Research Saves Lives | Icahn School of Medicine

At the Mount Sinai Health System, our researchers are asking questions and finding answers that save lives. From mapping the brain circuits that link stress to disease, to engineering personalized cancer vaccines, our investigators work at the intersection of breakthrough science and urgent clinical need. Our goal is to understand the mechanisms of disease so completely that we can prevent it, treat it more effectively, and ultimately cure it.

Mount Sinai’s commitment to discovery spans every stage of life and every system of the body. We are developing new therapies for childhood leukemia and creating brain-computer interfaces to restore independence to patients with paralysis. We are uncovering why aging is the single biggest risk factor for cancer—and identifying drugs that might ameliorate its effects.

Each study at Mount Sinai serves as a bridge between what is possible today and what can be standard care tomorrow. Your support, collaboration, and participation accelerate our journey to save lives.

Make A Gift Find a Clinical Trial Explore Education Opportunities

The Brain Immune Cells That Fight Alzheimer's Disease

Investigators at the Icahn School of Medicine at Mount Sinai, working in collaboration with the Max Planck Institute for Biology of Ageing, The Rockefeller University, and The City University of New York, have identified a sub-type of microglia that limit inflammation and slow the buildup of amyloid plaques and toxic tau proteins, the hallmarks of Alzheimer's disease. This discovery establishes a new molecular framework for understanding how the brain can protect itself, and points to microglia-targeted immunotherapies as a promising new approach for modifying the course of Alzheimer's disease.

How APOE4 Damages the Brain's Blood Vessels

A research team led by Joel Blanchard, PhD, has discovered how APOE4, the strongest genetic risk factor for Alzheimer's disease, causes protective cells that wrap around blood vessels to transform into scar-tissue-producing cells, trapping toxic amyloid within vessel walls and accelerating disease progression. Critically, the researchers identified a specific signaling pathway, TGF-β, as the driver of this transformation, and showed that blocking it in experimental models restored healthy vascular function and reduced amyloid buildup, revealing a new therapeutic target.

A Gene Therapy Approach to Autism and Phelan-McDermid Syndrome

Researchers at the Seaver Autism Center for Research and Treatment, led by Alexander Kolevzon, MD, are testing a first-of-its-kind gene therapy treatment for autism spectrum disorder associated with Phelan-McDermid syndrome, a rare genetic disorder caused by a mutation in the SHANK3 gene. With implications that could extend to other genetic forms of autism, this trial represents a concrete step toward tackling the disorder at its core.

How Noncoding Genes Drive Neurodevelopmental Disorders

Investigators at the Icahn School of Medicine at Mount Sinai discovered that three of the most common monogenic neurodevelopmental disorders are caused by variants in the noncoding RNA genes RNU4-2 and RNU2-2. This series of landmark studies, led by Ernest Turro, PhD, and Daniel Greene, PhD, could bring long-awaited diagnoses to hundreds of thousands of currently undiagnosed individuals worldwide. The discoveries also lay the groundwork for future gene therapies.

Mapping How Viruses Escape Our Immune Defenses

A first-of-its-kind structural atlas of COVID-19 antibodies, created by Yi Shi, PhD, and collaborators, reveals how antibodies attach to the SARS-CoV-2 virus and how viral mutations weaken that attachment. The research explains why variants like Omicron can evade immune defenses and points to new strategies for longer-lasting antibody therapies and vaccines.

New Approaches to Prevent Avian Flu Infection

In a study published in Nature Communications, Florian Krammer, PhD, and colleagues generated human antibodies capable of neutralizing H5N1, the highly pathogenic avian influenza virus that has been spreading since 2022. Effective both as a preventative and a treatment in animal models these antibodies could be mass-produced as a life-saving intervention if bird flu begins to spread widely among humans.

COVID-19 Immunity Lasts Longer Than We Thought

A large-scale study led by Viviana Simon, MD, PhD and Florian Krammer, PhD, analyzing more than 8,000 samples collected over three years in New York City, has shown that antibody responses to SARS-CoV-2 infection and vaccination are far more durable than previously believed, with an initial decline followed by a prolonged stabilization phase. The findings, published in the journal Immunity, offer important insights into the long-term dynamics of COVID-19 immunity and how boosters and breakthrough infections affect protection over time.