HIV-specific Immunity Derived from Chimeric Antigen Receptor-engineered Stem Cells

Molecular Therapy

Volumn 23, Issue 8, 2015, Pages 1358-1367

  Zhen, Anjie ^a   Kamata, Masakazu ^a   Rezek, Valerie ^a   Rick, Jonathan ^a   Levin, Bernard ^a   Kasparian, Saro ^a   Chen, Irvin Sy ^a   Yang, Otto O ^a   Zack, Jerome A ^a   Kitchen, Scott G ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CHIMERIC ANTIGEN RECEPTOR; T LYMPHOCYTE RECEPTOR; CD34 ANTIGEN; CYTOKINE; LYMPHOCYTE ANTIGEN RECEPTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL DIFFERENTIATION; CELL ENGINEERING; CONTROLLED STUDY; HEMATOPOIETIC STEM CELL; HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IN VIVO STUDY; MOUSE; NATURAL KILLER CELL; NONHUMAN; STEM CELL GENE THERAPY; T LYMPHOCYTE; VIRUS IMMUNITY; VIRUS REPLICATION; ANIMAL; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; CHEMISTRY; CYTOLOGY; CYTOTOXIC T LYMPHOCYTE; GENE THERAPY; GENE VECTOR; GENETIC ENGINEERING; HEK293 CELL LINE; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; IMMUNOLOGY; METABOLISM; PROCEDURES; SPLEEN; VIROLOGY;

HUMAN IMMUNODEFICIENCY VIRUS; MUS;

ANIMALS; ANTIGENS, CD34; CD4-POSITIVE T-LYMPHOCYTES; CD8-POSITIVE T-LYMPHOCYTES; CELL DIFFERENTIATION; CYTOKINES; GENETIC ENGINEERING; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HEMATOPOIETIC STEM CELLS; HIV INFECTIONS; HIV-1; HUMANS; KILLER CELLS, NATURAL; MICE; RECEPTORS, ANTIGEN; RECEPTORS, ANTIGEN, T-CELL; SPLEEN; T-LYMPHOCYTES, CYTOTOXIC;

EID: 84938414274     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.102     Document Type: Article

References (34)

