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.


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*


  • MYO7A protein, human
  • Myo7a protein, mouse
  • Myosin VIIa
  • Myosins