supported by the National Natural Science Foundation of China(81930121 and U1602224);the National Key Research and Development Program of China(2018YFA0107902,2017YFC1001902,and 2018YFA0801403);the Yunnan Fundamental Research Projects(2019FY002 and 2018FB114);the Major Basic Research Project of Science and Technology of Yunnan(202001BC070001)。
Gene therapy is expected to fundamentally correct mutant genes to cure genetic diseases in vivo.As a delivery system for gene therapy,adenovirus-associated virus(AAV)vectors have achieved positive results in clinical ...