Zobrazeno 1 - 2
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pro vyhledávání: '"Chun Christopher Ngan"'
Autor:
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
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-17 (2022)
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.
Externí odkaz:
https://doaj.org/article/8c34ac5c54144838ba20e5afe10957da
Publikováno v:
Journal of Molecular Medicine (Berlin, Germany)
Gene therapy has entered a new era after decades-long efforts, where the recombinant adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene transfer and demonstrated excellent efficacy and safety profiles in numerous pr