A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

Giang N. Nguyen(Children's Hospital of Philadelphia), J.K. Everett(University of Pennsylvania), Samita Kafle(Children's Hospital of Philadelphia), Aoife M. Roche(University of Pennsylvania), Hayley E. Raymond(University of Pennsylvania), Jacob S. Leiby(University of Pennsylvania), Christian M. Wood(Children's Hospital of Philadelphia), Charles‐Antoine Assenmacher(University of Pennsylvania), Elizabeth P. Merricks(University of North Carolina at Chapel Hill), Christian Long(University of North Carolina at Chapel Hill), Haig H. Kazazian(Johns Hopkins University), Timothy C. Nichols(University of North Carolina at Chapel Hill), Frederic D. Bushman(University of Pennsylvania), Denise E. Sabatino(Children's Hospital of Philadelphia)
Nature Biotechnology
November 16, 2020
Cited by 442Open Access
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