1. Gill, S and June, CH (2015). Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies. Immunol Rev 263: 68-89.
2. Maus, MV, Grupp, SA, Porter, DL and June, CH (2014). Antibody-modifed T cells: CARs take the front seat for hematologic malignancies. Blood 123: 2625-2635.
3. Sadelain, M, Brentjens, R and Rivière, I (2013). The basic principles of chimeric antigen receptor design. Cancer Discov 3: 388-398.
4. Kitchen, SG, Shimizu, S and An, DS (2011). Stem cell-based anti-HIV gene therapy. Virology 411: 260-272.
5. Kiem, H P, Jerome, KR, Deeks, SG and McCune, JM (2012). Hematopoietic-stem-cell-based gene therapy for HIV disease. Cell Stem Cell 10: 137-147.
6. Yang, OO, Tran, AC, Kalams, SA, Johnson, RP, Roberts, MR, Walker, BD (1997). Lysis of HIV-1-infected cells and inhibition of viral replication by universal receptor T cells. Proc Natl Acad Sci USA 94: 11478-22483.
7. Scholler, JJ, Brady, TLT, Binder-Scholl, GG, Hwang, W-TW, Plesa, GG, Hege, KMK et al. (2012). Decade-long safety and function of retroviral-modifed chimeric antigen receptor T cells. Sci Translat Med 4: 132ra53-132ra53.
8. van den Bent, MJ, Afra, D, de Witte, O, Ben Hassel, M, Schraub, S, Hoang-Xuan, K et al.; EORTC Radiotherapy and Brain Tumor Groups and the UK Medical Research Council. (2005). Long-term effcacy of early versus delayed radiotherapy for low-grade astrocytoma and oligodendroglioma in adults: the EORTC 22845 randomised trial. Lancet 366: 985-990.
9. Yang, OO, Tran, AC, Kalams, SA, Johnson, RP, Roberts, MR and Walker, BD (1997). Lysis of HIV-1-infected cells and inhibition of viral replication by universal receptor T cells. Proc Natl Acad Sci USA 94: 11478-11483.
10. Kitchen, SG, Bennett, M, Galic, Z, Kim, J, Xu, Q, Young, A et al. (2009). Engineering antigen-specifc T cells from genetically modifed human hematopoietic stem cells in immunodefcient mice. PLoS One 4: e8208.
11. Vatakis, DN, Koya, RC, Nixon, CC, Wei, L, Kim, SG, Avancena, P et al. (2011). Antitumor activity from antigen-specifc CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci USA 108: E1408-E1416.
12. Vatakis, DN, Arumugam, B, Kim, SG, Bristol, G, Yang, O and Zack, JA (2013). Introduction of exogenous T-cell receptors into human hematopoietic progenitors results in exclusion of endogenous T-cell receptor expression. Mol Ther 21: 1055-1063.
13. Kitchen, SG, Levin, BR, Bristol, G, Rezek, V, Kim, S, Aguilera-Sandoval, C et al. (2012). In vivo suppression of HIV by antigen specifc T cells derived from engineered hematopoietic stem cells. PLoS Pathog 8: e1002649.
14. Lin, WY and Roberts, MR (2002). Developmental dissociation of T cells from B, NK, and myeloid cells revealed by MHC class II-specifc chimeric immune receptors bearing TCR-zeta or FcR-gamma chain signaling domains. Blood 100: 3045-3048.
15. Hege, KM, Cooke, KS, Finer, MH, Zsebo, KM and Roberts, MR (1996). Systemic T cell-independent tumor immunity after transplantation of universal receptor-modifed bone marrow into SCID mice. J Exp Med 184: 2261-2269.
16. Gill, S and Porter, DL (2014). CAR-modifed anti-CD19 T cells for the treatment of B-cell malignancies: rules of the road. Expert Opin Biol Ther 14: 37-49.
17. Dudek, TE, No, DC, Seung, E, Vrbanac, VD, Fadda, L, Bhoumik, P et al. (2012). Rapid evolution of HIV-1 to functional CD8+ T cell responses in humanized BLT mice. Sci Transl Med 4: 143ra98.
18. Brainard, DM, Seung, E, Frahm, N, Cariappa, A, Bailey, CC, Hart, WK et al. (2009). Induction of robust cellular and humoral virus-specifc adaptive immune responses in human immunodefciency virus-infected humanized BLT mice. J Virol 83: 7305-7321.
19. Ito, R, Takahashi, T, Katano, I and Ito, M (2012). Current advances in humanized mouse models. Cell Mol Immunol 9: 208-214.
20. Shimizu, S, Hong, P, Arumugam, B, Pokomo, L, Boyer, J, Koizumi, N et al. (2010). A highly effcient short hairpin RNA potently down-regulates CCR5 expression in systemic lymphoid organs in the hu-BLT mouse model. Blood 115: 1534-1544.
21. Ringpis, GE, Shimizu, S, Arokium, H, Camba-Colón, J, Carroll, MV, Cortado, R et al. (2012). Engineering HIV-1-resistant T-cells from short-hairpin RNA-expressing hematopoietic stem/progenitor cells in humanized BLT mice. PLoS One 7: e53492.
22. Qin, XF, An, DS, Chen, IS and Baltimore, D (2003). Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc Natl Acad Sci USA 100: 183-188.
23. Holt, N, Wang, J, Kim, K, Friedman, G, Wang, X, Taupin, V et al. (2010). Human hematopoietic stem/progenitor cells modifed by zinc-fnger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol 28: 839-847.
24. Kitchen, SG, LaForge, S, Patel, VP, Kitchen, CM, Miceli, MC and Zack, JA (2002). Activation of CD8 T cells induces expression of CD4, which functions as a chemotactic receptor. Blood 99: 207-212.
25. Hall, C, Berkhout, B, Alarcon, B, Sancho, J, Wileman, T and Terhorst, C (1991). Requirements for cell surface expression of the human TCR/CD3 complex in non-T cells. Int Immunol 3: 359-368.
26. Verschoor, C P, Johnstone, J, Millar, J, Parsons, R, Lelic, A, Loeb, M et al. (2014). Alterations to the frequency and function of peripheral blood monocytes and associations with chronic disease in the advanced-age, frail elderly. PLoS One 9: e104522.
27. Zhen, A and Kitchen, S (2014). Stem-cell-based gene therapy for HIV infection. Viruses 6: 1-12.
28. Deng, K, Pertea, M, Rongvaux, A, Wang, L, Durand, CM, Ghiaur, G et al. (2015). Broad CTL response is required to clear latent HIV-1 due to dominance of escape mutations. Nature 517: 381-385.
29. Mitsuyasu, RT, Merigan, TC, Carr, A, Zack, JA, Winters, MA, Workman, C et al. (2009). Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells. Nat Med 15: 285-292.
30. Tey, SK (2014). Adoptive T-cell therapy: adverse events and safety switches. Clin Transl Immunology 3: e17.
31. Tran, AC, Zhang, D, Byrn, R and Roberts, MR (1995). Chimeric zeta-receptors direct human natural killer (NK) effector function to permit killing of NK-resistant tumor cells and HIV-infected T lymphocytes. J Immunol 155: 1000-1009.
32. Mitsuyasu, RT, Anton, PA, Deeks, SG, Scadden, DT, Connick, E, Downs, MT et al. (2000). Prolonged survival and tissue traffcking following adoptive transfer of CD4zeta gene-modifed autologous CD4(+) and CD8(+) T cells in human immunodefciency virus-infected subjects. Blood 96: 785-793.
33. Bernstein, HB, Plasterer, MC, Schiff, SE, Kitchen, CM, Kitchen, S and Zack, JA (2006). CD4 expression on activated NK cells: ligation of CD4 induces cytokine expression and cell migration. J Immunol 177: 3669-3676.
34. Douek, DC, Vescio, RA, Betts, MR, Brenchley, JM, Hill, BJ, Zhang, L et al. (2000). Assessment of thymic output in adults after haematopoietic stem-cell transplantation and prediction of T-cell reconstitution. Lancet 355: 1875-1881.