Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies

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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Bibliographic Details
Main Authors: Nanda Boon, Jan Wijnholds, Lucie P. Pellissier
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-08-01
Series:Frontiers in Neuroscience
Subjects:
retinitis pigmentosa
leber congenital amaurosis
crumbs homolog 1
gene therapy
mouse model
Online Access:https://www.frontiersin.org/article/10.3389/fnins.2020.00860/full

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