso). For over a decade, Dr. Yuan’s group has collaborated with experiment biologists to elucidate the gene regulatory networks in stem cell maintenance and cell differentiations.

Profile

Lab

Lior Zangi, PhD, is an Assistant Professor in Cardiology at the Icahn School of Medicine at Mount Sinai. He completed his education and training at the Weizmann Institute of Science and at Harvard University. He has established a new method, mRNA based, for gene delivery to promote cardiovascular regeneration. Dr. Zangi’s laboratory investigates cellular and organ level regenerative mechanisms that can contribute to new therapeutic approaches to repair and regenerate the heart following myocardial infarction. The Zangi lab's goal is to induce cardiac regeneration after myocardial infarction using a gene therapy approach with modified mRNA. Modified mRNA is an attractive and novel in vivo gene delivery method that allows high gene expression in a variety of organs, including the heart. The Zangi Laboratory will use modified mRNA to transiently change the non-regenerative gene expression profile of adult cardiac muscle to grant regenerative capacity to the adult heart after injury. The lab plans to investigate specific genes or signaling networks on both cellular (cell specific) and tissue (whole muscle) levels, using modified mRNA and other gene delivery approaches.

Ongoing research interests include:

• Induce cardiomyocytes proliferation and survival by manipulating cardiomyocytes metabolic pathways
• Use cardiac reprograming to induce cardiovascular regeneration
• Employ long non-coding RNA to induce cardiac regeneration

Lab

Profile

The Zwaka Lab’s main line of research investigates ways to direct pluripotent stem cells to replace human cells affected by injury or disease. Dr. Zwaka was recruited to Mount Sinai in 2013 to become Professor of Developmental and Regenerative Biology. After earning his MD and PhD degrees from Ulm University in Germany, Dr. Zwaka trained as a cardiologist and discovered the link between C-reactive protein and atherosclerotic inflammation, a connection that has had enormous importance for cardiology. Dr. Zwaka then went to the University of Wisconsin to do his postdoctoral fellowship in the lab of Jamie Thomson, who derived the first human embryonic stem cell line in 1998. In Thomson’s lab, Dr. Zwaka pioneered methods to genetically manipulate stem cells (gene editing). He then joined the faculty of Baylor College of Medicine, serving in both the Department of Molecular and Cellular Biology and in the Center for Cell and Gene Therapy. At Baylor, the Zwaka Lab discovered a key regulator of pluripotency that behaved so differently from canonical stem cell factors that it was named Ronin.

Lab

Profile

Victoria Zyulina, MD PhD, is an Assistant Professor in the Department of Microbiology at the Icahn School of Medicine at Mount Sinai, working in Dr. Ana Fernandez-Sesma’s Lab. Her research focuses on investigating mechanisms of innate immunity in the context of Influenza and Dengue viruses. This includes the Dengue virus that have triggered historically largest outbreak of in Argentina and Brazil in year 2024. Her work specifically examines the immune responses occurring in the skin after an infected mosquito bite—a key factor that shapes the virus’s systemic progression and clinical outcomes. To date, little is known about the first cells targeted by mosquito-borne viruses in the skin as well as subsequent dissemination of the virus to other target cells. Her studies leverage recent advances in immunology research in order to delineate how skin microenvironment regulates systemic immune response to viral infections. In the lab Dr. Zyulina incorporates technical innovation in utilizing hematopoietic stem cell derived dendritic cells to understand skin-derived signals that can impact the viral pathogenesis. Ultimately, this research lays the groundwork for next-generation vaccines and antiviral therapies at the forefront of microbiology and infectious disease.

Ongoing research interests include:

• Understanding of viral immune antagonism and responses to Influenza and Dengue viral infections

• Investigating of immunogenicity and efficacy of vaccines in ex vivo primary human model systems

• Utilizing hematopoietic stem cell-derived dendritic cells as a complementary model to study viral diseases

Biosketch