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Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc−/− stem cells

Si, Yue, Pulliam, Anna C., Linka, Yvonne, Ciccone, Samantha, Leurs, Cordula, Yuan, Jin, Eckermann, Olaf, Fruehauf, Stefan, Mooney, Sean, Hanenberg, Helmut, Clapp, D. Wade·2008·Blood
DOI 10.1182/blood-2007-07-102947
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Abstract

Fanconi anemia (FA) is a complex genetic disorder characterized by congenital abnormalities, bone marrow failure, and myeloid malignancies. Identification of 13 FA genes has been instrumental to explore gene transfer technologies aimed at correction of autologous FA-deficient stem cells. To date, 3 human FA stem cell gene therapy trials with standard 4-day transduction protocols using gammaretroviral vectors failed to provide clinical benefit. In addition, 2- to 4 day ex vivo manipulation of bone marrow from mice containing a disruption of the homologue of human FANCC (Fancc) results in a time-dependent increase in apoptosis and a risk for malignant transformation of hematopoietic cells. Here, we show that a 14-hour transduction period allows a foamyviral vector construct expressing the human FANCC cDNA to efficiently transduce murine FA stem cells with 1 to 2 proviral integrations per genome. Functionally, the repopulating activity of Fancc−/− stem cells from reconstituted mice expressing the recombinant FANCC transgene was comparable with wild-type controls. Collectively, these data provide evidence that short-term transduction of c-kit+ cells with a foamyviral vector is sufficient for functional correction of a stem cell phenotype in a murine FA model. These data could have implications for future gene therapy trials for FA patients.

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Cited by

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Metadata

Authors
Si, Y., Pulliam, A. C., Linka, Y., Ciccone, S., Leurs, C., Yuan, J., Eckermann, O., Fruehauf, S., Mooney, S., Hanenberg, H., & Clapp, D. W.
Affiliations
  • Departments ofMicrobiology and Immunology and
  • Department of Pediatric Oncology, Hematology and Clinical Immunology, Children's Hospital, Heinrich Heine University, Duesseldorf, Germany;
  • Department of Internal Medicine III, University of Heidelberg, Heidelberg, Germany; and
  • Center for Computational Biology and Bioinformatics and
  • Pediatrics, Herman B Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis;
Venue
Blood
Publisher
American Society of Hematology
Published
2008-12-01
Volume
112
Issue
12
Pages
4458-4465
ISSN
  • 0006-4971
  • 1528-0020
DOI
10.1182/blood-2007-07-102947
Cited by
28
References
4
Language
en

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