Article
Bietti crystalline corneoretinal dystrophy: Advances in understanding and gene therapeutic approaches.
Molecular vision - 1 Jan 2025
Zhao Guangrong, Ding Dayou
Abstract excerpt
Bietti crystalline dystrophy (BCD), an autosomal recessive inherited retinal disorder caused by mutations in the CYP4V2 gene, has long remained therapeutically challenging. Recent advances in adeno-associated virus-based gene therapy have emerged as promising therapeutic strategies for patients with BCD. This review synthesizes current knowledge regarding the molecular genetic mechanisms underlying BCD...
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