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Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.

Autor: Río P; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Zubicaray J; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Foundation for the Biomedical Research, Hospital Infantil Universitario Niño Jesús, Madrid, Spain., Navarro S; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Gálvez E; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Foundation for the Biomedical Research, Hospital Infantil Universitario Niño Jesús, Madrid, Spain., Sánchez-Domínguez R; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Nicoletti E; Rocket Pharmaceuticals, Cranbury, NJ, USA., Sebastián E; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Foundation for the Biomedical Research, Hospital Infantil Universitario Niño Jesús, Madrid, Spain., Rothe M; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Pujol R; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Institut de Recerca Sant Pau, Universitat Autónoma de Barcelona, Barcelona, Spain; Unit of Genomic Medicine, Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau, Barcelona, Spain., Bogliolo M; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Institut de Recerca Sant Pau, Universitat Autónoma de Barcelona, Barcelona, Spain; Serra Hunter Fellow, Department of Genetics and Microbiology, Faculty of Biosciences, Universitat Autònoma de Barcelona, Barcelona, Spain; Unit of Genomic Medicine, Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau, Barcelona, Spain., John-Neek P; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Bastone AL; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Wang W; Genwerk, Heidelberg, Germany., Schmidt M; Genwerk, Heidelberg, Germany., Larcher L; Université Paris Cité, Inserm, CNRS, Hôpital Saint-Louis, APHP, Paris, France., Segovia JC; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Yáñez RM; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Alberquilla O; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Díez B; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Fernández-García M; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., García-García L; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain., Ramírez M; Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Sanitary Research Institute Fundación La Princesa, Madrid, Spain., Galy A; Genethon, UMR_S951, Université Paris-Saclay, Univ Evry, Inserm, Evry-Courcouronnes, France., Lefrere F; Hôpital Universitaire Necker Enfants-Malades, Assistance Publique Hôpitaux de Paris GHU Paris Centre, Université Paris Cité, Paris, France; Centre D'Investigation Clinique en Biotherapie INSERM, Institut Imagine, Paris, France., Cavazzana M; Hôpital Universitaire Necker Enfants-Malades, Assistance Publique Hôpitaux de Paris GHU Paris Centre, Université Paris Cité, Paris, France; Centre D'Investigation Clinique en Biotherapie INSERM, Institut Imagine, Paris, France., Leblanc T; Robert-Debré University Hospital (APHP and Université Paris Cité), Paris, France., García de Andoin N; Donostia Universitary Hospital, San Sebastián, Spain; Biogipuzkoa Health Research Institute, San Sebastián, Spain., López-Almaraz R; Cruces Universitary Hospital, Barakaldo, Spain; Biocruces Bizkaia Health Research Institute, Barakaldo, Spain., Catalá A; Hospital Sant Joan de Déu, Barcelona, Spain; Research Institute Sant Joan de Déu, Barcelona, Spain., Barquinero J; Vall d'Hebron Institut de Recerca, Barcelona, Spain., Rodríguez-Perales S; Spanish National Cancer Research Centre (CNIO), Madrid, Spain., Rao G; Rocket Pharmaceuticals, Cranbury, NJ, USA., Surrallés J; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Institut de Recerca Sant Pau, Universitat Autónoma de Barcelona, Barcelona, Spain; Unit of Genomic Medicine, Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau, Barcelona, Spain., Soulier J; Université Paris Cité, Inserm, CNRS, Hôpital Saint-Louis, APHP, Paris, France., Díaz-de-Heredia C; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Vall d'Hebron Institut de Recerca, Barcelona, Spain; Pediatric Haematology and Oncology Department, Hospital Universitari Vall d'Hebron, Barcelona, Spain., Schwartz JD; Rocket Pharmaceuticals, Cranbury, NJ, USA., Sevilla J; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Pediatric Hematology and Oncology Department, Hospital Infantil Universitario Niño Jesús, Madrid, Spain; Foundation for the Biomedical Research, Hospital Infantil Universitario Niño Jesús, Madrid, Spain., Bueren JA; Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain. Electronic address: juan.bueren@ciemat.es.

Publikováno v: Lancet (London, England) [Lancet] 2024 Dec 03. Date of Electronic Publication: 2024 Dec 03.

TGF β Inhibitor A83-01 Enhances Murine HSPC Expansion for Gene Therapy.

Autor: Fleischauer J; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany., Bastone AL; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany., Selich A; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany., John-Neek P; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany., Weisskoeppel L; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany., Schaudien D; Department of Inhalation Toxicology, Fraunhofer Institute for Toxicology and Experimental Medicine ITEM, Nikolai Fuchs Strasse 1, 30625 Hannover, Germany., Schambach A; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany.; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, 30625 Hannover, Germany., Rothe M; Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany.; REBIRTH-Research Center for Translational Regenerative Medicine, Hannover Medical School, 30625 Hannover, Germany.

Publikováno v: Cells [Cells] 2023 Jul 31; Vol. 12 (15). Date of Electronic Publication: 2023 Jul 31.