Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye-Reference-Cited by-同舟云学术

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Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

Published:2018-12-27 Issue:2 Volume:36 Page:
ISSN:0724-8741
Container-title:Pharmaceutical Research
language:en
Short-container-title:Pharm Res

Author:

Rodrigues Gerard A.,Shalaev Evgenyi,Karami Thomas K.,Cunningham James,Slater Nigel K. H.,Rivers Hongwen M.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Organic Chemistry,Pharmaceutical Science,Pharmacology,Molecular Medicine,Biotechnology

Link

http://link.springer.com/article/10.1007/s11095-018-2554-7/fulltext.html

Reference135 articles.

1. Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science. 2018;359(6372):eaan4672.

2. 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(21):2231–9.

3. Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, et al. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008;19(10):979–90.

4. Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008;358(21):2240–8.

5. Naldini L. Medicine. A comeback for gene therapy. Science. 2009;326(5954):805–6.

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