CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs

Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes. Well-characterized animal models are valuable for determining the pathobiology of the disease...

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Veröffentlicht in:Haematologica
Hauptverfasser: Chen, Jiahuan, An, Beiying, Yu, Biao, Peng, Xiaohuan, Yuan, Hongming, Yang, Qiangbing, Chen, Xue, Yu, Tingting, Wang, Lingyu, Zhang, Xinwei, Wang, He, Zou, Xiaodong, Pang, Daxin, Ouyang, Hongsheng, Tang, Xiaochun
Format: Artigo
Sprache:Inglês
Veröffentlicht: Fondazione Ferrata Storti 2020
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7927883/
https://ncbi.nlm.nih.gov/pubmed/31974191
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2019.224063
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