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...

Full description

Saved in:
Bibliographic Details
Published inCold Spring Harbor perspectives in medicine Vol. 5; no. 6; p. a017319
Main Authors Lopes, Vanda S, Williams, David S
Format Journal Article
LanguageEnglish
Published United States Cold Spring Harbor Laboratory Press 01.06.2015
Subjects
Adenoviridae
Animals
Disease Models, Animal
Gene Transfer Techniques
Genetic Therapy - methods
Humans
Lentivirus
Mice
Mutation - genetics
Myosins - genetics
Phenotype
Usher Syndromes - genetics
Usher Syndromes - therapy
Online AccessGet full text

Cover

More Information
Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:2157-1422
2472-5412
DOI:10.1101/cshperspect.a017319