Efficient Rescue of Retinal Degeneration in Pde6a Mice by Engineered Base Editing and Prime Editing

Abstract Retinitis pigmentosa (RP) is a complex spectrum of inherited retinal diseases marked by the gradual loss of photoreceptor cells, ultimately leading to blindness. Among these, mutations in PDE6A, responsible for encoding a cGMP‐specific phosphodiesterase, stand out as pivotal in autosomal re...

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Bibliographic Details
Main Authors: Zhiquan Liu, Siyu Chen, Alexander E. Davis, Chien‐Hui Lo, Qing Wang, Tingting Li, Ke Ning, Qi Zhang, Jingyu Zhao, Sui Wang, Yang Sun
Format: Article
Language:English
Published: Wiley 2024-11-01
Series:Advanced Science
Subjects:
Online Access:https://doi.org/10.1002/advs.202405628
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