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Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction

In vivo genome editing represents a powerful strategy for both understanding basic biology and treating inherited diseases. However, it remains a challenge to develop universal and efficient in vivo genome-editing tools for tissues that comprise diverse cell types in either a dividing or non-dividin...

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Publicado en:Cell Res
Autores principales: Suzuki, Keiichiro, Yamamoto, Mako, Hernandez-Benitez, Reyna, Li, Zhe, Wei, Christopher, Soligalla, Rupa Devi, Aizawa, Emi, Hatanaka, Fumiyuki, Kurita, Masakazu, Reddy, Pradeep, Ocampo, Alejandro, Hishida, Tomoaki, Sakurai, Masahiro, Nemeth, Amy N., Nuñez Delicado, Estrella, Campistol, Josep M., Magistretti, Pierre, Guillen, Pedro, Rodriguez Esteban, Concepcion, Gong, Jianhui, Yuan, Yilin, Gu, Ying, Liu, Guang-Hui, López-Otín, Carlos, Wu, Jun, Zhang, Kun, Izpisua Belmonte, Juan Carlos
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group UK 2019
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6796851/
https://ncbi.nlm.nih.gov/pubmed/31444470
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41422-019-0213-0
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