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Article

CRISPR-Mediated In Situ Introduction or Integration of F9-Padua in Human iPSCs for Gene Therapy of Hemophilia B.

International journal of molecular sciences - 19 May 2023

Tang Qiyu, Hu Zhiqing, Zhao Junya, Zhou Tao, Tang Shuqing, Wang Peiyun, Xiao Rou, Chen Yan, Wu Lingqian, Zhou Miaojin, Liang Desheng

Abstract excerpt

Hemophilia B (HB) is an X-linked recessive disease caused by F9 gene mutation and functional coagulation factor IX (FIX) deficiency. Patients suffer from chronic arthritis and death threats owing to excessive bleeding. Compared with traditional treatments, gene therapy for HB has obvious advantages, especially when the hyperactive FIX mutant (FIX-Padua) is used. However, the mechanism by which FIX-Padua works...

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PMID: 37240366
DOI: 10.3390/ijms24109013
PMCID: PMC10219373

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