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 ...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 and preclinical research,including the treatment of genetic diseases,such as those affecting the blood and eyes[1].展开更多
基金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)+1 种基金the Yunnan Fundamental Research Projects(2019FY002 and 2018FB114)the Major Basic Research Project of Science and Technology of Yunnan(202001BC070001)。
文摘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 and preclinical research,including the treatment of genetic diseases,such as those affecting the blood and eyes[1].
