Allele-Specific Knockout by CRISPR/Cas to Treat Autosomal Dominant Retinitis Pigmentosa Caused by the G56R Mutation in NR2E3

Allele-Specific Knockout by CRISPR/Cas to Treat Autosomal Dominant Retinitis Pigmentosa Caused by the G56R Mutation in NR2E3

Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes progressive vision loss. The G56R mutation in NR2E3 is the second most common mutation causing autosomal dominant (ad) RP, a transcription factor that is essential for photoreceptor development and maintenance. The G56R variant...

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Bibliographic Details
Main Authors: Michalitsa Diakatou, Gregor Dubois, Nejla Erkilic, Carla Sanjurjo-Soriano, Isabelle Meunier, Vasiliki Kalatzis
Format: Article
Language:English
Published: MDPI AG 2021-03-01
Series:International Journal of Molecular Sciences
Subjects:
inherited retinal dystrophies
autosomal dominant retinitis pigmentosa
rod-cone dystrophy
NR2E3
CRISPR/Cas
photoreceptor
Online Access:https://www.mdpi.com/1422-0067/22/5/2607

Internet

https://www.mdpi.com/1422-0067/22/5/2607

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