Persistence of non-viral vector mediated RPE65 expression: Case for viability as a gene transfer therapy for RPE-based diseases-Reference-Cited by-同舟云学术

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Persistence of non-viral vector mediated RPE65 expression: Case for viability as a gene transfer therapy for RPE-based diseases

Published:2013-12 Issue:3 Volume:172 Page:745-752
ISSN:0168-3659
Container-title:Journal of Controlled Release
language:en
Short-container-title:Journal of Controlled Release

Author:

Koirala Adarsha,Conley Shannon M.,Makkia Rasha,Liu Zhao,Cooper Mark J.,Sparrow Janet R.,Naash Muna I.

Funder

National Eye Institute

Foundation Fighting Blindness

Oklahoma Center for the Advancement of Science and Technology

State of Ohio Biomedical Research Commercialization Program

Publisher

Elsevier BV

Subject

Pharmaceutical Science

Reference54 articles.

1. Leber Congenital Amaurosis — a model for efficient genetic testing of heterogeneous disorders: LXIV Edward Jackson Memorial Lecture;Stone;Am. J. Ophthalmol.,2007

2. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber Congenital Amaurosis;Morimura;Proc. Natl. Acad. Sci. U. S. A.,1998

3. Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle;Redmond;Nat. Genet.,1998

4. Human RPE65 gene therapy for Leber Congenital Amaurosis: persistence of early visual improvements and safety at 1year;Cideciyan;Hum. Gene Ther.,2009

5. 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;Hauswirth;Hum. Gene Ther.,2008

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