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
Database: MEDLINE Ultimate
Description
ISSN:1546-1696
DOI:10.1038/s41587-020-0741-7