《Table 1 AAV-mediated gene therapy in clinical trials for neurodegenerative diseases in the central

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《Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases》

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*Estimated number of enrolled participants.AAV:Adeno-associated virus；GDNF:glial cell line-derived neurotrophic factor；NTN:neurturin；CERE:Ceregene；GAD:glutamic acid decarboxylase；UPDRS:unified PD rating scale；AADC:aromatic L-amino acid decarboxylase；PD:Pa

The distinct structures of AAV-mediated gene therapy have allowed novel treatments of neurodegenerative diseases in the central nervous system，particularly with recombinant AAV.To analyze the application and related function of AAV，this review investigates the past，present and future statuses of AAV for the treatment of neurodegenerative diseases in the central nervous system.We performed literature searches by using PubMed，Scopus，Embase and Web of Science databases with no language restrictions.The time period ranged from July 1，2005 to May 31，2018.Search terms include“central nervous system”or“gene therapy”or“neurodegenerative disease”or“viral vector”or“adeno-associated virus”or“Alzheimer’s disease”or“Parkinson’s disease”or“Huntington’s disease”or“amyotrophic lateral sclerosis”or“spinal muscular atrophy”or“Canavan disease”or“Metachromatic leukodystrophy”.Eligibility criteria were as follows:All articles and reviews regarding AAV-mediated gene therapy for neurodegenerative diseases or any specific neurodegenerative disease of the central nervous system were included.In addition，we searched the reference lists of the retrieved papers for relevant articles.The full texts of the relevant articles were read to extract information on the topic of interest（summarized in Table 1）.The procedures used generalized AAV-mediated gene therapy for the different neurodegenerative diseases of the central nervous system as follows.