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pro vyhledávání: '"Anguela XM"'
Akademický článek
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Autor:
Anguela XM; Estuary Biotherapeutics, Inc, Menlo Park, CA., High KA; Rockefeller University, New York, NY.; Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA.
Publikováno v:
Blood advances [Blood Adv] 2024 Apr 09; Vol. 8 (7), pp. 1796-1803.
Autor:
Elkouby L; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.; Spark Therapeutics, Inc., Philadelphia, PA, USA., Armour SM; Spark Therapeutics, Inc., Philadelphia, PA, USA., Toso R; Spark Therapeutics, Inc., Philadelphia, PA, USA., DiPietro M; Spark Therapeutics, Inc., Philadelphia, PA, USA., Davidson RJ; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA., Nguyen GN; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA., Willet M; Spark Therapeutics, Inc., Philadelphia, PA, USA., Kutza S; Spark Therapeutics, Inc., Philadelphia, PA, USA., Silverberg J; Spark Therapeutics, Inc., Philadelphia, PA, USA., Frick J; Spark Therapeutics, Inc., Philadelphia, PA, USA., Crosariol M; Spark Therapeutics, Inc., Philadelphia, PA, USA., Wang Y; Spark Therapeutics, Inc., Philadelphia, PA, USA., Wang C; Spark Therapeutics, Inc., Philadelphia, PA, USA., High KA; Spark Therapeutics, Inc., Philadelphia, PA, USA., Sabatino DE; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Colket Translational Research Building, Rm 5020, Philadelphia, PA 19104, USA.; Division of Hematology, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Anguela XM; Spark Therapeutics, Inc., Philadelphia, PA, USA.
Publikováno v:
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 Nov 24; Vol. 24, pp. 20-29. Date of Electronic Publication: 2021 Nov 24 (Print Publication: 2022).
Autor:
George LA; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Monahan PE; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Eyster ME; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Sullivan SK; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Ragni MV; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Croteau SE; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Rasko JEJ; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Recht M; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Samelson-Jones BJ; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., MacDougall A; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Jaworski K; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Noble R; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Curran M; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Kuranda K; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Mingozzi F; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Chang T; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Reape KZ; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., Anguela XM; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.)., High KA; From the Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania (L.A.G., B.J.S.-J.), the Division of Hematology and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia (L.A.G., B.J.S.-J.), and Spark Therapeutics (P.E.M., A.M., K.J., R.N., M.C., K.K., F.M., T.C., K.Z.R., X.M.A., K.A.H.), Philadelphia, the Department of Medicine, Division of Hematology and Oncology, Penn State Health Milton S. Hershey Medical Center, Hershey (M.E.E.), and the Department of Medicine, University of Pittsburgh, Pittsburgh (M.V.R.) - all in Pennsylvania; the Department of Pediatrics, Division of Hematology, Mississippi Center for Advanced Medicine, Madison (S.K.S.); the Department of Pediatrics, Harvard Medical School, and the Division of Hematology and Oncology, Boston Children's Hospital - both in Boston (S.E.C.); the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney - both in Camperdown, NSW, Australia (J.E.J.R.); the Hemophilia Center, Oregon Health and Science University, Portland (M.R.); and the American Thrombosis and Hemostasis Network, Rochester, NY (M.R.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2021 Nov 18; Vol. 385 (21), pp. 1961-1973.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Autor:
Costa-Verdera H; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.; Sorbonne University Paris and INSERM U974, 75013, Paris, France., Collaud F; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Riling CR; Spark Therapeutics, Philadelphia, PA, 19104, USA., Sellier P; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Nordin JML; Spark Therapeutics, Philadelphia, PA, 19104, USA., Preston GM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Cagin U; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Fabregue J; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Barral S; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Moya-Nilges M; Pasteur Institute, 75015, Paris, France., Krijnse-Locker J; Pasteur Institute, 75015, Paris, France., van Wittenberghe L; Genethon, 91000, Evry, France., Daniele N; Genethon, 91000, Evry, France., Gjata B; Genethon, 91000, Evry, France., Cosette J; Genethon, 91000, Evry, France., Abad C; Université de Rouen Normandie-IRIB, 76183, Rouen, France., Simon-Sola M; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Charles S; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Li M; Spark Therapeutics, Philadelphia, PA, 19104, USA., Crosariol M; Spark Therapeutics, Philadelphia, PA, 19104, USA., Antrilli T; Spark Therapeutics, Philadelphia, PA, 19104, USA., Quinn WJ 3rd; Spark Therapeutics, Philadelphia, PA, 19104, USA., Gross DA; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Boyer O; Université de Rouen Normandie-IRIB, 76183, Rouen, France., Anguela XM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Armour SM; Spark Therapeutics, Philadelphia, PA, 19104, USA., Colella P; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Ronzitti G; Genethon, 91000, Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France., Mingozzi F; Genethon, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France. Federico.mingozzi@sparktx.com.; Sorbonne University Paris and INSERM U974, 75013, Paris, France. Federico.mingozzi@sparktx.com.; Spark Therapeutics, Philadelphia, PA, 19104, USA. Federico.mingozzi@sparktx.com.
Publikováno v:
Nature communications [Nat Commun] 2021 Nov 04; Vol. 12 (1), pp. 6393. Date of Electronic Publication: 2021 Nov 04.
