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BioWorld - Tuesday, June 9, 2026
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Home » NGGT-001 gene therapy shows efficacy in cell and animal models of Bietti’s crystalline dystrophy
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Ocular

NGGT-001 gene therapy shows efficacy in cell and animal models of Bietti’s crystalline dystrophy

May 30, 2023
Bietti’s crystalline corneoretinal dystrophy (BCD) is an autosomal recessive inherited disease caused by mutations in the cytochrome P450 (CYP) family 4 subfamily V member 2 (CYP4V2) gene, which encodes a polyunsaturated fatty acid (PUFA) hydroxylase dominantly expressed in retinal pigment epithelium (RPE) cells.
BioWorld Science Conferences Genetic/congenital Ocular Gene therapy

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