Article
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
Nature - 26 Jun 2011
Li Hojun, Haurigot Virginia, Doyon Yannick, Li Tianjian, Wong Sunnie Y, Bhagwat Anand S, Malani Nirav, Anguela Xavier M, Sharma Rajiv, Ivanciu Lacramiora, Murphy Samuel L, Finn Jonathan D, Khazi Fayaz R, Zhou Shangzhen, Paschon David E, Rebar Edward J, Bushman Frederic D, Gregory Philip D, Holmes Michael C, High Katherine A
Abstract excerpt
Editing of the human genome to correct disease-causing mutations is a promising approach for the treatment of genetic disorders. Genome editing improves on simple gene-replacement strategies by effecting in situ correction of a mutant gene, thus restoring normal gene function under the control of endogenous regulatory elements and reducing risks associated with random insertion into the genome. Gene-specific...
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