CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

Abstract The X‐linked genetic bleeding disorder caused by deficiency of coagulator factor IX, hemophilia B, is a disease ideally suited for gene therapy with genome editing technology. Here, we identify a family with hemophilia B carrying a novel mutation, Y371D, in the human F9 gene. The CRISPR/Cas...

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Main Authors: Yuting Guan, Yanlin Ma, Qi Li, Zhenliang Sun, Lie Ma, Lijuan Wu, Liren Wang, Li Zeng, Yanjiao Shao, Yuting Chen, Ning Ma, Wenqing Lu, Kewen Hu, Honghui Han, Yanhong Yu, Yuanhua Huang, Mingyao Liu, Dali Li
Format: Article
Language:English
Published: Springer Nature 2016-03-01
Series:EMBO Molecular Medicine
Subjects:
gene therapy
genome editing
hemophilia B
hemostasis
monogenetic disease
Online Access:https://doi.org/10.15252/emmm.201506039
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https://doi.org/10.15252/emmm.201506039

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