Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease

Autor: Laurie L. Molday, Vince A. Chiodo, Frank M. Dyka, William W. Hauswirth, Robert S. Molday
Rok vydání: 2019
Předmět:
Zdroj: Human Gene Therapy. 30:1361-1370
ISSN: 1557-7422
1043-0342
DOI: 10.1089/hum.2019.132
Popis: Autosomal recessive Stargardt disease is the most common inherited macular degeneration in humans. It is caused by mutations in the retina-specific ATP binding cassette transporter A4 (ABCA4) that ...
Databáze: OpenAIRE