AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa

AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa

Retinitis pigmentosa (RP) consists of a group of inherited, retinal degenerative disorders and is characterized by progressive loss of rod photoreceptors and eventual degeneration of cones in advanced stages, resulting in vision loss or blindness. Gene therapy has been effective in treating autosoma...

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Bibliographic Details
Main Authors: Saba Shahin, Hui Xu, Bin Lu, Augustus Mercado, Melissa K. Jones, Benjamin Bakondi, Shaomei Wang
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:Pharmaceutics
Subjects:
autosomal dominant retinitis pigmentosa
Rhodopsin P23H
CRISPR/Cas9
AAV delivery
allele-specific ablation
photoreceptors
Online Access:https://www.mdpi.com/1999-4923/14/4/824

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https://www.mdpi.com/1999-4923/14/4/824

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