Advances in gene therapy technologies to treat retinitis pigmentosa

Hilda Petrs-Silva, Rafael LindenInstitute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, BrazilAbstract: 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 inherite...

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Autores principales:	Petrs-Silva H, Linden R
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2013
Materias:	Ophthalmology RE1-994
Acceso en línea:	https://doaj.org/article/7f01bffafbc14439ad854c2c4da5fbe4
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spelling	oai:doaj.org-article:7f01bffafbc14439ad854c2c4da5fbe42021-12-02T04:31:05ZAdvances in gene therapy technologies to treat retinitis pigmentosa1177-5483https://doaj.org/article/7f01bffafbc14439ad854c2c4da5fbe42013-12-01T00:00:00Zhttp://www.dovepress.com/advances-in-gene-therapy-technologies-to-treat-retinitis-pigmentosa-a15384https://doaj.org/toc/1177-5483 Hilda Petrs-Silva, Rafael LindenInstitute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, BrazilAbstract: 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, AAVPetrs-Silva HLinden RDove Medical PressarticleOphthalmologyRE1-994ENClinical Ophthalmology, Vol 2014, Iss default, Pp 127-136 (2013)
institution	DOAJ
collection	DOAJ
language	EN
topic	Ophthalmology RE1-994
spellingShingle	Ophthalmology RE1-994 Petrs-Silva H Linden R Advances in gene therapy technologies to treat retinitis pigmentosa
description	Hilda Petrs-Silva, Rafael LindenInstitute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, BrazilAbstract: 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
format	article
author	Petrs-Silva H Linden R
author_facet	Petrs-Silva H Linden R
author_sort	Petrs-Silva H
title	Advances in gene therapy technologies to treat retinitis pigmentosa
title_short	Advances in gene therapy technologies to treat retinitis pigmentosa
title_full	Advances in gene therapy technologies to treat retinitis pigmentosa
title_fullStr	Advances in gene therapy technologies to treat retinitis pigmentosa
title_full_unstemmed	Advances in gene therapy technologies to treat retinitis pigmentosa
title_sort	advances in gene therapy technologies to treat retinitis pigmentosa
publisher	Dove Medical Press
publishDate	2013
url	https://doaj.org/article/7f01bffafbc14439ad854c2c4da5fbe4
work_keys_str_mv	AT petrssilvah advancesingenetherapytechnologiestotreatretinitispigmentosa AT lindenr advancesingenetherapytechnologiestotreatretinitispigmentosa
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Advances in gene therapy technologies to treat retinitis pigmentosa

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