Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

Abstract Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using a mouse model Atp2b2 Obl/+, with a dominant hearing loss mutation (Oblivion), we show that liposome-mediated in vivo delivery of CRISPR-Cas9 ribonucleoprotein complexes leads to specific editing of th...

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Bibliographic Details
Main Authors: Yong Tao, Veronica Lamas, Wan Du, Wenliang Zhu, Yiran Li, Madelynn N. Whittaker, John A. Zuris, David B. Thompson, Arun Prabhu Rameshbabu, Yilai Shu, Xue Gao, Johnny H. Hu, Charles Pei, Wei-Jia Kong, Xuezhong Liu, Hao Wu, Benjamin P. Kleinstiver, David R. Liu, Zheng-Yi Chen
Format: Article
Language:English
Published: Nature Portfolio 2023-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-023-40476-7