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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Published inBiomedicines Vol. 11; no. 10; p. 2656
Main Authors Becherucci, Valentina, Bacci, Giacomo Maria, Marziali, Elisa, Sodi, Andrea, Bambi, Franco, Caputo, Roberto
Format Journal Article
LanguageEnglish
Published Basel MDPI AG 28.09.2023
MDPI
Subjects
Analysis
Care and treatment
Cell culture
cell therapies
Dystrophy
Gene therapy
Genes
Genetic aspects
Health aspects
inherited retinal dystrophies
Mutation
Pediatrics
Photoreceptors
Proteins
Rare diseases
Retina
Retinitis pigmentosa
Review
therapeutic strategies
Visual impairment
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Summary: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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These authors contributed equally to this work.
ISSN:2227-9059
2227-9059
DOI:10.3390/biomedicines11102656