Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis-Reference-Cited by-同舟云学术

Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis

Published:2018-01 Issue:1 Volume:26 Page:256-268
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Le Meur Guylène,Lebranchu Pierre,Billaud Fanny,Adjali Oumeya,Schmitt Sébastien,Bézieau Stéphane,Péréon Yann,Valabregue Romain,Ivan Catherine,Darmon Christophe,Moullier Philippe,Rolling Fabienne,Weber Michel

Funder

AFM-Téléthon (Association Française contre les Myopathies)

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

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3. Review and update on the molecular basis of Leber congenital amaurosis;Chacon-Camacho;World J. Clin. Cases,2015

4. Leber congenital amaurosis: survey of the genetic heterogeneity, refinement of the clinical definition and phenotype-genotype correlations as a strategy for molecular diagnosis. Clinical and molecular survey in LCA;Hanein;Adv. Exp. Med. Biol.,2006

5. Rpe65 is a retinyl ester binding protein that presents insoluble substrate to the isomerase in retinal pigment epithelial cells;Mata;J. Biol. Chem.,2004

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