Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs

Open Access

Peer-reviewed

Research Article

Mary Beth Callan,

Affiliation Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America
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Mark E. Haskins,

Affiliation Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America
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Ping Wang,

Affiliation Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America
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Shangzhen Zhou,

Affiliation Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Katherine A. High,

Current address: Spark Therapeutics, Philadelphia, Pennsylvania, United States of America

Affiliations Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America
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Valder R. Arruda

* E-mail: arruda@email.chop.edu

Affiliations Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America
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Dogs
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Hemorrhage
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Gene therapy
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Hemophilia
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Hemophilia A
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Dose prediction methods
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Mutation
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Recombinant proteins
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