Gene Editing at NYSCF

GENE EDITING Gene editing is a powerful tool that yields new insights into diseases and ways to treat or cure them.

By allowing scientists to precisely manipulate the DNA of human cells, gene editing has revolutionized biomedical research.

NYSCF’s state-of-the-art stem cell technologies unlock the full potential of gene editing for understanding and treating disease.

NYSCF GLOBAL STEM CELL ARRAY ®

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The NYSCF Global Stem Cell Array generates large numbers of high-quality, patient-specific cells that capture the diverse genetics of the human population.

By gene editing these cells with CRISPR, a tool pioneered in human cells by NYSCF – Robertson Investigator Dr. Feng Zhang, we can see how different genetic mutations impact different diseases. This gives us a path to cure these diseases by correcting the underlying genetics.

STEM CELLS AND GENE EDITING ARE REVOLUTIONIZING MEDICAL RESEARCH

A NEW WINDOW INTO WHAT CAUSES RARE DISEASES Taken together, rare diseases affect an estimated 30 million Americans, many of which are children. These diseases are usually caused by a single genetic mutation, but scientists often don’t have effective models to study them.

NYSCF collaborates with disease-focused foundations to create stem cell models of rare diseases such as Alexander Syndrome, Jordan’s Syndrome, and Pelizaeus-Merzbacher Disease. When combined with gene editing, these models allow researchers to study the genetic origins of rare diseases in human cells as well as uncover fundamental biological processes that go wrong in other disorders.

UNLOCKING THE GENETIC COMPLEXITY OF COMMON DISEASES Diseases like Alzheimer’s, Parkinson’s, diabetes, MS, and cancer also carry a significant genetic component, but involve many genetic factors rather than just one.

Gene editing cells from patients with these diseases helps us uncover which genetic mutations matter for which patients and develop tailored therapies that account for individual variations.

CURING DISEASE BY CORRECTING GENETIC DEFECTS Gene editing therapies for sickle cell anemia and beta thalassemia are already in clinical trials, and many more diseases will be targeted in the future.

NYSCF is creating clinical-grade stem cells for use in future therapies. Gene editing these stem cells will help us address the root cause of different diseases when introduced into patients.

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