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Tingting Yang
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Gene therapy for Bestrophin-1 (BEST1) mutations in retinal degenerative diseases
CU21279
This technology is an AAV-mediated BEST1 gene therapy for the treatment of retinal degenerative disorders associated with Bestrophin-1 (BEST1) dominant mutations.
7
Adeno-associated virus
Gene therapy
Genetic disorder
Tingting Yang
Stephen H. Tsang M.D., Ph.D.
Yu Zhang
Kristin Neuman
Ophthalmology
College of Physicians and Surgeons (CUMC
Columbia University Medical Center (CUMC)
2023-09-08
