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Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies

Retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) are inherited degenerative retinal dystrophies with vision loss that ultimately lead to blindness. Several genes have been shown to be involved in early onset retinal dystrophies, including CRB1 and RPE65. Gene therapy recently became av...

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Detalles Bibliográficos
Autores principales: Boon, Nanda, Wijnholds, Jan, Pellissier, Lucie P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7456964/
https://www.ncbi.nlm.nih.gov/pubmed/32922261
http://dx.doi.org/10.3389/fnins.2020.00860