因此，许多AAV介导的治疗方法已经或者正在试验中，以治疗溶酶体储存障碍、阿尔茨海默病、帕金森病、肌萎缩侧索硬化症（ALS）、癫痫、1型脊髓性肌萎缩症、异染性脑白质营养不良、芳香族L-氨基酸脱羧酶（AADC）缺乏症和巴顿病（Goswami et al. 2019） 1 ...

“We have seen remarkable results from clinical trials using AAV vectors for AADC gene transfer,” says Shin-ichi Muramatsu, a pioneer of AAV vector research, and a professor in Jichi Medical ...

For central nervous system (CNS) applications, attempts are being made to deliver AAV gene therapy directly to the brain parenchyma or the cerebrospinal fluid (CSF) space by intra-cisterna magna ...