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

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Title: A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Authors: Nguyen GN; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Kafle S; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Roche AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Raymond HE; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Leiby J; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wood C; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Assenmacher CA; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA., Merricks EP; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Long CT; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Kazazian HH; Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA., Nichols TC; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Sabatino DE; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. dsabatin@pennmedicine.upenn.edu.; Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. dsabatin@pennmedicine.upenn.edu.
Source: Nature biotechnology [Nat Biotechnol] 2021 Jan; Vol. 39 (1), pp. 47-55. Date of Electronic Publication: 2020 Nov 16.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature America Publishing Country of Publication: United States NLM ID: 9604648 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-1696 (Electronic) Linking ISSN: 10870156 NLM ISO Abbreviation: Nat Biotechnol Subsets: MEDLINE
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  Data: A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
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  Data: <searchLink fieldCode="AU" term="%22Nguyen+GN%22">Nguyen GN</searchLink>; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Everett+JK%22">Everett JK</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Kafle+S%22">Kafle S</searchLink>; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Roche+AM%22">Roche AM</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Raymond+HE%22">Raymond HE</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Leiby+J%22">Leiby J</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Wood+C%22">Wood C</searchLink>; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Assenmacher+CA%22">Assenmacher CA</searchLink>; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Merricks+EP%22">Merricks EP</searchLink>; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Long+CT%22">Long CT</searchLink>; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Kazazian+HH%22">Kazazian HH</searchLink>; Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Nichols+TC%22">Nichols TC</searchLink>; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Bushman+FD%22">Bushman FD</searchLink>; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Sabatino+DE%22">Sabatino DE</searchLink>; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA. dsabatin@pennmedicine.upenn.edu.; Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. dsabatin@pennmedicine.upenn.edu.
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