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The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies

Retinitis pigmentosa, defined more properly as cone–rod dystrophy, is a paradigm of inherited diffuse retinal dystrophies, one of the rare diseases with the highest prevalence in the worldwide population and one of the main causes of low vision in the pediatric and elderly age groups. Advancements i...

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Autores principales: Becherucci, Valentina, Bacci, Giacomo Maria, Marziali, Elisa, Sodi, Andrea, Bambi, Franco, Caputo, Roberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604477/
https://www.ncbi.nlm.nih.gov/pubmed/37893030
http://dx.doi.org/10.3390/biomedicines11102656
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author Becherucci, Valentina
Bacci, Giacomo Maria
Marziali, Elisa
Sodi, Andrea
Bambi, Franco
Caputo, Roberto
author_facet Becherucci, Valentina
Bacci, Giacomo Maria
Marziali, Elisa
Sodi, Andrea
Bambi, Franco
Caputo, Roberto
author_sort Becherucci, Valentina
collection PubMed
description Retinitis pigmentosa, defined more properly as cone–rod dystrophy, is a paradigm of inherited diffuse retinal dystrophies, one of the rare diseases with the highest prevalence in the worldwide population and one of the main causes of low vision in the pediatric and elderly age groups. Advancements in and the understanding of molecular biology and gene-editing technologies have raised interest in laying the foundation for new therapeutic strategies for rare diseases. As a consequence, new possibilities for clinicians and patients are arising due to the feasibility of treating such a devastating disorder, reducing its complications. The scope of this review focuses on the pathomolecular mechanisms underlying RP better to understand the prospects of its treatment using innovative approaches.
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spelling pubmed-106044772023-10-28 The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies Becherucci, Valentina Bacci, Giacomo Maria Marziali, Elisa Sodi, Andrea Bambi, Franco Caputo, Roberto Biomedicines Review Retinitis pigmentosa, defined more properly as cone–rod dystrophy, is a paradigm of inherited diffuse retinal dystrophies, one of the rare diseases with the highest prevalence in the worldwide population and one of the main causes of low vision in the pediatric and elderly age groups. Advancements in and the understanding of molecular biology and gene-editing technologies have raised interest in laying the foundation for new therapeutic strategies for rare diseases. As a consequence, new possibilities for clinicians and patients are arising due to the feasibility of treating such a devastating disorder, reducing its complications. The scope of this review focuses on the pathomolecular mechanisms underlying RP better to understand the prospects of its treatment using innovative approaches. MDPI 2023-09-28 /pmc/articles/PMC10604477/ /pubmed/37893030 http://dx.doi.org/10.3390/biomedicines11102656 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
Becherucci, Valentina
Bacci, Giacomo Maria
Marziali, Elisa
Sodi, Andrea
Bambi, Franco
Caputo, Roberto
The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title_full The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title_fullStr The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title_full_unstemmed The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title_short The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: A Narrative Review of Pathomolecular Mechanisms for the Development of Cell-Based Therapies
title_sort new era of therapeutic strategies for the treatment of retinitis pigmentosa: a narrative review of pathomolecular mechanisms for the development of cell-based therapies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604477/
https://www.ncbi.nlm.nih.gov/pubmed/37893030
http://dx.doi.org/10.3390/biomedicines11102656
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