| |  Online-Ressource |
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| Verfasst von: | Tolar, Jakub [VerfasserIn]  |
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| | Adair, Jennifer E [VerfasserIn] |
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| | Antoniou, Michael [VerfasserIn] |
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| | Bartholomä, Cynthia C. [VerfasserIn] |
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| | Becker, Pamela S [VerfasserIn] |
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| | Blazar, Bruce R [VerfasserIn] |
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| | Bueren, Juan [VerfasserIn] |
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| | Carroll, Thomas [VerfasserIn] |
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| | Cavazzana-Calvo, Marina [VerfasserIn] |
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| | Clapp, D Wade [VerfasserIn] |
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| | Dalgleish, Robert [VerfasserIn] |
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| | Galy, Anne [VerfasserIn] |
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| | Gaspar, H Bobby [VerfasserIn] |
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| | Hanenberg, Helmut [VerfasserIn] |
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| | Kalle, Christof von [VerfasserIn] |
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| | Kiem, Hans-Peter [VerfasserIn] |
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| | Lindeman, Dirk [VerfasserIn] |
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| | Naldini, Luigi [VerfasserIn] |
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| | Navarro, Susana [VerfasserIn] |
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| | Renella, Raffaele [VerfasserIn] |
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| | Rio, Paula [VerfasserIn] |
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| | Sevilla, Julián [VerfasserIn] |
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| | Schmidt, Manfred [VerfasserIn] |
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| | Verhoeyen, Els [VerfasserIn] |
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| | Wagner, John E [VerfasserIn] |
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| | Williams, David A [VerfasserIn] |
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| | Thrasher, Adrian J [VerfasserIn] |
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| Titel: | Stem cell gene therapy for Fanconi anemia |
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| Titelzusatz: | report from the 1st International Fanconi anemia gene therapy working group meeting |
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| Verf.angabe: | Jakub Tolar, Jennifer E Adair, Michael Antoniou, Cynthia C Bartholomae, Pamela S Becker, Bruce R Blazar, Juan Bueren, Thomas Carroll, Marina Cavazzana-Calvo, D Wade Clapp, Robert Dalgleish, Anne Galy, H Bobby Gaspar, Helmut Hanenberg, Christof Von Kalle, Hans-Peter Kiem, Dirk Lindeman, Luigi Naldini, Susana Navarro, Raffaele Renella, Paula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E Wagner, David A Williams and Adrian J Thrasher |
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| Jahr: | 2011 |
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| Umfang: | 6 S. |
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| Fussnoten: | Gesehen am 10.11.2022 ; Erstmals am 14. Dezember 2016 online veröffentlicht |
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| Titel Quelle: | Enthalten in: Molecular therapy |
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| Ort Quelle: | Amsterdam : Elsevier, 2000 |
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| Jahr Quelle: | 2011 |
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| Band/Heft Quelle: | 19(2011), 7 vom: Juli, Seite 1193-1198 |
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| ISSN Quelle: | 1525-0024 |
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| Abstract: | 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. |
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| DOI: | doi:10.1038/mt.2011.78 |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
Volltext ; Verlag: https://doi.org/10.1038/mt.2011.78 |
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| | Volltext: https://www.sciencedirect.com/science/article/pii/S1525001616324856 |
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| | DOI: https://doi.org/10.1038/mt.2011.78 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| K10plus-PPN: | 1821591372 |
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| Verknüpfungen: | → Zeitschrift |
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Stem cell gene therapy for Fanconi anemia / Tolar, Jakub [VerfasserIn]; 2011 (Online-Ressource)
