rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters-Reference-Cited by-同舟云学术

rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

Published:2010-04-29 Issue:9 Volume:17 Page:1162-1174
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Beltran W A,Boye S L,Boye S E,Chiodo V A,Lewin A S,Hauswirth W W,Aguirre G D

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201056.pdf

Reference38 articles.

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2. Pang JJ, Chang B, Kumar A, Nusinowitz S, Noorwez SM, Li J et al. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Mol Ther 2006; 13: 565–572.

3. Weber M, Rabinowitz J, Provost N, Conrath H, Folliot S, Briot D et al. Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol Ther 2003; 7: 774–781.

4. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008; 358: 2231–2239.

5. Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008; 358: 2240–2248.

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