Volume 19, Issue 7, Pages (July 2011)

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Volume 19, Issue 7, Pages 1193-1198 (July 2011) 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 Renella, Paula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E Wagner, David A Williams, Adrian J Thrasher Molecular Therapy Volume 19, Issue 7, Pages 1193-1198 (July 2011) DOI: 10.1038/mt.2011.78 Copyright © 2011 The American Society of Gene & Cell Therapy Terms and Conditions

Figure 1 The lentiviral vector pC/RRL-PGK-FANCA-WPRE. CMV, cytomegalovirus; cPPT, central polypurine tract; FANCA, Fanconi anemia cDNA; PGK, phosphoglycerate kinase; RRE, rev responsive element; SA, splice acceptor; SD, splice donor; WPRE, safety-optimized version of the woodchuck hepatitis virus posttranscriptional regulatory element. Molecular Therapy 2011 19, 1193-1198DOI: (10.1038/mt.2011.78) Copyright © 2011 The American Society of Gene & Cell Therapy Terms and Conditions