Versatility of AAV vectors for retinal gene transfer

被引：111

作者：

Surace, Enrico M. ^{[1
]}

Auricchio, Alberto ^{[1
,2
]}

机构：

[1] Telethon Inst Genet & Med, I-80131 Naples, Italy

[2] Univ Naples Federico II, Dept Pediat, Naples, Italy

来源：

VISION RESEARCH | 2008年 / 48卷 / 03期

关键词：

AAV; gene therapy; retina;

D O I：

10.1016/j.visres.2007.07.027

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

Gene therapy represents a promising therapeutic option for many inherited and acquired retinal diseases. Recombinant adeno-associated viral vectors (AAV) are the most efficient tools to transfer genes in vivo to the retina. The recent identification of dozens of novel AAV serotypes enormously expands on the versatility of AAV as vector system for in vivo somatic gene transfer. The results from the forthcoming trials with AAV in the retina of patients with Leber Congenital Amaurosis will be critical for the rapid development of AAV-based therapeutics for retinal diseases. (c) 2007 Elsevier Ltd. All rights reserved.

引用

收藏

页码：353 / 359

页数：7

相关论文

共 104 条

[1] Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness [J].

Acland, GM ;

Aguirre, GD ;

Bennett, J ;

Aleman, TS ;

Cideciyan, AV ;

Bennicelli, J ;

Dejneka, NS ;

Pearce-Kelling, SE ;

Maguire, AM ;

Palczewski, K ;

Hauswirth, WW ;

Jacobson, SG .

MOLECULAR THERAPY, 2005, 12 (06) :1072-1082

[2]

Acland GM, 2001, NAT GENET, V28, P92, DOI 10.1038/88327

[3] Adeno-associated virus gene transfer to mouse retina [J].

Ali, RR ;

Reichel, MB ;

De Alwis, M ;

Kanuga, N ;

Kinnon, C ;

Levinsky, RJ ;

Hunt, DM ;

Bhattacharya, SS ;

Thrasher, AJ .

HUMAN GENE THERAPY, 1998, 9 (01) :81-86

[4] 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

[5] Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy [J].

Ali, RR ;

Sarra, GM ;

Stephens, C ;

de Alwis, M ;

Bainbridge, JWB ;

Munro, PM ;

Fauser, S ;

Reichell, MB ;

Kinnon, C ;

Hunt, DM ;

Bhattacharya, SS ;

Thrasher, AJ .

NATURE GENETICS, 2000, 25 (03) :306-310

[6] AAV-mediated gene transfer for retinal diseases [J].

Allocca, Mariacarmela ;

Tessitore, Alessandra ;

Cotugno, Gabriella ;

Auricchio, Alberto .

EXPERT OPINION ON BIOLOGICAL THERAPY, 2006, 6 (12) :1279-1294

[7] Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases [J].

Auricchio, A ;

Rolling, F .

CURRENT GENE THERAPY, 2005, 5 (03) :339-348

[8] Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye [J].

Auricchio, A .

VISION RESEARCH, 2003, 43 (08) :913-918

[9] Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents [J].

Auricchio, A ;

Behling, KC ;

Maguire, AM ;

O'Connor, EE ;

Bennett, J ;

Wilson, JM ;

Tolentino, MJ .

MOLECULAR THERAPY, 2002, 6 (04) :490-494

[10] 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

← 1 2 3 4 5 6 7 8 9 10 →