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

Vanda S Lopes; David S Williams

doi:10.1101/cshperspect.a017319

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

Cold Spring Harb Perspect Med. 2015 Jan 20;5(6):a017319. doi: 10.1101/cshperspect.a017319.

Authors

Vanda S Lopes¹, David S Williams¹

Affiliation

¹ Stein Eye Institute, University of California at Los Angeles, Los Angeles, California 90095-7000.

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.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Adenoviridae
Animals
Disease Models, Animal
Gene Transfer Techniques
Genetic Therapy / methods*
Humans
Lentivirus
Mice
Mutation / genetics*
Myosin VIIa
Myosins / genetics*
Phenotype
Usher Syndromes / genetics
Usher Syndromes / therapy*

Substances

MYO7A protein, human
Myo7a protein, mouse
Myosin VIIa
Myosins