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

收藏

页码：463 / 483

页数：21

相关论文

共 236 条

[91] Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis [J].

Kaspar, BK ;

Frost, LM ;

Christian, L ;

Umapathi, P ;

Gage, FH .

ANNALS OF NEUROLOGY, 2005, 57 (05) :649-655

[92]

KAY MA, 2003, NAT GENET, V34, P251

[93] AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease [J].

Kells, AP ;

Fong, DM ;

Dragunow, M ;

During, MJ ;

Young, D ;

Connor, B .

MOLECULAR THERAPY, 2004, 9 (05) :682-688

[94] Localized striatal delivery of GDNF as a treatment for Parkinson disease [J].

Kirik, D ;

Georgievska, B ;

Björklund, A .

NATURE NEUROSCIENCE, 2004, 7 (02) :105-110

[95] Long-term rAAV-mediated gene transfer of GDNF in the rat parkinson's model:: Intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system [J].

Kirik, D ;

Rosenblad, C ;

Björkland, A ;

Mandel, RJ .

JOURNAL OF NEUROSCIENCE, 2000, 20 (12) :4686-4700

[96] Modeling CNS neurodegeneration by overexpression of disease-causing proteins using viral vectors [J].

Kirik, D ;

Björklund, A .

TRENDS IN NEUROSCIENCES, 2003, 26 (07) :386-392

[97] Nigrostriatal α-synucleinopathy induced by viral vector-mediated overexpression of human α-synuclein:: A new primate model of Parkinson's disease [J].

Kirik, D ;

Annett, LE ;

Burger, C ;

Muzyczka, N ;

Mandel, RJ ;

Björklund, A .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (05) :2884-2889

[98] Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer [J].

Kirik, D ;

Georgievska, B ;

Burger, C ;

Winkler, C ;

Muzyczka, N ;

Mandel, RJ ;

Björklund, A .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (07) :4708-4713

[99] Growth and functional efficacy of intrastriatal nigral transplants depend on the extent of nigrostriatal degeneration [J].

Kirik, D ;

Winkler, C ;

Björklund, A .

JOURNAL OF NEUROSCIENCE, 2001, 21 (08) :2889-2896

[100] Parkinson-like neurodegeneration induced by targeted overexpression of α-synuclein in the nigrostriatal system [J].

Kirik, D ;

Rosenblad, C ;

Burer, C ;

Lundberg, C ;

Johansen, TE ;

Muzyczka, N ;

Mandel, RJ ;

Björklund, A .

JOURNAL OF NEUROSCIENCE, 2002, 22 (07) :2780-2791

← 5 6 7 8 9 10 11 12 13 14 →