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Rescuing Trafficking Mutants of the ATP-binding Cassette Protein, ABCA4, with Small Molecule Correctors as a Treatment for Stargardt Eye Disease - PubMed Stargardt disease is the most common form of early onset macular degeneration. Mutations in ABCA4, a member of the ATP-binding cassette (ABC) family, are associated with Stargardt disease. Here, we ha...

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In this #experimentalstudy, we demonstrated that certain #CFTRmodulators rescue misfolded variants in #ABCA4, a protein involved in macular degeneration. These may represent promising #therapeutics for #Stargard #eyedisease 🧑🏻‍🔬🧬💊
#drugdevelopment

pubmed.ncbi.nlm.nih.gov/26092729/

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