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Stem cell gene therapy for Fanconi anemia: report from the 1st International Fanconi anemia gene therapy working group meeting

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 po...

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Main Authors: Tolar, Jakub (Author) , Adair, Jennifer E (Author) , Antoniou, Michael (Author) , Bartholomä, Cynthia C. (Author) , Becker, Pamela S (Author) , Blazar, Bruce R (Author) , Bueren, Juan (Author) , Carroll, Thomas (Author) , Cavazzana-Calvo, Marina (Author) , Clapp, D Wade (Author) , Dalgleish, Robert (Author) , Galy, Anne (Author) , Gaspar, H Bobby (Author) , Hanenberg, Helmut (Author) , Kalle, Christof von (Author) , Kiem, Hans-Peter (Author) , Lindeman, Dirk (Author) , Naldini, Luigi (Author) , Navarro, Susana (Author) , Renella, Raffaele (Author) , Rio, Paula (Author) , Sevilla, Julián (Author) , Schmidt, Manfred (Author) , Verhoeyen, Els (Author) , Wagner, John E (Author) , Williams, David A (Author) , Thrasher, Adrian J (Author)
Format: Article (Journal) Conference Paper
Language:English
Published: 2011
In: Molecular therapy
Year: 2011, Volume: 19, Issue: 7, Pages: 1193-1198
ISSN:1525-0024
DOI:10.1038/mt.2011.78
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/mt.2011.78
Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S1525001616324856
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Author Notes: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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Summary: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.
Item Description:Gesehen am 10.11.2022
Erstmals am 14. Dezember 2016 online veröffentlicht
Physical Description:Online Resource
ISSN:1525-0024
DOI:10.1038/mt.2011.78

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