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

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 is essential for the clearance of all-trans-retinal from photoreceptor cells. Loss of this function res...

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Опубликовано в: :Hum Gene Ther
Главные авторы: Dyka, Frank M., Molday, Laurie L., Chiodo, Vince A., Molday, Robert S., Hauswirth, William W.
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc., publishers 2019
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Research Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6854433/
https://ncbi.nlm.nih.gov/pubmed/31418294
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.132
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