CRISPR/Cas9-Mediated in vivo Genetic Correction in a Mouse Model of Hemophilia A

CRISPR/Cas9-Mediated in vivo Genetic Correction in a Mouse Model of Hemophilia A

Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII), has long been considered an attractive target for gene therapy studies. However, full-length F8 cDNA cannot be packaged efficiently by adeno-associated virus (AAV) vectors. As the second most pre...

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Bibliographic Details
Main Authors: Sanchuan Luo, Zhongxiang Li, Xin Dai, Rui Zhang, Zhibing Liang, Wenzhou Li, Ming Zeng, Jinfeng Su, Jun Wang, Xia Liang, Yong Wu, Desheng Liang
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-08-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
CIRSPR/SaCas9
hemophilia A
intron 1 inversion
gene therapy
gene editing
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2021.672564/full

Internet

https://www.frontiersin.org/articles/10.3389/fcell.2021.672564/full

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