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Neural Regen Res. 2019 Jun;14(6):931-938. doi: 10.4103/1673-5374.250570.

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

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Department of Hand Surgery, the Second Hospital of Jilin University, Changchun, Jilin Province, China.
Department of Orthopedics, Qilu Hospital of Shandong University, Jinan, Shandong Province, China.
Department of Chemistry, University of Massachusetts Lowell, Lowell, MA, USA.
Department of Premedical and Health Studies, Massachusetts College of Pharmacy and Health Sciences, Boston, MA, USA.


Common neurodegenerative diseases of the central nervous system are characterized by progressive damage to the function of neurons, even leading to the permanent loss of function. Gene therapy via gene replacement or gene correction provides the potential for transformative therapies to delay or possibly stop further progression of the neurodegenerative disease in affected patients. Adeno-associated virus has been the vector of choice in recent clinical trials of therapies for neurodegenerative diseases due to its safety and efficiency in mediating gene transfer to the central nervous system. This review aims to discuss and summarize the progress and clinical applications of adeno-associated virus in neurodegenerative disease in central nervous system. Results from some clinical trials and successful cases of central neurodegenerative diseases deserve further study and exploration.


Alzheimer's disease; Huntington's disease; Parkinson's disease; adeno-associated virus; amyotrophic lateral sclerosis; central nervous system; gene therapy; nerve regeneration; neural regeneration; neurodegenerative disease; spinal muscular atrophy; viral vector

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