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Advances in gene therapy technologies to treat retinitis pigmentosa

Authors Petrs-Silva H, Linden R

Received 26 September 2013

Accepted for publication 5 November 2013

Published 24 December 2013 Volume 2014:8 Pages 127—136


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Hilda Petrs-Silva, Rafael Linden

Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil

Abstract: Retinitis pigmentosa (RP) is a class of diseases that leads to progressive degeneration of the retina. Experimental approaches to gene therapy for the treatment of inherited retinal dystrophies have advanced in recent years, inclusive of the safe delivery of genes to the human retina. This review is focused on the development of gene therapy for RP using recombinant adenoassociated viral vectors, which show a positive safety record and have so far been successful in several clinical trials for congenital retinal disease. Gene therapy for RP is under development in a variety of animal models, and the results raise expectations of future clinical application. Nonetheless, the translation of such strategies to the bedside requires further understanding of the mutations and mechanisms that cause visual defects, as well as thorough examination of potential adverse effects.
Keywords: retinitis pigmentosa, photoreceptor, gene therapy, AAV

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