Gene Therapy in Patients with the Crigler-Najjar Syndrome.
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| Title: | Gene Therapy in Patients with the Crigler-Najjar Syndrome. |
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| Authors: | D'Antiga L; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Beuers U; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Ronzitti G; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Brunetti-Pierri N; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Baumann U; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Di Giorgio A; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Aronson S; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Hubert A; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Romano R; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Junge N; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Bosma P; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Bortolussi G; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Muro AF; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Soumoudronga RF; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Veron P; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Collaud F; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Knuchel-Legendre N; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Labrune P; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.)., Mingozzi F; From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.). |
| Source: | The New England journal of medicine [N Engl J Med] 2023 Aug 17; Vol. 389 (7), pp. 620-631. |
| Publication Type: | Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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