Article
CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.
Human genetics - 1 Jul 2017
Huai Cong, Jia Chenqiang, Sun Ruilin, Xu Peipei, Min Taishan, Wang Qihan, Zheng Chengde, Chen Hongyan, Lu Daru
Abstract excerpt
Hemophilia B (HB) is an X-linked disorder caused by defects of F9 encoded coagulation factor IX, which is an ideal model for gene therapy. Most existing HB gene therapies are based on viral mediated gene supplementation, which could increase immunoreaction. In this study, CRISPR/Cas9 system was used for gene correction in an F9 mutant HB mouse model in both adult mice (in vivo) and in germline cells (ex vivo). In...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.