The new era of therapeutic strategies for the treatment of retinitis pigmentosa: A narrative review of pathomolecular mechanisms for the development of cell-based …

V Becherucci, GM Bacci, E Marziali, A Sodi, F Bambi… - Biomedicines, 2023 - mdpi.com
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 …

[HTML][HTML] Gene therapy and therapeutic editing with outer or inner retina animal models

W Baehr, SH Tsang - Vision Research, 2023 - Elsevier
This Special Issue contains 16 contributions focusing on gene augmentation and genome
editing methodologies that have the potential of delaying or curing inherited retinal diseases …

Genotypic and phenotypic characterization of a cohort of patients affected by rod CNG channel-associated retinitis pigmentosa

L Colombo, G Bonetti, PE Maltese, G Iarossi… - Ophthalmic …, 2024 - karger.com
Abstract Introduction: Retinitis pigmentosa (RP), a heterogeneous inherited retinal disorder
causing gradual vision loss, affects over 1 million people worldwide. Pathogenic variants in …

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

V Becherucci, GM Bacci, E Marziali, A Sodi, F Bambi… - 2023 - preprints.org
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 …