A promising vector for TCR gene therapy: Differential effect of siRNA, 2A peptide, and disulfide bond on the introduced TCR expression

Molecular Therapy Nucleic Acids

Volumn 1, Issue DEC, 2012, Pages e63-

  Okamoto, Sachiko ^a   Amaishi, Yasunori ^a   Goto, Yumi ^a   Ikeda, Hiroaki ^b   Fujiwara, Hiroshi ^c   Kuzushima, Kiyotaka ^d   Yasukawa, Masaki ^c   Shiku, Hiroshi ^b   Mineno, Junichi ^a  

^a TAKARA BIO INC   (Japan)

^b MIE UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^c EHIME UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^d AICHI CANCER CENTER HOSPITAL   (Japan)

Author keywords

Mixed TCR; Retroviral vector; siRNA; TCR gene therapy

Indexed keywords

2A PEPTIDE; CYSTEINE; DISULFIDE; PEPTIDE; RETROVIRUS VECTOR; SMALL INTERFERING RNA; T LYMPHOCYTE RECEPTOR; UNCLASSIFIED DRUG;

ARTICLE; CONTROLLED STUDY; DISULFIDE BOND; GENE THERAPY; GENE VECTOR; PRIORITY JOURNAL; PROTEIN PROCESSING; TRANSGENE; TREATMENT OUTCOME;

EID: 84892392184     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2012.52     Document Type: Article

References (40)

