Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease-Reference-Cited by-同舟云学术

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Published:2015-09-29 Issue:23 Volume:24 Page:6811-6825
ISSN:0964-6906
Container-title:Human Molecular Genetics
language:en
Short-container-title:Hum. Mol. Genet.

Author:

Trapani Ivana,Toriello Elisabetta,de Simone Sonia,Colella Pasqualina,Iodice Carolina,Polishchuk Elena V.,Sommella Andrea,Colecchi Linda,Rossi Settimio,Simonelli Francesca,Giunti Massimo,Bacci Maria L.,Polishchuk Roman S.,Auricchio Alberto

Funder

European Research Council/ERC

Italian Telethon Foundation

Publisher

Oxford University Press (OUP)

Subject

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

Link

http://academic.oup.com/hmg/article-pdf/24/23/6811/17262849/ddv386.pdf

Reference71 articles.

1. A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Starqardt macular dystrophy

2. Defective lipid transport and biosynthesis in recessive and dominant Stargardt macular degeneration

3. Insights into the Function of Rim Protein in Photoreceptors and Etiology of Stargardt's Disease from the Phenotype in abcr Knockout Mice

4. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

5. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

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