In vivo genome editing restores haemostasis in a mouse model of haemophilia

Hojun Li(Children's Hospital of Philadelphia), Virginia Haurigot(Children's Hospital of Philadelphia), Yannick Doyon(Point Richmond Tech Center), Tianjian Li(Point Richmond Tech Center), Sunnie Wong(Point Richmond Tech Center), Anand Bhagwat(Children's Hospital of Philadelphia), Nirav Malani(University of Pennsylvania), Xavier M. Anguela(Children's Hospital of Philadelphia), Rajiv P. Sharma(Children's Hospital of Philadelphia), Lacramiora Ivanciu(Children's Hospital of Philadelphia), Samuel L. Murphy(Children's Hospital of Philadelphia), Jonathan D. Finn(Children's Hospital of Philadelphia), Fayaz R. Khazi(Children's Hospital of Philadelphia), Shangzhen Zhou(Children's Hospital of Philadelphia), David E. Paschon(Point Richmond Tech Center), Edward J. Rebar(Point Richmond Tech Center), Frederic D. Bushman(University of Pennsylvania), Philip D. Gregory(Sangamo BioSciences (United States)), Michael C. Holmes(Sangamo BioSciences (United States)), Katherine A. High(Howard Hughes Medical Institute)
Nature
June 24, 2011
Cited by 542

Abstract


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