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The ABCs of Stargardt disease: the latest advances in precision medicine

26 Jul 2024

Abstract excerpt

Stargardt disease (STGD) is the most common form of inherited juvenile macular dystrophy and is caused by sequence variants in the ABCA4 gene. Due to its genetic complexity and phenotypic variability, STGD poses significant therapeutic challenges. In the past decade, a lot of progress has been made regarding our understanding of the molecular and clinical aspects of STGD, along with its mechanisms. This has led...

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PMID: 39060921
DOI: 10.1186/s13578-024-01272-y

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