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,
Giacomo Maria Bacci,
Elisa Marziali,
Andrea Sodi,
Franco Bambi,
Roberto Caputo
Affiliations
Valentina Becherucci
Cell Factory Meyer, Children’s Hospital A. Meyer Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Florence, 50139 Florence, Italy
Giacomo Maria Bacci
Pediatric Ophthalmology Unit, Children’s Hospital A. Meyer Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Florence, 50139 Florence, Italy
Elisa Marziali
Pediatric Ophthalmology Unit, Children’s Hospital A. Meyer Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Florence, 50139 Florence, Italy
Andrea Sodi
Department of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, 50139 Florence, Italy
Franco Bambi
Cell Factory Meyer, Children’s Hospital A. Meyer Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Florence, 50139 Florence, Italy
Roberto Caputo
Pediatric Ophthalmology Unit, Children’s Hospital A. Meyer Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS), University of Florence, 50139 Florence, Italy
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.
