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

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 the Obl al...

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Detalles Bibliográficos
Autores principales: Tao, Yong, Lamas, Veronica, Du, Wan, Zhu, Wenliang, Li, Yiran, Whittaker, Madelynn N., Zuris, John A., Thompson, David B., Rameshbabu, Arun Prabhu, Shu, Yilai, Gao, Xue, Hu, Johnny H., Pei, Charles, Kong, Wei-Jia, Liu, Xuezhong, Wu, Hao, Kleinstiver, Benjamin P., Liu, David R., Chen, Zheng-Yi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10427623/
https://www.ncbi.nlm.nih.gov/pubmed/37582836
http://dx.doi.org/10.1038/s41467-023-40476-7

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