title: Let There Be Light: Gene and Cell Therapy for Blindness.
abstract: Retinal degenerative diseases are a leading cause of irreversible blindness. Retinal cell death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa, Stargardt disease, and Leber congenital amaurosis, as well as in complex age-related diseases such as age-related macular degeneration. For these blinding conditions, gene and cell therapy approaches offer therapeutic intervention at various disease stages. The present review outlines advances in therapies for retinal degenerative disease, focusing on the progress and challenges in the development and clinical translation of gene and cell therapies. A significant body of preclinical evidence and initial clinical results pave the way for further development of these cutting edge treatments for patients with retinal degenerative disorders.
authors.affiliation: 1 Sorbonne Universités, UPMC Université Paris 06, INSERM, CNRS, Institut de la Vision , France .
authors.forename: Deniz
authors.initials: D
authors.lastname: Dalkara
authors.affiliation: 1 Sorbonne Universités, UPMC Université Paris 06, INSERM, CNRS, Institut de la Vision , France .
authors.forename: Olivier
authors.initials: O
authors.lastname: Goureau
authors.affiliation: 1 Sorbonne Universités, UPMC Université Paris 06, INSERM, CNRS, Institut de la Vision , France .
authors.forename: Katia
authors.initials: K
authors.lastname: Marazova
authors.affiliation: 1 Sorbonne Universités, UPMC Université Paris 06, INSERM, CNRS, Institut de la Vision , France .|2 Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts , DHU Sight Restore, INSERM-DHOS CIC 1423, France .|3 Fondation Ophtalmologique Adolphe de Rothschild, Paris, France .
authors.forename: José-Alain
authors.initials: JA
authors.lastname: Sahel
mesh_terms: D000260:Adenoviruses, Human / Q000235:genetics; D001766:Blindness / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D064113:CRISPR-Cas Systems; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000737:chemistry / Q000378:metabolism; D006801:Humans; D057130:Leber Congenital Amaurosis / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D016086:Lentivirus / Q000235:genetics; D008268:Macular Degeneration / Q000151:congenital* / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D062308:Optogenetics / Q000379:methods; D012160:Retina / Q000378:metabolism / Q000473:pathology; D012174:Retinitis Pigmentosa / Q000235:genetics / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D000080362:Stargardt Disease; D019745:cis-trans-Isomerases / Q000235:genetics / Q000378:metabolism; D000099178:Retinoid Isomerohydrolase
chemical_list: D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase