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Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary, Pratima; Shapiro, Susan; Makris, Mike; Evans, Gillian; Boyce, Sara; Talks, Kate; Dolan, Gerard; Reiss, Ulrike; Phillips, Mark; Riddell, Anne; Peralta, Maria R; Quaye, Michelle; Patch, David W; Tuddenham, Edward; Dane, Allison; Watissée, Marie; Long, Alison; Nathwani, Amit.
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  • Chowdary P; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Shapiro S; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Makris M; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Evans G; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Boyce S; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Talks K; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Dolan G; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Reiss U; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Phillips M; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Riddell A; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Peralta MR; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Quaye M; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Patch DW; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Tuddenham E; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Dane A; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Watissée M; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Long A; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
  • Nathwani A; From the Katharine Dormandy Haemophilia and Thrombosis Centre (P.C., M.P., A.R., M.R.P., E.T., A.N.), Health Services Laboratory, Sonic Healthcare (A.R.), and the Department of Hepatology and Liver Transplantation (D.W.P.), Royal Free Hospital, University College London (P.C., M.P., M.Q., A.N.), Guy
N Engl J Med ; 387(3): 237-247, 2022 07 21.
Article en En | MEDLINE | ID: mdl-35857660
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Hemofilia B / Dependovirus / Glucocorticoides Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Revista: N Engl J Med Año: 2022 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Hemofilia B / Dependovirus / Glucocorticoides Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Revista: N Engl J Med Año: 2022 Tipo del documento: Article