中国神经再生研究(英文版)

中国神经再生研究(英文版) ›› 2019, Vol. 14 ›› Issue (6): 931-938.doi: 10.4103/1673-5374.250570

• 综述:退行性病与再生 • 上一篇    下一篇

腺相关病毒载体介导基因疗法在神经退行性疾病应用中的特点与优势

  

  • 出版日期:2019-06-15 发布日期:2019-06-15

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

Yuan Qu 1 , Yi Liu 2 , Ahmed Fayyaz Noor 3 , Johnathan Tran 4 , Rui Li 1   

  1. 1 Department of Hand Surgery, the Second Hospital of Jilin University, Changchun, Jilin Province, China
    2 Department of Orthopedics, Qilu Hospital of Shandong University, Jinan, Shandong Province, China
    3 Department of Chemistry, University of Massachusetts Lowell, Lowell, MA, USA
    4 Department of Premedical and Health Studies, Massachusetts College of Pharmacy and Health Sciences, Boston, MA, USA
  • Online:2019-06-15 Published:2019-06-15
  • Contact: Rui Li, MD, PhD, L_rui@jlu.edu.cn

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摘要：

中枢神经系统退行性疾病的特征为神经元功能的进行性损伤，甚至导致神经功能的永久丧失。以基因疗法进行基因替换或校正具有临床转化潜力，或可能延迟或抑制神经变性疾病的进展。由于腺相关病毒介导基因转染至中枢神经系统安全、有效且无明显副作用，因而已成为近年来神经退行性疾病临床基因疗法的首选载体。文章总结了腺相关病毒载体介导基因疗法在中枢性神经退行性疾病临床应用的进展，显示了其在神经退行性疾病治疗中具有的前景，且已有在部分中枢性神经退行性疾病的临床试验应用和取得成功的案例，值得进一步研究探索。

orcid: 0000-0002-9922-4308 (Rui Li)

关键词: 中枢神经系统, 基因治疗, 神经退行性疾病, 病毒载体, 腺相关病毒, 阿尔茨海默病, 帕金森病, 亨廷顿病, 肌萎缩侧索硬化, 脊髓性肌萎缩, 神经再生

Abstract:

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 summa¬rize 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.

Key words: nerve regeneration, central nervous system, gene therapy, neurodegenerative disease, viral vector, adeno-associated virus, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis, spinal muscular atrophy, neural regeneration