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

被引：38

作者：

Qu, Yuan ^{[1
]}

Liu, Yi ^{[2
]}

Noor, Ahmed Fayyaz ^{[3
]}

Tran, Johnathan ^{[4
]}

Li, Rui ^{[1
]}

机构：

[1] Jilin Univ, Dept Hand Surg, Hosp 2, Changchun, Jilin, Peoples R China

[2] Shandong Univ, Dept Orthoped, Qilu Hosp, Jinan, Shandong, Peoples R China

[3] Univ Massachusetts, Dept Chem, Lowell, MA USA

[4] Massachusetts Coll Pharm & Hlth Sci, Dept Premed & Hlth Studies, Boston, MA USA

来源：

NEURAL REGENERATION RESEARCH | 2019年 / 14卷 / 06期

关键词：

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; MUSCULAR-ATROPHY PHENOTYPE; MOUSE MODEL; ALZHEIMERS-DISEASE; CANAVAN-DISEASE; VIRAL VECTORS; DOUBLE-BLIND; AAV VECTORS; SPINAL-CORD; DELIVERY; BRAIN;

D O I：

10.4103/1673-5374.250570

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

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.

引用

页码：931 / 938

页数：8

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