title: Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A.
abstract: 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.
authors.affiliation: Stein Eye Institute, University of California at Los Angeles, Los Angeles, California 90095-7000.
authors.forename: Vanda S
authors.initials: VS
authors.lastname: Lopes
authors.affiliation: Stein Eye Institute, University of California at Los Angeles, Los Angeles, California 90095-7000.
authors.forename: David S
authors.initials: DS
authors.lastname: Williams
mesh_terms: D000256:Adenoviridae; D000818:Animals; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016086:Lentivirus; D051379:Mice; D009154:Mutation / Q000235:genetics*; D000081412:Myosin VIIa; D009218:Myosins / Q000235:genetics*; D010641:Phenotype; D052245:Usher Syndromes / Q000235:genetics / Q000628:therapy*
chemical_list: C000631455:MYO7A protein, human; C000631456:Myo7a protein, mouse; D000081412:Myosin VIIa; D009218:Myosins