Article

Allele-specific correction of dominant Best vitelliform macular dystrophy in patient-derived retinal pigment epithelium

2026-07-15

Abstract excerpt

<h4>ABSTRACT</h4> Autosomal dominant Best vitelliform macular dystrophy (BVMD) caused by variants in the BEST1 gene is characterized by dysfunction of the macular retinal pigment epithelium (RPE) and secondary degeneration of the photoreceptors. There are currently no approved treatments for BVMD, and owing to its dominant nature, there remains uncertainty regarding the utility of traditional gene augmentation....

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Literature Corpus work: 99221281-70d2-5356-a07d-d99871a87865
DOI: 10.64898/2026.07.09.737567

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