The Department of Genetics and Genomic Sciences and the Icahn Genomics Institute are advancing precision techniques to modify gene and cell function - driving progress in both fundamental biology and translational research.
Recent breakthroughs in gene editing technologies, including CRISPR/Cas9 and TALEN, have made it possible to precisely modify the genome. These tools enable the creation of highly accurate cellular and animal models of disease and hold transformative potential for treating genetic disorders through targeted gene and cell therapies. In particular, CRISPR-based approaches are opening new avenues for understanding disease mechanisms and accelerating drug discovery through high-throughput screening across diverse tissue types and disease states.
Beyond gene editing, researchers at Mount Sinai use modified RNA and a variety of viral and non-viral vectors to introduce genes or proteins into cells, reprogramming cellular behavior for therapeutic or experimental purposes.
These technologies are applied to both somatic and stem cells, including induced pluripotent stem cells (iPSCs), which can be differentiated into multiple tissue types. This allows researchers to model normal development, study disease progression, investigate tissue regeneration, and conduct drug screening in systems that closely mimic human biology. When combined with gene editing, iPSCs can be used to generate patient-specific disease models, explore therapeutic responses, and even correct genetic defects—laying the foundation for personalized regenerative medicine.
Gene editing and cell engineering have broad applications across biomedical science, with ongoing research focused on cardiovascular, neurodegenerative, hematologic, immune, metabolic, hereditary, and oncologic diseases.