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 |
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Rok vydání: | 2019 |
Předmět: |
0303 health sciences
genetic structures biology ABCA4 Retinal ATP-binding cassette transporter Macular degeneration medicine.disease Molecular biology eye diseases Stargardt disease 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine chemistry 030220 oncology & carcinogenesis Genetics biology.protein medicine Molecular Medicine sense organs Vector (molecular biology) Molecular Biology 030304 developmental biology |
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 |
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