Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting
Year: 2011
Type: article
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.
Source: Molecular Therapy
DOI: 10.1038/mt.2011.78
Authors: 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, Adrian J Thrasher
Institutions: University of Minnesota, Fred Hutch Cancer Center, Guy's Hospital, King's College London, German Cancer Research Center, National Center for Tumor Diseases, University of Washington, Centre for Biomedical Network Research on Rare Diseases, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust, University of Sheffield, Hôpital Necker-Enfants Malades, Inserm, Assistance Publique – Hôpitaux de Paris, Université Paris Descartes, Riley Hospital for Children, Indiana University School of Medicine, Genethon (France), Université d'Évry Val-d'Essonne, Great Ormond Street Hospital, University College London, Heinrich Heine University Düsseldorf, Technische Universität Dresden, Center for Regenerative Therapies Dresden, Biotechnologisches Zentrum, Vita-Salute San Raffaele University, The San Raffaele Telethon Institute for Gene Therapy, Boston Children's Hospital, Harvard University, Dana-Farber Cancer Institute, Hospital Infantil Universitario Niño Jesús, Instituto de Investigación Sanitaria del Hospital Universitario de La Princesa, Lyon 1 Université, École Normale Supérieure de Lyon
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2170958764