1. Rosenberg, SA and Dudley, ME (2009). Adoptive cell therapy for the treatment of patients with metastatic melanoma. Curr Opin Immunol 21: 233-240.
2. Chinnasamy, N, Wargo, JA, Yu, Z, Rao, M, Frankel, TL, Riley, JP et al. (2011). A TCR targeting the HLA-A*0201-restricted epitope of MAGE-A3 recognizes multiple epitopes of the MAGE-A antigen superfamily in several types of cancer. J Immunol 186: 685-696.
3. Goff, SL, Johnson, LA, Black, MA, Xu, H, Zheng, Z, Cohen, CJ et al. (2010). Enhanced receptor expression and in vitro effector function of a murine-human hybrid MART-1-reactive T cell receptor following a rapid expansion. Cancer Immunol Immunother 59: 1551-1560.
4. Morgan, RA, Dudley, ME, Wunderlich, JR, Hughes, MS, Yang, JC, Sherry, RM et al. (2006). Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 314: 126-129. (Pubitemid 44547756)
5. Johnson, LA, Morgan, RA, Dudley, ME, Cassard, L, Yang, JC, Hughes, MS et al. (2009). Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen. Blood 114: 535-546.
6. Robbins, PF, Morgan, RA, Feldman, SA, Yang, JC, Sherry, RM, Dudley, ME et al. (2011). Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol 29: 917-924.
7. Ochi, T, Fujiwara, H and Yasukawa, M (2010). Application of adoptive T-cell therapy using tumor antigen-specific T-cell receptor gene transfer for the treatment of human leukemia. J Biomed Biotechnol 2010: 521248.
8. Hiasa, A, Hirayama, M, Nishikawa, H, Kitano, S, Nukaya, I, Yu, SS et al. (2008). Longterm phenotypic, functional and genetic stability of cancer-specific T-cell receptor (TCR) alphabeta genes transduced to CD8+ T cells. Gene Ther 15: 695-699. (Pubitemid 351524524)
9. Shirakura, Y, Mizuno, Y, Wang, L, Imai, N, Amaike, C, Sato, E et al. (2012). T-cell receptor gene therapy targeting melanoma-associated antigen-A4 inhibits human tumor growth in non-obese diabetic/SCID/?cnull mice. Cancer Sci 103: 17-25.
10. Heemskerk, MH, Hagedoorn, RS, van der Hoorn, MA, van der Veken, LT, Hoogeboom, M, Kester, MG et al. (2007). Efficiency of T-cell receptor expression in dual-specific T cells is controlled by the intrinsic qualities of the TCR chains within the TCR-CD3 complex. Blood 109: 235-243. (Pubitemid 46053064)
11. Bendle, GM, Linnemann, C, Hooijkaas, AI, Bies, L, de Witte, MA, Jorritsma, A et al. (2010). Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy. Nat Med 16: 565-70, 1p following 570.
12. van Loenen, MM, de Boer, R, Amir, AL, Hagedoorn, RS, Volbeda, GL, Willemze, R et al. (2010). Mixed T cell receptor dimers harbor potentially harmful neoreactivity. Proc Natl Acad Sci USA 107: 10972-10977.
13. Kustikova, OS, Wahlers, A, Kuhlcke, K, Stahle, B, Zander, AR, Baum, C et al. (2003). Dose finding with retroviral vectors: correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population. Blood 102: 3934-3937. (Pubitemid 37486973)
14. Nair, V (2008). Retrovirus-induced oncogenesis and safety of retroviral vectors. Curr Opin Mol Ther 10: 431-438.
15. Sadelain, M (2004). Insertional oncogenesis in gene therapy: how much of a risk? Gene Ther 11: 569-573.
16. Hacein-Bey-Abina, S, Von Kalle, C, Schmidt, M, McCormack, MP, Wulffraat, N, Leboulch, P et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419. (Pubitemid 37296260)
17. Cohen, CJ, Li, YF, El-Gamil, M, Robbins, PF, Rosenberg, SA and Morgan, RA (2007). Enhanced antitumor activity of T cells engineered to express T-cell receptors with a second disulfide bond. Cancer Res 67: 3898-3903. (Pubitemid 46762178)
18. Aggen, DH, Chervin, AS, Schmitt, TM, Engels, B, Stone, JD, Richman, SA et al. (2012). Single-chain VaVß T-cell receptors function without mispairing with endogenous TCR chains. Gene Ther 19: 365-374.
19. Sebestyén, Z, Schooten, E, Sals, T, Zaldivar, I, San José, E, Alarcón, B et al. (2008). Human TCR that incorporate CD3zeta induce highly preferred pairing between TCRalpha and beta chains following gene transfer. J Immunol 180: 7736-7746.
20. Cohen, CJ, Zhao, Y, Zheng, Z, Rosenberg, SA and Morgan, RA (2006). Enhanced antitumor activity of murine-human hybrid T-cell receptor (TCR) in human lymphocytes is associated with improved pairing and TCR/CD3 stability. Cancer Res 66: 8878-8886. (Pubitemid 44449206)
