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

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

Here, the authors develop an AAV-CRISPR mediated somatic knock-in. They apply this system to restore hemostasis in neonatal hemophilia B mice and show liver-specificity of the knock-in and low serum antibody production.

Bibliographic Details
Main Authors: Xiangjun He, Zhenjie Zhang, Junyi Xue, Yaofeng Wang, Siqi Zhang, Junkang Wei, Chenzi Zhang, Jue Wang, Brian Anugerah Urip, Chun Christopher Ngan, Junjiang Sun, Yuefeng Li, Zhiqian Lu, Hui Zhao, Duanqing Pei, Chi-Kong Li, Bo Feng
Format: Article
Language:English
Published: Nature Portfolio 2022-11-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-34898-y

Internet

https://doi.org/10.1038/s41467-022-34898-y

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