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Innovation

Patient-Specific Stem Cells and Personalized Gene Therapy

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

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