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Characteristics and advantages of adenoassociated virus vector-mediated gene therapy for neurodegenerative diseases 预览

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摘要 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.
出处 《中国神经再生研究:英文版》 SCIE CAS CSCD 2019年第6期931-938,共8页 Neural Regeneration Research
作者简介 Correspondence to: Rui Li, MD, PhD, L_rui@jlu.edu.cn.
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