Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting
| Autor: | Juan A. Bueren, Susana Navarro, Jennifer E. Adair, Paula Río, Michael Antoniou, Cynthia C. Bartholomae, Julián Sevilla, Pamela S. Becker, Anne Galy, Adrian J. Thrasher, Bruce R. Blazar, David A. Williams, Marina Cavazzana-Calvo, Thomas Carroll, Luigi Naldini, Robert Dalgleish, Els Verhoeyen, Helmut Hanenberg, Raffaele Renella, H. Bobby Gaspar, D. Wade Clapp, Dirk Lindeman, Hans-Peter Kiem, Jakub Tolar, John E. Wagner, Manfred Schmidt, Christof von Kalle |
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| Přispěvatelé: | Tolar, J, Adair, Je, Antoniou, M, Bartholomae, Cc, Becker, P, Blazar, Br, Bueren, J, Carroll, T, Cavazzana Calvo, M, Clapp, Dw, Dalgleish, R, Galy, A, Gaspar, Hb, Hanenberg, H, Von Kalle, C, Kiem, Hp, Lindeman, D, Naldini, Luigi, Navarro, S, Renella, R, Rio, P, Sevilla, J, Schmidt, M, Verhoeyen, E, Wagner, Je, Williams, Da, Thrasher, Aj, CHU Necker - Enfants Malades [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Immunologie moléculaire et biothérapies innovantes (IMBI), Généthon-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université d'Évry-Val-d'Essonne (UEVE)-École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL), Immunologie des maladies infectieuses allergiques et autoimmunes, Université Nice Sophia Antipolis (... - 2019) (UNS), COMUE Université Côte d'Azur (2015-2019) (COMUE UCA)-COMUE Université Côte d'Azur (2015-2019) (COMUE UCA)-Institut National de la Santé et de la Recherche Médicale (INSERM), Virus enveloppés, vecteurs et immunothérapie – Enveloped viruses, Vectors and Immuno-therapy (EVIR), Centre International de Recherche en Infectiologie - UMR (CIRI), École normale supérieure - Lyon (ENS Lyon)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-École normale supérieure - Lyon (ENS Lyon)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), École Pratique des Hautes Études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, Université Nice Sophia Antipolis (1965 - 2019) (UNS), Centre International de Recherche en Infectiologie (CIRI), École normale supérieure de Lyon (ENS de Lyon)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Université Jean Monnet - Saint-Étienne (UJM)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-École normale supérieure de Lyon (ENS de Lyon)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Université Jean Monnet - Saint-Étienne (UJM)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS) |
| Jazyk: | angličtina |
| Rok vydání: | 2011 |
| Předmět: |
Oncology
medicine.medical_specialty Stem Cell Transplantation/methods Genetic enhancement [SDV]Life Sciences [q-bio] Review Genetic Therapy/*methods Gene product 03 medical and health sciences 0302 clinical medicine Fanconi anemia Internal medicine Drug Discovery medicine Genetics Humans Gene Molecular Biology 030304 developmental biology Pharmacology 0303 health sciences Hematopoietic Stem Cells/cytology Fanconi Anemia/*therapy business.industry Genetic Therapy Congresses as Topic medicine.disease Hematopoietic Stem Cells 3. Good health Clinical trial Transplantation Fanconi Anemia 030220 oncology & carcinogenesis Immunology Savior sibling Molecular Medicine Stem cell business Stem Cell Transplantation |
| Zdroj: | Molecular Therapy; Vol 19 Molecular Therapy Molecular Therapy, Cell Press, 2011, 19 (7), pp.1193-8. ⟨10.1038/mt.2011.78⟩ Molecular Therapy, 2011, 19 (7), pp.1193-8. ⟨10.1038/mt.2011.78⟩ |
| ISSN: | 1525-0024 1525-0016 |
| DOI: | 10.1038/mt.2011.78 |
| Popis: | Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potentially avoid the early and late complications of allogeneic HCT. Over the last decades, gene therapy has experienced a high degree of optimism interrupted by periods of diminished expectation. Optimism stems from recent examples of successful gene correction in several congenital immunodeficiencies, whereas diminished expectations come from the realization that gene therapy will not be free of side effects. The goal of the 1st International Fanconi Anemia Gene Therapy Working Group Meeting was to determine the optimal strategy for moving stem cell gene therapy into clinical trials for individuals with FA. To this end, key investigators examined vector design, transduction method, criteria for large-scale clinical-grade vector manufacture, hematopoietic cell preparation, and eligibility criteria for FA patients most likely to benefit. The report summarizes the roadmap for the development of gene therapy for FA. |
| Databáze: | OpenAIRE |
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