Recombinant adeno-associated viral vectors in the nervous system

被引：88

作者：

Burger, C

Nash, K

Mandel, RJ

机构：

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

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

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

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

来源：

HUMAN GENE THERAPY | 2005年 / 16卷 / 07期

关键词：

D O I：

10.1089/hum.2005.16.781

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 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduction efficiencies in various regions of the brain and spinal cord. This review discusses some properties of rAAV that have been studied in the nervous system such as cell tropism, duration of transgene expression, and distribution of viral transduction, as well as immunity and regulation of transgene expression issues, all of which are important for optimization of the use of rAAV in the nervous system.

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收藏

页码：781 / 791

页数：11

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共 136 条

[1] Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors - art. no. 5 [J].

Ahmed, BY ;

Chakravarthy, S ;

Eggers, R ;

Hermens, WTJMC ;

Zhang, JY ;

Niclou, SP ;

Levelt, C ;

Sablitzky, F ;

Anderson, PN ;

Lieberman, AR ;

Verhaagen, J .

BMC NEUROSCIENCE, 2004, 5 (1)

[2] Gene transfer into the mouse retina mediated by an adeno-associated viral vector [J].

Ali, RR ;

Reichel, MB ;

Thrasher, AJ ;

Levinsky, RJ ;

Kinnon, C ;

Kanuga, N ;

Hunt, DM ;

Bhattacharya, SS .

HUMAN MOLECULAR GENETICS, 1996, 5 (05) :591-594

[3] Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors [J].

Alisky, JM ;

Hughes, SM ;

Sauter, SL ;

Jolly, D ;

Dubensky, TW ;

Staber, PD ;

Chiorini, JA ;

Davidson, BL .

NEUROREPORT, 2000, 11 (12) :2669-2673

[4] Constitutive and regulated expression of processed insulin following in vivo hepatic gene transfer [J].

Auricchio, A ;

Gao, GP ;

Yu, QC ;

Raper, S ;

Rivera, VM ;

Clackson, T ;

Wilson, JM .

GENE THERAPY, 2002, 9 (14) :963-971

[5] Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye [J].

Auricchio, A ;

Rivera, VM ;

Clackson, T ;

O'Connor, EE ;

Maguire, AM ;

Tolentino, MJ ;

Bennett, J ;

Wilson, JM .

MOLECULAR THERAPY, 2002, 6 (02) :238-242

[6] Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model [J].

Auricchio, A ;

Kobinger, G ;

Anand, V ;

Hildinger, M ;

O'Connor, E ;

Maguire, AM ;

Wilson, JM ;

Bennett, J .

HUMAN MOLECULAR GENETICS, 2001, 10 (26) :3075-3081

[7] Selective and rapid uptake of adeno-associated virus type 2 in brain [J].

Bartlett, JS ;

Samulski, RJ ;

McCown, TJ .

HUMAN GENE THERAPY, 1998, 9 (08) :1181-1186

[8] Towards a neuroprotective gene therapy for Parkinson's disease:: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat Parkinson model [J].

Björklund, A ;

Kirik, D ;

Rosenblad, C ;

Georgievska, B ;

Lundberg, C ;

Mandel, RJ .

BRAIN RESEARCH, 2000, 886 (1-2) :82-98

[9] Improving rAAV production and purification: towards the definition of a scaleable process [J].

Blouin, V ;

Brument, N ;

Toublanc, E ;

Raimbaud, I ;

Moullier, P ;

Salvetti, A .

JOURNAL OF GENE MEDICINE, 2004, 6 :S223-S228

[10] Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector [J].

Bohl, D ;

Salvetti, A ;

Moullier, P ;

Heard, JM .

BLOOD, 1998, 92 (05) :1512-1517

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