AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models
AAV2 介导的 Bietti 结晶性营养不良症基因治疗可在多种相关细胞模型中提供功能性 CYP4V2
期刊:Scientific Reports
影响因子:3.8
doi:10.1038/s41598-022-12210-8
Jiang-Hui Wang, Grace E Lidgerwood, Maciej Daniszewski, Monica L Hu, Georgina E Roberts, Raymond C B Wong, Sandy S C Hung, Michelle E McClements, Alex W Hewitt, Alice Pébay, Doron G Hickey, Thomas L Edwards
