CRISPR-Mediated In Situ Introduction or Integration of <i>F9-Padua</i> in Human iPSCs for Gene Therapy of Hemophilia B

CRISPR-Mediated In Situ Introduction or Integration of <i>F9-Padua</i> in Human iPSCs for Gene Therapy of Hemophilia B

Hemophilia B (HB) is an X-linked recessive disease caused by <i>F9</i> 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...

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Bibliographic Details
Main Authors: Qiyu Tang, Zhiqing Hu, Junya Zhao, Tao Zhou, Shuqing Tang, Peiyun Wang, Rou Xiao, Yan Chen, Lingqian Wu, Miaojin Zhou, Desheng Liang
Format: Article
Language:English
Published: MDPI AG 2023-05-01
Series:International Journal of Molecular Sciences
Subjects:
hemophilia
CRISPR/Cas9
iPSCs
FIX-Padua
<i>F9-Padua</i> mutation
gene therapy
Online Access:https://www.mdpi.com/1422-0067/24/10/9013

Internet

https://www.mdpi.com/1422-0067/24/10/9013

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