21. Kuball, J, Dossett, ML, Wolfl, M, Ho, WY, Voss, RH, Fowler, C et al. (2007). Facilitating matched pairing and expression of TCR chains introduced into human T cells. Blood 109: 2331-2338. (Pubitemid 46425872)
22. Hiasa, A, Nishikawa, H, Hirayama, M, Kitano, S, Okamoto, S, Chono, H et al. (2009). Rapid alphabeta TCR-mediated responses in gammadelta T cells transduced with cancer-specific TCR genes. Gene Ther 16: 620-628.
23. Okamoto, S, Mineno, J, Ikeda, H, Fujiwara, H, Yasukawa, M, Shiku, H et al. (2009). Improved expression and reactivity of transduced tumor-specific TCRs in human lymphocytes by specific silencing of endogenous TCR. Cancer Res 69: 9003-9011.
24. Ochi, T, Fujiwara, H, Okamoto, S, An, J, Nagai, K, Shirakata, T et al. (2011). Novel adoptive T-cell immunotherapy using a WT1-specific TCR vector encoding silencers for endogenous TCRs shows marked antileukemia reactivity and safety. Blood 118: 1495-1503.
25. Szymczak, AL, Workman, CJ, Wang, Y, Vignali, KM, Dilioglou, S, Vanin, EF et al. (2004). Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat Biotechnol 22: 589-594. (Pubitemid 38585911)
26. Yang, S, Cohen, CJ, Peng, PD, Zhao, Y, Cassard, L, Yu, Z et al. (2008). Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition. Gene Ther 15: 1411-1423.
27. Lee, JT, Yu, SS, Han, E, Kim, S and Kim, S (2004). Engineering the splice acceptor for improved gene expression and viral titer in an MLV-based retroviral vector. Gene Ther 11: 94-99. (Pubitemid 38111393)
28. Yu, SS, Kim, JM and Kim, S (2000). High efficiency retroviral vectors that contain no viral coding sequences. Gene Ther 7: 797-804. (Pubitemid 30255762)
29. Kim, S, Lee, K, Kim, MD, Kang, S, Joo, CW, Kim, JM et al. (2006). Factors affecting the performance of different long terminal repeats in the retroviral vector. Biochem Biophys Res Commun 343: 1017-1022.
30. Hong, Y, Yu, SS, Kim, JM, Lee, K, Na, YS, Whitley, CB et al. (2003). Construction of a high efficiency retroviral vector for gene therapy of Hunter's syndrome. J Gene Med 5: 18-29. (Pubitemid 40310364)
31. Chono, H, Yoshioka, H, Ueno, M and Kato, I (2001). Removal of inhibitory substances with recombinant fibronectin-CH-296 plates enhances the retroviral transduction efficiency of CD34(+)CD38(-) bone marrow cells. J Biochem 130: 331-334. (Pubitemid 32845685)
32. Hanenberg, H, Xiao, XL, Dilloo, D, Hashino, K, Kato, I and Williams, DA (1996). Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med 2: 876-882. (Pubitemid 26296757)
33. Chono, H, Matsumoto, K, Tsuda, H, Saito, N, Lee, K, Kim, S et al. (2011). Acquisition of HIV-1 resistance in T lymphocytes using an ACA-specific E. coli mRNA interferase. Hum Gene Ther 22: 35-43.
34. Hayashita, Y, Osada, H, Tatematsu, Y, Yamada, H, Yanagisawa, K, Tomida, S et al. (2005). A polycistronic microRNA cluster, miR-17-92, is overexpressed in human lung cancers and enhances cell proliferation. Cancer Res 65: 9628-9632. (Pubitemid 41541436)
35. Mineno, J, Okamoto, S, Ando, T, Sato, M, Chono, H, Izu, H et al. (2006). The expression profile of microRNAs in mouse embryos. Nucleic Acids Res 34: 1765-1771.
36. Miyahara, Y, Naota, H, Wang, L, Hiasa, A, Goto, M, Watanabe, M et al. (2005). Determination of cellularly processed HLA-A2402-restricted novel CTL epitopes derived from two cancer germ line genes, MAGE-A4 and SAGE. Clin Cancer Res 11: 5581-5589. (Pubitemid 41060836)
37. Ohminami, H, Yasukawa, M and Fujita, S (2000). HLA class I-restricted lysis of leukemia cells by a CD8(+) cytotoxic T-lymphocyte clone specific for WT1 peptide. Blood 95: 286-293. (Pubitemid 30017254)
38. Makita, M, Hiraki, A, Azuma, T, Tsuboi, A, Oka, Y, Sugiyama, H et al. (2002). Antilung cancer effect of WT1-specific cytotoxic T lymphocytes. Clin Cancer Res 8: 2626-2631. (Pubitemid 34856350)
39. Tsuji, T, Yasukawa, M, Matsuzaki, J, Ohkuri, T, Chamoto, K, Wakita, D et al. (2005). Generation of tumor-specific, HLA class I-restricted human Th1 and Tc1 cells by cell engineering with tumor peptide-specific T-cell receptor genes. Blood 106: 470-476. (Pubitemid 40981240)
40. Tajima, K, Ito, Y, Demachi, A, Nishida, K, Akatsuka, Y, Tsujimura, K et al. (2004). Interferon-gamma differentially regulates susceptibility of lung cancer cells to telomerase-specific cytotoxic T lymphocytes. Int J Cancer 110: 403-412. (Pubitemid 38649892)