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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Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
Materias:
Review
Acceso en línea: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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