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Functional Analysis and Classification of Homozygous and Hypomorphic ABCA4 Variants Associated with Stargardt Macular Degeneration

Stargardt macular degeneration (STGD1) is caused by mutations in the gene encoding ABCA4, an ATP-binding cassette protein that transports N-retinylidene-phosphatidylethanolamine (N-Ret-PE) across photoreceptor membranes. Reduced ABCA4 activity results in retinoid accumulation leading to photorecepto...

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Bibliografiske detaljer
Udgivet i:Hum Mutat
Main Authors: Curtis, Susan B., Molday, Laurie L., Garces, Fabian A., Molday, Robert S.
Format: Artigo
Sprog:Inglês
Udgivet: 2020
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7755071/
https://ncbi.nlm.nih.gov/pubmed/32845050
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/humu.24100
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