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Current trends in gene therapy for retinal diseases (Review)

The eye is considered an effective target for genetic therapy, as it has a privileged immune status, it is easily accessed for medication delivery and it is affected by a number of inherited disorders. In particular, the retina is considered for gene therapy due to the fact that it can be visualized...

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Autores principales: Moraru, Andreea Dana, Costin, Dănuț, Iorga, Raluca Eugenia, Munteanu, Mihnea, Moraru, Radu Lucian, Branisteanu, Daniel Constantin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: D.A. Spandidos 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8593927/
https://www.ncbi.nlm.nih.gov/pubmed/34815778
http://dx.doi.org/10.3892/etm.2021.10948
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author Moraru, Andreea Dana
Costin, Dănuț
Iorga, Raluca Eugenia
Munteanu, Mihnea
Moraru, Radu Lucian
Branisteanu, Daniel Constantin
author_facet Moraru, Andreea Dana
Costin, Dănuț
Iorga, Raluca Eugenia
Munteanu, Mihnea
Moraru, Radu Lucian
Branisteanu, Daniel Constantin
author_sort Moraru, Andreea Dana
collection PubMed
description The eye is considered an effective target for genetic therapy, as it has a privileged immune status, it is easily accessed for medication delivery and it is affected by a number of inherited disorders. In particular, the retina is considered for gene therapy due to the fact that it can be visualized with ease, it does not have lymphatic vessels, nor a direct blood network for the outer layers and its cells do not divide after birth, and thus transgene expression is not affected. As gene therapy is currently on a continuously progressive development trend, this emerging field of gene manipulation techniques has yielded promising results. This involves the development of treatments for a number of debilitating and blinding diseases, which were to date considered intractable. However, numerous unanswered questions remain as regards the long-term efficacy and safety profile of these treatments. The present review article discusses the current research status regarding genetic manipulation techniques aimed at addressing visual impairment related to retinal disorders, both inherited and degenerative.
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spelling pubmed-85939272021-11-22 Current trends in gene therapy for retinal diseases (Review) Moraru, Andreea Dana Costin, Dănuț Iorga, Raluca Eugenia Munteanu, Mihnea Moraru, Radu Lucian Branisteanu, Daniel Constantin Exp Ther Med Review The eye is considered an effective target for genetic therapy, as it has a privileged immune status, it is easily accessed for medication delivery and it is affected by a number of inherited disorders. In particular, the retina is considered for gene therapy due to the fact that it can be visualized with ease, it does not have lymphatic vessels, nor a direct blood network for the outer layers and its cells do not divide after birth, and thus transgene expression is not affected. As gene therapy is currently on a continuously progressive development trend, this emerging field of gene manipulation techniques has yielded promising results. This involves the development of treatments for a number of debilitating and blinding diseases, which were to date considered intractable. However, numerous unanswered questions remain as regards the long-term efficacy and safety profile of these treatments. The present review article discusses the current research status regarding genetic manipulation techniques aimed at addressing visual impairment related to retinal disorders, both inherited and degenerative. D.A. Spandidos 2022-01 2021-11-05 /pmc/articles/PMC8593927/ /pubmed/34815778 http://dx.doi.org/10.3892/etm.2021.10948 Text en Copyright: © Moraru et al. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License (https://creativecommons.org/licenses/by-nc-nd/4.0/) , which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.
spellingShingle Review
Moraru, Andreea Dana
Costin, Dănuț
Iorga, Raluca Eugenia
Munteanu, Mihnea
Moraru, Radu Lucian
Branisteanu, Daniel Constantin
Current trends in gene therapy for retinal diseases (Review)
title Current trends in gene therapy for retinal diseases (Review)
title_full Current trends in gene therapy for retinal diseases (Review)
title_fullStr Current trends in gene therapy for retinal diseases (Review)
title_full_unstemmed Current trends in gene therapy for retinal diseases (Review)
title_short Current trends in gene therapy for retinal diseases (Review)
title_sort current trends in gene therapy for retinal diseases (review)
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8593927/
https://www.ncbi.nlm.nih.gov/pubmed/34815778
http://dx.doi.org/10.3892/etm.2021.10948
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