Global spectrum of USH2A mutation in inherited retinal dystrophies: Prompt message for development of base editing therapy

Global spectrum of USH2A mutation in inherited retinal dystrophies: Prompt message for development of base editing therapy

PurposeMutation in the USH2A gene is the most common cause of inherited retinal dystrophy (IRD), including non-syndromic retinitis pigmentosa (RP) and Usher syndrome II (USH2). Gene editing and therapy targeting USH2A, especially the hotspot region, would benefit a large proportion of IRD patients....

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Bibliographic Details
Main Authors: Bing-Nan Su, Ren-Juan Shen, Zhuo-Lin Liu, Yang Li, Zi-Bing Jin
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-08-01
Series:Frontiers in Aging Neuroscience
Subjects:
USH2A
mutation hotspot
Usher syndrome
retinitis pigmentosa
gene editing
inherited retinal dystrophy
Online Access:https://www.frontiersin.org/articles/10.3389/fnagi.2022.948279/full

Internet

https://www.frontiersin.org/articles/10.3389/fnagi.2022.948279/full

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