Retroviral integration and human gene therapy

Journal of Clinical Investigation

Volumn 117, Issue 8, 2007, Pages 2083-2086

  Bushman, Frederic D ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYTOKINE; RETROVIRUS VECTOR;

ACCURACY; CHROMOSOME; COMBINED IMMUNODEFICIENCY; CPG ISLAND; DNA INTEGRATION; DRUG DELIVERY SYSTEM; DRUG EFFICACY; GENETIC DISORDER; HUMAN; NONHUMAN; ONCOGENE; OUTCOME ASSESSMENT; PRIORITY JOURNAL; REVIEW; VIRAL GENE THERAPY;

ANTIGENS, CD34; CELL PROLIFERATION; CELL SURVIVAL; GAMMARETROVIRUS; GENE THERAPY; GENETIC VECTORS; GENOME, HUMAN; HEMATOPOIETIC STEM CELLS; HUMANS; LYMPHOPOIESIS; MUTAGENESIS, INSERTIONAL; QUANTITATIVE TRAIT LOCI; T-LYMPHOCYTES; TIME FACTORS; VIRUS INTEGRATION; X-LINKED COMBINED IMMUNODEFICIENCY DISEASES;

EID: 34547691354     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI32949     Document Type: Review

References (25)

1. Cavazzana-Calvo, M., et al. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288:669-672.
2. Blaese, R.M., et al. 1995. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science. 270:475-480.
3. Deichmann, A., et al. 2007. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J. Clin. Invest. 117:2225-2232. doi:10.1172/JCI31659.
4. Schwarzwaelder, K., et al. 2007. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J. Clin. Invest. 117:2241-2249. doi:10.1172/JCI31661.
5. Ott, M.G., et al. 2006. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12:401-409.
6. Aiuti, A., et al. 2002. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 296:2410-2413.
7. Hacein-Bey-Abina, S., et al. 2003. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348:255-256.
8. Hacein-Bey-Abina, S., et al. 2003. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 302:415-419.
9. Bushman, F., et al. 2005. Genome-wide analysis of retroviral DNA integration. Nat. Rev. Microbiol. 3:848-858.
10. Wu, X., Li, Y., Crise, B., and Burgess, S.M. 2003. Transcription start regions in the human genome are favored targets for MLV integration. Science. 300:1749-1751.
11. Schröder, A.R., et al. 2002. HIV-1 integration in the human genome favors active genes and local hotspots. Cell. 110:521-529.
12. Mitchell, R.S., et al. 2004. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol. 2:e234.
13. Narezkina, A., et al. 2004. Genome-wide analyses of avain sarcoma virus integration sites. J. Virol. 78:11656-11663.
14. Lewinski, M.K., et al. 2006. Retroviral DNA integration: viral and cellular determinants of target-site selection. PLoS Pathog. 2:e60.
15. Aiuti, A., et al. 2007. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 117:2233-2240. doi:10.1172/JCI31666.
16. + cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. Blood. 105:2699-2706.
17. Berry, C., Hannenhalli, S., Leipzig, J., and Bushman, F.D. 2006. Selection of target sites for mobile DNA integration in the human genome. PLoS Comput. Biol. 2:e157.
18. Wu, X., and Burgess, S.M. 2004. Integration target site selection for retroviruses and transposable elements. Cell. Mol. Life Sci. 61:2588-2596.
19. Craig, N.L., Craigie, R., Gellert, M., and Lambowitz, A.M. 2002. Mobile DNA II. ASM Press. Washington, DC, USA. 1204 pp.
20. Bushman, F.D. 2001. Lateral DNA transfer: mechanisms and consequences. Cold Spring Harbor Laboratory Press. Cold Spring Harbor, New York, USA. 448 pp.
21. Lewinski, M., et al. 2005. Genome-wide analysis of chromosomal features repressing HIV transcription. J. Virol. 79:6610-6619.
22. Jordan, A., Bisgrove, D., and Verdin, E. 2003. HIV reproducibly establishes a latent infection after acute infection of T cells in vitro. EMBO J. 22:1868-1877.
23. Coffin, J.M., Hughes, S.H., and Varmus, H.E. 1997. Retroviruses. Cold Spring Harbor Laboratory Press. Cold Spring Harbor, New York, USA. 843 pp.
24. Levine, B.L., et al. 2006. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc. Natl. Acad. Sci. U. S. A. 103:17372-17377.
25. Naldini, L., et al. 1996. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:263-267.