Stem cell gene therapy for fanconi anemia: Report from the 1st international fanconi anemia gene therapy working group meeting

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 RenellaPaula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E. Wagner, David A. Williams, Adrian J. Thrasher

Research output: Contribution to journalArticle

37 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1193-1198
Number of pages6
JournalMolecular Therapy
Issue number7
StatePublished - Jul 2011

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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