Promising and delivering gene therapies for vision loss

被引：34

作者：

Carvalho, Livia S. ^{[1
]}

Vandenberghe, Luk H. ^{[1
]}

机构：

[1] Harvard Univ, Schepens Eye Res Inst, Massachusetts Eye & Ear Infirm, Dept Ophthalmol,Med Sch, Boston, MA 02114 USA

来源：

VISION RESEARCH | 2015年 / 111卷

关键词：

Retina; Gene therapy; AAV; Lentiviral vector; LCA; Clinical trial; LEBER CONGENITAL AMAUROSIS; ADENOASSOCIATED VIRAL VECTORS; CILIARY NEUROTROPHIC FACTOR; LONG-TERM; RETINAL DEGENERATION; VIRUS VECTORS; SUBRETINAL INJECTION; VISUAL FUNCTION; MOUSE MODEL; RETINITIS-PIGMENTOSA;

D O I：

10.1016/j.visres.2014.07.013

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

The maturity in our understanding of the genetics and the pathogenesis of disease in degenerative retinal disorders has intersected in past years with a novel treatment paradigm in which a genetic intervention may lead to sustained therapeutic benefit, and in some cases even restoration of vision. Here, we review this prospect of retinal gene therapy, discuss the enabling technologies that have led to first-in-human demonstrations of efficacy and safety, and the road that led to this exciting point in time. (C) 2014 Elsevier Ltd. All rights reserved.

引用

页码：124 / 133

页数：10

共 112 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] Gene therapy restores vision in a canine model of childhood blindness [J].

Acland, GM ;

Aguirre, GD ;

Ray, J ;

Zhang, Q ;

Aleman, TS ;

Cideciyan, AV ;

Pearce-Kelling, SE ;

Anand, V ;

Zeng, Y ;

Maguire, AM ;

Jacobson, SG ;

Hauswirth, WW ;

Bennett, J .

NATURE GENETICS, 2001, 28 (01) :92-95

[3] Retinal Laminar architecture in human retinitis pigmentosa caused by Rhodopsin gene mutations [J].

Aleman, Tomas S. ;

Cideciyan, Artur V. ;

Sumaroka, Alexander ;

Windsor, Elizabeth A. M. ;

Herrera, Waldo ;

White, D. Alan ;

Kaushal, Shalesh ;

Naidu, Anjani ;

Roman, Alejandro J. ;

Schwartz, Sharon B. ;

Stone, Edwin M. ;

Jacobson, Samuel G. .

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2008, 49 (04) :1580-1590

[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] Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice [J].

Allocca, Mariacarmela ;

Doria, Monica ;

Petrillo, Marco ;

Colella, Pasqualina ;

Garcia-Hoyos, Maria ;

Gibbs, Daniel ;

Kim, So Ra ;

Maguire, Albert ;

Rex, Tonia S. ;

Di Vicino, Umberto ;

Cutillo, Luisa ;

Sparrow, Janet R. ;

Williams, David S. ;

Bennett, Jean ;

Auricchio, Alberto .

JOURNAL OF CLINICAL INVESTIGATION, 2008, 118 (05) :1955-1964

[6] Successful Gene Therapy in Older Rpe65-Deficient Dogs Following Subretinal Injection of an Adeno-Associated Vector Expressing RPE65 [J].

Annear, Matthew J. ;

Mowat, Freya M. ;

Bartoe, Joshua T. ;

Querubin, Janice ;

Azam, Selina A. ;

Basche, Mark ;

Curran, Paul G. ;

Smith, Alexander J. ;

Bainbridge, James W. B. ;

Ali, Robin R. ;

Petersen-Jones, Simon M. .

HUMAN GENE THERAPY, 2013, 24 (10) :883-893

[7] Gene therapy progress and prospects: the eye [J].

Bainbridge, J. W. B. ;

Tan, M. H. ;

Ali, R. R. .

GENE THERAPY, 2006, 13 (16) :1191-1197

[8] Effect of gene therapy on visual function in Leber's congenital amaurosis [J].

Bainbridge, James W. B. ;

Smith, Alexander J. ;

Barker, Susie S. ;

Robbie, Scott ;

Henderson, Robert ;

Balaggan, Kamaljit ;

Viswanathan, Ananth ;

Holder, Graham E. ;

Stockman, Andrew ;

Tyler, Nick ;

Petersen-Jones, Simon ;

Bhattacharya, Shomi S. ;

Thrasher, Adrian J. ;

Fitzke, Fred W. ;

Carter, Barrie J. ;

Rubin, Gary S. ;

Moore, Anthony T. ;

Ali, Robin R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) :2231-2239

[9] In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium [J].

Bainbridge, JWB ;

Stephens, C ;

Parsley, K ;

Demaison, C ;

Halfyard, A ;

Thrasher, AJ ;

Ali, RR .

GENE THERAPY, 2001, 8 (21) :1665-1668

[10] Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy [J].

Bennett, J ;

Tanabe, T ;

Sun, DX ;

Zeng, Y ;

Kjeldbye, H ;

Gouras, P ;

Maguire, AM .

NATURE MEDICINE, 1996, 2 (06) :649-654

← 1 2 3 4 5 6 7 8 9 10 →