Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

The New Era of Therapeutic Strategies for the Treatment of Retinitis Pigmentosa: From Pathomolecular Mechanisms to the Development of Cell-Based Therapies

Valentina Becherucci
,
Giacomo Maria Bacci
* ORCID logo ,
Elisa Marziali
ORCID logo ,
Andrea Sodi
,
Franco Bambi
,
Roberto Caputo
Version 1 : Received: 1 September 2023 / Approved: 4 September 2023 / Online: 4 September 2023 (11:29:44 CEST)

A peer-reviewed article of this Preprint also exists.

Becherucci, V.; Bacci, G.M.; Marziali, E.; Sodi, A.; Bambi, F.; Caputo, R. 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. Biomedicines 2023, 11, 2656. Becherucci, V.; Bacci, G.M.; Marziali, E.; Sodi, A.; Bambi, F.; Caputo, R. 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. Biomedicines 2023, 11, 2656.

Abstract

Retinitis Pigmentosa, defined more properly as rod-cone dystrophy, is the paradigm of diffuse inherited 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 pediatric and elderly age groups. Advancement and understanding in molecular biology and gene editing technologies raised the interest in putting the basis for new therapeutic strategies in 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 is to focus on the pathomolecular mechanisms underlying RP better to understand the prospective for its treatment with innovative approaches.

Keywords

Inherited Retinal Dystrophies; Retinitis Pigmentosa; therapeutic strategies; cell therapies

Subject

Public Health and Healthcare, Primary Health Care

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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