S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA-Reference-Cited by-同舟云学术

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S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA

Published:2013-01-18 Issue:8 Volume:22 Page:1632-1642
ISSN:1460-2083
Container-title:Human Molecular Genetics
language:en
Short-container-title:

Author:

Koirala Adarsha,Makkia Rasha S.,Conley Shannon M.,Cooper Mark J.,Naash Muna I.

Publisher

Oxford University Press (OUP)

Subject

Genetics (clinical),Genetics,Molecular Biology,General Medicine

Link

http://academic.oup.com/hmg/article-pdf/22/8/1632/14141116/ddt013.pdf

Reference50 articles.

1. Diseases caused by defects in the visual cycle: retinoids as potential therapeutic agents;Travis;Annu. Rev. Pharmacol. Toxicol.,2007

2. Bestrophins and retinopathies;Xiao;Pflugers Arch.,2010

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

4. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics;Cideciyan;Proc. Natl Acad. Sci. USA,2008

5. Safety and efficacy of gene transfer for Leber's congenital amaurosis;Maguire;N. Engl. J. Med.,2008

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