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Review
. 2009:675-81.
doi: 10.1182/asheducation-2009.1.675.

Overview of gene therapy clinical progress including cancer treatment with gene-modified T cells

Malcolm K Brenner  1 , Fatma V Okur
Affiliations
Review

Overview of gene therapy clinical progress including cancer treatment with gene-modified T cells

Malcolm K Brenner et al. Hematology Am Soc Hematol Educ Program. 2009.

Abstract

It is now twenty years since the first legal gene transfer studies were approved, and there has been considerable disappointment in the slow rate of progress that followed the initial studies. Gradually, however, as the limitations of available vectors are acknowledged and overcome, and with advances in our understanding of the molecular and cell biology of genetic diseases and of cancer, unequivocal successes are now being reported. In this paper we describe the remaining major roadblocks to successful gene therapy and outline approaches to overcome them. We also illustrate how genetically modified immune system cells are already being used for the effective treatment of hematological and other malignancies, and how these approaches are being modified so that they can be effective in treating a broader range of malignancies.

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Conflict of interest statement

Disclosures

Conflict-of-interest disclosures: The authors declare no competing financial interests.

Off-label drug use: None disclosed

Figures

Figure 1
Figure 1. Virus-specific cytotoxic T cells
(A) Generation of virus-specific cytotoxic T lymphocyte (CTL) by using plasmid system. (B) Specificity of plasmid-activated CTL. The frequency of Adv (Hexon and Penton), Epstein-Barr virus (EBV; LMP2 and BZLF1) and cytomegalovirus (CMV; pp65) reactive T cells in multivirus CTL population was analyzed by interferon (IFN)-γ Elispot assay. The control was IFN-γ release in response to stimulation with an irrelevant peptide mixture.
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References

    1. Qiao J, Wang H, Kottke T, et al. Loading of oncolytic vesicular stomatitis virus onto antigen-specific T cells enhances the efficacy of adoptive T-cell therapy of tumors. Gene Ther. 2008;15:604–616. - PMC - PubMed
    1. Yotnda P, Savoldo B, Charlet-Berguerand N, Rooney C, Brenner M. Targeted delivery of adenoviral vectors by cytotoxic T cells. Blood. 2004;104:2272–2280. - PubMed
    1. Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. N Engl J Med. 2008;358:2240–2248. - PMC - PubMed
    1. Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat Med. 2006;12:342–347. - PubMed
    1. Hoffmann D, Bayer W, Wildner O. Therapeutic immune response induced by intratumoral expression of the fusogenic membrane protein of vesicular stomatitis virus and cytokines encoded by adenoviral vectors. Int J Mol Med. 2007;20:673–681. - PubMed

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