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

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/Cas9 system...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:EMBO Mol Med
Päätekijät: Guan, Yuting, Ma, Yanlin, Li, Qi, Sun, Zhenliang, Ma, Lie, Wu, Lijuan, Wang, Liren, Zeng, Li, Shao, Yanjiao, Chen, Yuting, Ma, Ning, Lu, Wenqing, Hu, Kewen, Han, Honghui, Yu, Yanhong, Huang, Yuanhua, Liu, Mingyao, Li, Dali
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: John Wiley and Sons Inc. 2016
Aiheet:
Research Articles
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC5125832/
https://ncbi.nlm.nih.gov/pubmed/26964564
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201506039
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