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Review
. 2023 Sep 28;11(10):2656.
doi: 10.3390/biomedicines11102656.

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

Valentina Becherucci  1 , Giacomo Maria Bacci  2 , Elisa Marziali  2 , Andrea Sodi  3 , Franco Bambi  1 , Roberto Caputo  2
Affiliations
Review

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

Valentina Becherucci et al. Biomedicines. .

Abstract

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.

Keywords: cell therapies; inherited retinal dystrophies; retinitis pigmentosa; therapeutic strategies.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Simplified clinical flow chart for assessing suspected RP. FAF: fundus autofluorescence, OCT: optical coherence tomography, VF: visual field, ffERG: full-field electroretinography, WES: whole-exome sequencing, WGS: whole-genome sequencing.
Figure 2
Figure 2
Schematic representation of general processes underlying retinitis pigmentosa.
Figure 3
Figure 3
Schematic representation of various therapeutic strategies for treating retinitis pigmentosa.
See this image and copyright information in PMC

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