Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders

被引：103

作者：

Mandel, RJ

Manfredsson, FP

Foust, KD

Rising, A

Reimsnider, S

Nash, K

Burger, C

机构：

[1] Univ Florida, Coll Med, Dept Neurosci, Gainesville, FL 32610 USA

[2] Univ Florida, Coll Med, Dept Pediat, Gainesville, FL 32610 USA

[3] Univ Florida, Coll Med, Dept Mol Genet & Microbiol, Gainesville, FL 32610 USA

[4] Univ Florida, Coll Med, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA

[5] Univ Florida, Coll Med, McKnight Brain Inst, Gainesville, FL 32610 USA

来源：

MOLECULAR THERAPY | 2006年 / 13卷 / 03期

关键词：

Parkinson disease; Alzheimer disease; Huntington disease; lysosomal storage disorder; amyotrophic lateral sclerosis; spinal cord; epilepsy; stroke;

D O I：

10.1016/j.ymthe.2005.11.009

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Recombinant adeno-associated virus (rAAV) is derived from a small human parvovirus with an excellent safety profile. In addition, this viral vector efficiently transduces and supports long-term transgene expression in the nervous system. These properties make rAAV a reasonable candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early stage clinical trials for various neurodegenerative disorders. Therefore, we will review the currently available preclinical data using rAAV in animal models of central nervous system (CNS) disorders. Moreover, potential caveats for rAAV-based gene therapy in the CNS are also presented.

引用

页码：463 / 483

页数：21

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