A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang Yang(University of Pennsylvania), Lili Wang(University of Pennsylvania), Peter Bell(University of Pennsylvania), Deirdre McMenamin(University of Pennsylvania), Zhenning He(University of Pennsylvania), John White(University of Pennsylvania), Hongwei Yu(University of Pennsylvania), Chenyu Xu(Children's National), Hiroki Morizono(Children's National), Kiran Musunuru(Harvard University), Mark L. Batshaw(Children's National), James M. Wilson(University of Pennsylvania)
Nature Biotechnology
February 1, 2016
Cited by 554Open Access
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