Preserved Outer Retina in AIPL1 Leber's Congenital Amaurosis: Implications for Gene Therapy-Reference-Cited by-同舟云学术

Preserved Outer Retina in AIPL1 Leber's Congenital Amaurosis: Implications for Gene Therapy

Published:2015-04 Issue:4 Volume:122 Page:862-864
ISSN:0161-6420
Container-title:Ophthalmology
language:en
Short-container-title:Ophthalmology

Author:

Aboshiha Jonathan,Dubis Adam M.,van der Spuy Jacqueline,Nishiguchi Koji M.,Cheeseman Edward W.,Ayuso Carmen,Ehrenberg Miriam,Simonelli Francesca,Bainbridge James W.,Michaelides Michel

Publisher

Elsevier BV

Subject

Ophthalmology

Reference5 articles.

1. Leber congenital amaurosis;Weleber,2004

2. Effect of gene therapy on visual function in Leber's congenital amaurosis;Bainbridge;N Engl J Med,2008

3. AIPL1, the protein that is defective in Leber congenital amaurosis, is essential for the biosynthesis of retinal rod cGMP phosphodiesterase;Liu;Proc Natl Acad Sci U S A,2004

4. Residual electroretinograms in young Leber congenital amaurosis patients with mutations of AIPL1;Pennesi;Invest Ophthalmol Vis Sci,2011

5. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors;Tan;Hum Mol Genet,2009

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