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, Adair, Jennifer E, Antoniou, Michael, Bartholomae, Cynthia C, Becker, Pamela S, Blazar, Bruce R, Bueren, Juan, Carroll, Thomas, Cavazzana-Calvo, Marina, Clapp, D Wade, Dalgleish, Robert, Galy, Anne, Gaspar, H Bobby, Hanenberg, Helmut, Von Kalle, Christof, Kiem, Hans-Peter, Lindeman, Dirk, Naldini, Luigi, Navarro, Susana, Renella, Raffaele, Rio, Paula, Sevilla, Julián, Schmidt, Manfred, Verhoeyen, Els, Wagner, John E, Williams, David A, Thrasher, Adrian J
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2011
Teme:
Review
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3129570/
https://ncbi.nlm.nih.gov/pubmed/21540837
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.78
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