Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy
Year: 2025
Type: article
Abstract: mice. VGR-R01 was well tolerated in New Zealand rabbits and non-human primates (NHPs). Furthermore, after subretinal administration, VGR-R01 was primarily distributed in the ocular tissues, especially in the retina, with minimal systemic presence, notably in the gonads. Overall, these results support the potential for clinical application of VGR-R01 in treating BCD.
Authors: Wenjing Luo, Lu Guo, Liang Lu, Nan Huang, Yezheng Tao, Yufei Zhang, Yanrong Cao, Shin-Shay Tian, Xiaoping Zhao, Xi Zhu
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,900
OpenAlex ID: W4409158751