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Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response

AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues. However, the AAV input doses required were prohibitively high and posed serious risk of toxicity. Here, we performed AAV-CRISPR mediated ho...

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Detalles Bibliográficos
Autores principales:	He, Xiangjun, Zhang, Zhenjie, Xue, Junyi, Wang, Yaofeng, Zhang, Siqi, Wei, Junkang, Zhang, Chenzi, Wang, Jue, Urip, Brian Anugerah, Ngan, Chun Christopher, Sun, Junjiang, Li, Yuefeng, Lu, Zhiqian, Zhao, Hui, Pei, Duanqing, Li, Chi-Kong, Feng, Bo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9700734/ https://www.ncbi.nlm.nih.gov/pubmed/36434000 http://dx.doi.org/10.1038/s41467-022-34898-y

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