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Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors

Galimi, Francesco, Noll, Meenakshi, Kanazawa, Yoshiyuki, Lax, Timothy, Chen, Cindy, Grompe, Markus, Verma, Inder M.·2002·Blood
DOI 10.1182/blood-2002-04-1245
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Abstract

Fanconi anemia (FA) is an inherited cancer susceptibility syndrome caused by mutations in a DNA repair pathway including at least 6 genes(FANCA, FANCC, FANCD2, FANCE, FANCF, and FANCG). The clinical course of the disease is dominated by progressive, life-threatening bone marrow failure and high incidence of acute myelogenous leukemia and solid tumors. Allogeneic bone marrow transplantation (BMT) is a therapeutic option but requires HLA-matched donors. Gene therapy holds great promise for FA, but previous attempts to use retroviral vectors in humans have proven ineffective given the impaired proliferation potential of human FA hematopoietic progenitors (HPCs). In this work, we show that using lentiviral vectors efficient genetic correction can be achieved in quiescent hematopoietic progenitors from Fanca−/− andFancc−/−mice. Long-term repopulating HPCs were transduced by a single exposure of unfractionated bone marrow mononuclear cells to lentivectors carrying the normal gene. Notably, no cell purification or cytokine prestimulation was necessary. Resistance to DNA- damaging agents was fully restored by lentiviral transduction, allowing for in vivo selection of the corrected cells with nonablative doses of cyclophosphamide. This study strongly supports the use of lentiviral vectors for FA gene therapy in humans.

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Metadata

Authors
Galimi, F., Noll, M., Kanazawa, Y., Lax, T., Chen, C., Grompe, M., & Verma, I. M.
Affiliations
From the Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, CA; and the Department of Molecular and Medical Genetics, Oregon Health Science University, Portland.
Venue
Blood
Publisher
American Society of Hematology
Published
2002-10-15
Volume
100
Issue
8
Pages
2732-2736
ISSN
  • 1528-0020
  • 0006-4971
DOI
10.1182/blood-2002-04-1245
Cited by
66
References
1
Language
en

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