A review of the pharmacokinetics, efficacy and safety of high-purity factor X for the prophylactic treatment of hereditary factor X deficiency.

Autor: Payne J; Department of Paediatric Haematology, Sheffield Children's NHS Foundation Trust, Sheffield, UK., Batsuli G; Department of Pediatrics, Division of Pediatric Hematology and Oncology, Emory University, Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, Atlanta, Georgia, USA., Leavitt AD; Departments of Laboratory Medicine & Medicine (Hematology), University of California San Francisco, San Francisco, California, USA., Mathias M; Great Ormond Street Hospital for Children NHS Foundation Trust, Haemophilia Comprehensive Care Centre, London, UK., McGuinn CE; Department of Pediatrics, Division of Pediatric Hematology Oncology, Weill Cornell Medicine, New York, New York, USA.

Publikováno v: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2022 Jul; Vol. 28 (4), pp. 523-531. Date of Electronic Publication: 2022 May 02.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

Autor: George LA; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Sullivan SK; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Giermasz A; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Rasko JEJ; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Samelson-Jones BJ; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Ducore J; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Cuker A; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Sullivan LM; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Majumdar S; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Teitel J; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., McGuinn CE; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Ragni MV; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Luk AY; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Hui D; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Wright JF; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Chen Y; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Liu Y; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Wachtel K; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Winters A; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Tiefenbacher S; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Arruda VR; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., van der Loo JCM; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Zelenaia O; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Takefman D; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Carr ME; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Couto LB; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., Anguela XM; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.)., High KA; From the Division of Hematology (L.A.G., B.J.S.-J., A.W., V.R.A.) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (L.A.G., B.J.S.-J., A.W., V.R.A., J.C.M.L., O.Z.), Children's Hospital of Philadelphia, the Departments of Pediatrics (L.A.G., B.J.S.-J., V.R.A.) and Medicine (A.C.), Perelman School of Medicine at the University of Pennsylvania, and Spark Therapeutics (A.Y.L., D.H., J.F.W., Y.C., Y.L., K.W., D.T., M.E.C., L.B.C., X.M.A., K.A.H.) - all in Philadelphia; the Department of Pediatrics, Mississippi Center for Advanced Medicine, Madison (S.K.S.), and the Departments of Pathology (L.M.S.) and Pediatrics (S.M.), University of Mississippi Medical School, Jackson; the Departments of Medicine (A.G.) and Pediatrics (J.D.), University of California-Davis Medical School, Sacramento; the Department of Medicine, Sydney Medical School, and the Gene and Stem Cell Therapy Program, Centenary Institute (J.E.J.R.), University of Sydney, and Cell and Molecular Therapies, Royal Prince Alfred Hospital (J.E.J.R.) - both in Camperdown, NSW, Australia; the Department of Medicine, University of Toronto Faculty of Medicine and St. Michael's Hospital, Toronto (J.T.); the Department of Pediatrics, Weill Cornell Medical College, New York (C.E.M.); the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.); and Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO (S.T.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2017 Dec 07; Vol. 377 (23), pp. 2215-2227.