Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy

Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy

Bietti crystalline dystrophy (BCD) is an autosomal recessive disorder caused by loss-of-function mutations in the CYP4V2 gene, characterized by crystal-like lipid deposits in the retina, progressive photoreceptor loss, and retinal pigment epithelium (RPE) deterioration. Currently, there are no appro...

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Bibliographic Details
Main Authors: Wenjing Luo, Lu Guo, Liang Lu, Nan Huang, Yezheng Tao, Yufei Zhang, Yanrong Cao, Shin-Shay Tian, Xiaoping Zhao, Xi Zhu
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
BCD
AAV
CYP4V2
preclinical study
subretinal injection
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000555
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