Gene therapy of Fanconi anemia: Preclinical efficacy using lentiviral vectors

Blood

Volumn 100, Issue 8, 2002, Pages 2732-2736

  Galimi, Francesco ^a,b   Noll, Meenakshi ^a,b   Kanazawa, Yoshiyuki ^a,b   Lax, Timothy ^a,b   Chen, Cindy ^a,b   Grompe, Markus ^a,b   Verma, Inder M ^a,b  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BUTADIENE DIEPOXIDE; CYCLOPHOSPHAMIDE; CYTOKINE; DNA; FANCONI ANEMIA GROUP A PROTEIN; FANCONI ANEMIA GROUP C PROTEIN; GENE PRODUCT; LENTIVIRUS VECTOR; MITOMYCIN; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CELL SELECTION; CONTROLLED STUDY; CYTOKINE RESPONSIVE ELEMENT; DRUG EFFECT; DRUG EFFICACY; EXPRESSION VECTOR; FANCA GENE; FANCC GENE; FANCONI ANEMIA; GENE; GENE REPLACEMENT THERAPY; GENETIC TRANSDUCTION; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL; IN VIVO STUDY; MONONUCLEAR CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; TREATMENT INDICATION;

EID: 0037108187     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2002-04-1245     Document Type: Article

References (31)

1. Joenje H, Patel KJ. The emerging genetic and molecular basis of Fanconi anaemia. Nat Rev Genet. 2001;2:446-457.
2. Timmers C, Taniguchi T, Hejna J, et al. Positional cloning of a novel Fanconi anemia gene, FANCD2. Mol Cell. 2001;7:241-248.
3. Strathdee CA, Gavish H, Shannon WR, Buchwald M. Cloning of cDNAs for Fanconi's anaemia by functional complementation. Nature. 1992; 356:763-767.
4. Lo Ten Foe JR, Rooimans MA, Bosnoyan-Collins L, et al. Expression cloning of a cDNA for the major Fanconi anaemia gene, FAA. Nat Genet. 1996;14:320-323.
5. Positional cloning of the Fanconi anaemia group A gene: the Fanconi anaemia/breast cancer consortium. Nat Genet. 1996;14:324-328.
6. de Winter JP, Waisfisz Q, Rooimans MA, et al. The Fanconi anaemia group G gene FANCG is identical with XRCC9. Nat Genet. 1998;20:281-283.
7. de Winter JP, Leveille F, van Berkel CG, et al. Isolation of a cDNA representing the Fanconi anemia complementation group E gene. Am J Hum Genet. 2000;67:1306-1308.
8. de Winter JP, Rooimans MA, van Der Weel L, et al. The Fanconi anaemia gene FANCF encodes a novel protein with homology to ROM. Nat Genet. 2000;24:15-16.
9. Alter BP. Fanconi's anaemia and its variability. BrJ Haematol. 1993;85:9-14.
10. Auerbach AD, Allen RG. Leukemia and preleukemia in Fanconi anemia patients: a review of the literature and report of the International Fanconi Anemia Registry. Cancer Genet Cytogenet. 1991;51:1-12.
11. Alter BP. Fanconi's anemia and malignancies. Am J Hematol. 1996;53:99-110.
12. Guardiola P, Socie G, Pasquini R, et al. Allogeneic stem cell transplantation for Fanconi Anaemia: Severe Aplastic Anaemia Working Party of the EBMT and EUFAR: European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 1998;21 (suppl 2):24-27.
13. Gush KA, Fu KL, Grompe M, Walsh CE. Phenotypic correction of Fanconi anemia group C knockout mice. Blood. 2000;95:700-704.
14. Kohn DB. Gene therapy for genetic haematological disorders and immunodeficiencies. J Intern Med. 2001;249:379-390.
15. Whitney MA, Royle G, Low MJ, et al. Germ cell defects and hematopoietic hypersensitivity to gamma-interferon in mice with a targeted disruption of the Fanconi anemia C gene. Blood. 1996; 88:49-58.
16. Battaile KP, Bateman RL, Mortimer D, et al. In vivo selection of wild-type hematopoietic stem cells in a murine model of Fanconi anemia. Blood. 1999;94:2151-2158.
17. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science. 1999;283:682-686.
18. Noll M, Battaile KP, Bateman R, et al. Fanconi anemia group A and C double-mutant mice: functional evidence for a multi-protein Fanconi anemia complex. Exp Hematol. 2002;30:679-688.
19. Yamada K, Olsen JC, Patel M, Rao KW, Walsh CE. Functional correction of Fanconi anemia group C hematopoietic cells by the use of a novel lentiviral vector. Mol Ther. 2001;3:485-490.
20. Dull T, Zufferey R, Kelly M, et al. A third-generation lentivirus vector with a conditional packaging system. J Virol. 1998;72:8463-8471.
21. Noll M, Bateman RL, D'Andrea AD, Grompe M. Preclinical protocol for in vivo selection of hematopoietic stem cells corrected by gene therapy in Fanconi anemia group C. Mol Ther. 2001;3:14-23.
22. Miller SA, Dykes DD, Polesky HF. A simple salting out procedure for extracting DNA from human nucleated cells [abstract]. Nucleic Acids Res. 1988;16:1215.
23. Follenzi A, Allies LE, Bakovic S, Geuna M, Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet. 2000;25:217-222.
24. Auerbach AD, Wolman SR. Susceptibility of Fanconi's anaemia fibroblasts to chromosome damage by carcinogens. Nature. 1976;261: 494-496.
25. Auerbach AD, Rogatko A, Schroeder-Kurth TM. International Fanconi Anemia Registry: relation of clinical symptoms to diepoxybutane sensitivity. Blood. 1989;73:391-396.
26. Walsh CE, Grompe M, Vanin E, et al. A functionally active retrovirus vector for gene therapy in Fanconi anemia group C. Blood. 1994;84:453-459.
27. Lo Ten Foe JR, Kwee ML, Rooimans MA, et al. Somatic mosaicism in Fanconi anemia: molecular basis and clinical significance. Eur J Hum Genet. 1997;5:137-148.
28. Liu JM, Kim S, Read EJ, et al. Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC). Hum Gene Ther. 1999;10:2337-2346.
29. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288:669-672.
30. Aiuti A, Vai S, Mortellaro A, et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med. 2002;8:423-425.
31. Howlett NG, Taniguchi T, Olson S, et al. Biallelic inactivation of BRCA2 in Fanconi anemia. Science. 2002;297:606-609.