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Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies

Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized by the degeneration of rod photoreceptors, followed by the degeneration of cone photoreceptors. As a result of photoreceptor degeneration, affected individuals experience gradual loss of visual function, with...

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Autores principales: Nguyen, Xuan-Thanh-An, Moekotte, Lude, Plomp, Astrid S., Bergen, Arthur A., van Genderen, Maria M., Boon, Camiel J. F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10139437/
https://www.ncbi.nlm.nih.gov/pubmed/37108642
http://dx.doi.org/10.3390/ijms24087481
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author Nguyen, Xuan-Thanh-An
Moekotte, Lude
Plomp, Astrid S.
Bergen, Arthur A.
van Genderen, Maria M.
Boon, Camiel J. F.
author_facet Nguyen, Xuan-Thanh-An
Moekotte, Lude
Plomp, Astrid S.
Bergen, Arthur A.
van Genderen, Maria M.
Boon, Camiel J. F.
author_sort Nguyen, Xuan-Thanh-An
collection PubMed
description Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized by the degeneration of rod photoreceptors, followed by the degeneration of cone photoreceptors. As a result of photoreceptor degeneration, affected individuals experience gradual loss of visual function, with primary symptoms of progressive nyctalopia, constricted visual fields and, ultimately, central vision loss. The onset, severity and clinical course of RP shows great variability and unpredictability, with most patients already experiencing some degree of visual disability in childhood. While RP is currently untreatable for the majority of patients, significant efforts have been made in the development of genetic therapies, which offer new hope for treatment for patients affected by inherited retinal dystrophies. In this exciting era of emerging gene therapies, it remains imperative to continue supporting patients with RP using all available options to manage their condition. Patients with RP experience a wide variety of physical, mental and social-emotional difficulties during their lifetime, of which some require timely intervention. This review aims to familiarize readers with clinical management options that are currently available for patients with RP.
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spelling pubmed-101394372023-04-28 Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies Nguyen, Xuan-Thanh-An Moekotte, Lude Plomp, Astrid S. Bergen, Arthur A. van Genderen, Maria M. Boon, Camiel J. F. Int J Mol Sci Review Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized by the degeneration of rod photoreceptors, followed by the degeneration of cone photoreceptors. As a result of photoreceptor degeneration, affected individuals experience gradual loss of visual function, with primary symptoms of progressive nyctalopia, constricted visual fields and, ultimately, central vision loss. The onset, severity and clinical course of RP shows great variability and unpredictability, with most patients already experiencing some degree of visual disability in childhood. While RP is currently untreatable for the majority of patients, significant efforts have been made in the development of genetic therapies, which offer new hope for treatment for patients affected by inherited retinal dystrophies. In this exciting era of emerging gene therapies, it remains imperative to continue supporting patients with RP using all available options to manage their condition. Patients with RP experience a wide variety of physical, mental and social-emotional difficulties during their lifetime, of which some require timely intervention. This review aims to familiarize readers with clinical management options that are currently available for patients with RP. MDPI 2023-04-19 /pmc/articles/PMC10139437/ /pubmed/37108642 http://dx.doi.org/10.3390/ijms24087481 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Nguyen, Xuan-Thanh-An
Moekotte, Lude
Plomp, Astrid S.
Bergen, Arthur A.
van Genderen, Maria M.
Boon, Camiel J. F.
Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title_full Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title_fullStr Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title_full_unstemmed Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title_short Retinitis Pigmentosa: Current Clinical Management and Emerging Therapies
title_sort retinitis pigmentosa: current clinical management and emerging therapies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10139437/
https://www.ncbi.nlm.nih.gov/pubmed/37108642
http://dx.doi.org/10.3390/ijms24087481
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