[PERSPECTIVES] Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A

Usher syndrome is a deaf-blindness disorder. One of the subtypes, Usher 1B, is caused by loss of function of the gene encoding the unconventional myosin, MYO7A. A variety of different viral-based delivery approaches have been tested for retinal gene therapy to prevent the blindness of Usher 1B, and a clinical trial based on one of these approaches has begun. This review evaluates the different approaches.

Source: Cold Spring Harbor perspectives in medicine - June 1, 2015 Category: Research Authors: Lopes, V. S., Williams, D. S. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research