Patient-Specific Stem Cells and Personalized Gene Therapy

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Patients’ own cells transformed into model for studying disease and developing potential treatment

July 10, 2014

Gene Therapy Genetics Rare Diseases Retinitis Pigmentosa Stem Cells

Translational Research

Grantee

Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.