Autor:
George LA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA; Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Ragni MV; Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA., Rasko JEJ; Gene & Stem Cell Therapy Program, Centenary Institute, and Faculty of Medicine and Health, The University of Sydney, Sydney, Australia; Cell and Molecular Therapies, Royal Prince Alfred Hospital, Camperdown, NSW, Australia., Raffini LJ; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Samelson-Jones BJ; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA; Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Ozelo M; Department of Internal Medicine, Faculty of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil; IHTC Hemophilia Unit Cláudio Luiz Pizzigatti Corrêa, INCT do Sangue Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil., Hazbon M; Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Runowski AR; Division of Hematology and Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA., Wellman JA; Spark Therapeutics, Philadelphia, PA, USA., Wachtel K; Spark Therapeutics, Philadelphia, PA, USA., Chen Y; Spark Therapeutics, Philadelphia, PA, USA., Anguela XM; Spark Therapeutics, Philadelphia, PA, USA., Kuranda K; Spark Therapeutics, Philadelphia, PA, USA., Mingozzi F; Spark Therapeutics, Philadelphia, PA, USA., High KA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA; Spark Therapeutics, Philadelphia, PA, USA. Electronic address: khigh@rockefeller.edu.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2020 Sep 02; Vol. 28 (9), pp. 2073-2082. Date of Electronic Publication: 2020 Jun 10.
Autor:
Leborgne C; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Barbon E; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Alexander JM; Spark Therapeutics, Philadelphia, PA, USA., Hanby H; Spark Therapeutics, Philadelphia, PA, USA., Delignat S; Centre de Recherche des Cordeliers, Sorbonne Université, Université de Paris, Paris, France.; Institut National de la Santé et de la Recherche Médicale, Paris, France., Cohen DM; Spark Therapeutics, Philadelphia, PA, USA., Collaud F; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Muraleetharan S; Spark Therapeutics, Philadelphia, PA, USA., Lupo D; Spark Therapeutics, Philadelphia, PA, USA., Silverberg J; Spark Therapeutics, Philadelphia, PA, USA., Huang K; Spark Therapeutics, Philadelphia, PA, USA., van Wittengerghe L; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Marolleau B; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Miranda A; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Fabiano A; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Daventure V; Centre de Recherche des Cordeliers, Sorbonne Université, Université de Paris, Paris, France.; Institut National de la Santé et de la Recherche Médicale, Paris, France., Beck H; Spark Therapeutics, Philadelphia, PA, USA., Anguela XM; Spark Therapeutics, Philadelphia, PA, USA., Ronzitti G; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France., Armour SM; Spark Therapeutics, Philadelphia, PA, USA., Lacroix-Desmazes S; Centre de Recherche des Cordeliers, Sorbonne Université, Université de Paris, Paris, France. sebastien.lacroix-desmazes@crc.jussieu.fr.; Institut National de la Santé et de la Recherche Médicale, Paris, France. sebastien.lacroix-desmazes@crc.jussieu.fr., Mingozzi F; Généthon INTEGRARE UMR-S951 (Institut National de la Santé et de la Recherche Médicale, Université d'Evry, Université Paris Saclay), Evry, France. federico.mingozzi@sparktx.com.; Spark Therapeutics, Philadelphia, PA, USA. federico.mingozzi@sparktx.com.
Publikováno v:
Nature medicine [Nat Med] 2020 Jul; Vol. 26 (7), pp. 1096-1101. Date of Electronic Publication: 2020 Jun 01.
Autor:
Zou C; Division of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY 10065, USA.; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA., Vercauteren KOA; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA.; Laboratory of Liver Infectious Diseases, Ghent University, 9000 Ghent, Belgium., Michailidis E; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA., Kabbani M; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA., Zoluthkin I; Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32603, USA., Quirk C; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA., Chiriboga L; Department of Pathology, NYU Langone Health, New York, NY 10016, USA., Yazicioglu M; Spark Therapeutics, Philadelphia, PA 19104, USA., Anguela XM; Spark Therapeutics, Philadelphia, PA 19104, USA., Meuleman P; Laboratory of Liver Infectious Diseases, Ghent University, 9000 Ghent, Belgium., High KA; Spark Therapeutics, Philadelphia, PA 19104, USA., Herzog RW; Department of Pediatrics, Indiana University, Indianapolis, IN 46202, USA.; Herman B Wells Center for Pediatric Research, IUPUI, Indianapolis, IN 46202, USA., de Jong YP; Division of Gastroenterology and Hepatology, Weill Cornell Medicine, New York, NY 10065, USA.; Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY 10065, USA.
Publikováno v:
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Jun 02; Vol. 18, pp. 189-198. Date of Electronic Publication: 2020 Jun 02 (Print Publication: 2020).
Autor:
Anguela XM; Research & Development Spark Therapeutics Philadelphia Pennsylvania., High KA; Research & Development Spark Therapeutics Philadelphia Pennsylvania.
Publikováno v:
Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2019 Feb 28; Vol. 3 (2), pp. 149-151. Date of Electronic Publication: 2019 Feb 28 (Print Publication: 2019).
Autor:
Anguela XM; Spark Therapeutics, Inc., Philadelphia, Pennsylvania 19104, USA; email: xavier.anguela@sparktx.com , kathy.high@sparktx.com., High KA; Spark Therapeutics, Inc., Philadelphia, Pennsylvania 19104, USA; email: xavier.anguela@sparktx.com , kathy.high@sparktx.com.
Publikováno v:
Annual review of medicine [Annu Rev Med] 2019 Jan 27; Vol. 70, pp. 273-288. Date of Electronic Publication: 2018 Nov 26.