Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy

Molecular Therapy

Volumn 21, Issue 2, 2013, Pages 358-367

  Wang, Yuhua ^a   Su, Hsing Hao ^a   Yang, Yang ^a   Hu, Yunxia ^a   Zhang, Lu ^a   Blancafort, Pilar ^b,c   Huang, Leaf ^a  

^a University of North Carolina   (United States)

^b UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

DOUBLE STRANDED DNA; GANCICLOVIR; GENE THERAPY AGENT; HERPES SIMPLEX VIRUS 1 THYMIDINE KINASE; LIPOSOME PROTAMINE RNA NANOPARTICLE; MESSENGER RNA; NANOPARTICLE; PLASMID DNA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTINEOPLASTIC ACTIVITY; APOPTOSIS; ARTICLE; CANCER CELL CULTURE; CANCER GENE THERAPY; CANCER INHIBITION; DRUG DOSAGE FORM COMPARISON; DRUG EFFICACY; DRUG FORMULATION; GENE EXPRESSION; HUMAN; HUMAN CELL; IMMUNOSTIMULATION; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; INNATE IMMUNITY; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PARTICLE SIZE; PHYSICAL CHEMISTRY; SUICIDE GENE THERAPY; TRANSMISSION ELECTRON MICROSCOPY; ZETA POTENTIAL;

HUMAN HERPESVIRUS 1; MUS MUSCULUS;

EID: 84873406290     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.250     Document Type: Article

References (40)

1. Van Tendeloo, VF, Ponsaerts, P and Berneman, ZN (2007). mRNA-based gene transfer as a tool for gene and cell therapy. Curr Opin Mol Ther 9: 423-431.
2. Steinman, RM and Hemmi, H (2006). Dendritic cells: translating innate to adaptive immunity. Curr Top Microbiol Immunol 311: 17-58.
3. Ponsaerts, P, van Tendeloo, VF, Jorens, PG, Berneman, ZN and van Bockstaele, DR (2004). Current challenges in human embryonic stem cell research: directed differentiation and transplantation tolerance. J Biol Regul Homeost Agents 18: 347-351.
4. Warren, L, Manos, PD, Ahfeldt, T, Loh, YH, Li, H, Lau, F et al. (2010). Highly effcient reprogramming to pluripotency and directed differentiation of human cells with synthetic modifed mRNA. Cell Stem Cell 7: 618-630.
5. Van Driessche, A, Ponsaerts, P, Van Bockstaele, DR, Van Tendeloo, VF and Berneman, ZN (2005). Messenger RNA electroporation: an effcient tool in immunotherapy and stem cell research. Folia Histochem Cytobiol 43: 213-216.
6. Ponsaerts, P, Van Tendeloo, VF and Berneman, ZN (2003). Cancer immunotherapy using RNA-loaded dendritic cells. Clin Exp Immunol 134: 378-384.
7. Kormann, MS, Hasenpusch, G, Aneja, MK, Nica, G, Flemmer, AW, Herber-Jonat, S et al. (2011). Expression of therapeutic proteins after delivery of chemically modifed mRNA in mice. Nat Biotechnol 29: 154-157.
8. Karikó, K, Buckstein, M, Ni, H and Weissman, D (2005). Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modifcation and the evolutionary origin of RNA. Immunity 23: 165-175.
9. Li, SD, Chen, YC, Hackett, MJ and Huang, L (2008). Tumor-targeted delivery of siRNA by self-assembled nanoparticles. Mol Ther 16: 163-169.
10. Guo, ST and Huang, L (2011). Nanoparticles escaping RES and endosome: challenges for siRNA delivery for cancer therapy. J Nanomater (epub ahead of print).
11. Chono, S, Li, SD, Conwell, CC and Huang, L (2008). An effcient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor. J Control Release 131: 64-69.
12. Li, SD, Chono, S and Huang, L (2008). Effcient gene silencing in metastatic tumor by siRNA formulated in surface-modifed nanoparticles. J Control Release 126: 77-84.
13. Li, SD and Huang, L (2006). Surface-modifed LPD nanoparticles for tumor targeting. Ann N Y Acad Sci 1082: 1-8.
14. Chen, Y, Sen, J, Bathula, SR, Yang, Q, Fittipaldi, R and Huang, L (2009). Novel cationic lipid that delivers siRNA and enhances therapeutic effect in lung cancer cells. Mol Pharm 6: 696-705.
15. Wong, C, Stylianopoulos, T, Cui, J, Martin, J, Chauhan, V P, Jiang, W et al. (2011). Multistage nanoparticle delivery system for deep penetration into tumor tissue. Proc Natl Acad Sci USA 108: 2426-2431.
16. Popovic, Z, Liu, W, Chauhan, VP, Lee, J, Wong, C, Greytak, AB et al. (2010). A nanoparticle size series for in vivo fuorescence imaging. Angew Chem Int Ed Engl 49: 8649-8652.
17. Cabral, H, Matsumoto, Y, Mizuno, K, Chen, Q, Murakami, M, Kimura, M et al. (2011). Accumulation of sub-100 nm polymeric micelles in poorly permeable tumours depends on size. Nat Nanotechnol 6: 815-823.
18. Lee, H, Fonge, H, Hoang, B, Reilly, RM and Allen, C (2010). The effects of particle size and molecular targeting on the intratumoral and subcellular distribution of polymeric nanoparticles. Mol Pharm 7: 1195-1208.
19. Corish, P and Tyler-Smith, C (1999). Attenuation of green fuorescent protein half-life in mammalian cells. Protein Eng 12: 1035-1040.
20. Tousignant, JD, Zhao, H, Yew, NS, Cheng, SH, Eastman, SJ and Scheule, RK (2003). DNA sequences in cationic lipid:pDNA-mediated systemic toxicities. Hum Gene Ther 14: 203-214.
21. Rejman, J, Oberle, V, Zuhorn, IS and Hoekstra, D (2004). Size-dependent internalization of particles via the pathways of clathrin-and caveolae-mediated endocytosis. Biochem J 377(Pt 1): 159-169.
22. Yamamoto, A, Kormann, M, Rosenecker, J and Rudolph, C (2009). Current prospects for mRNA gene delivery. Eur J Pharm Biopharm 71: 484-489.
23. El Ouahabi, A, Pector, V, Fuks, R, Vandenbranden, M and Ruysschaert, JM (1996). Double long-chain amidine liposome-mediated self replicating RNA transfection. FEBS Lett 380: 108-112.
24. Bettinger, T, Carlisle, RC, Read, ML, Ogris, M and Seymour, LW (2001). Peptide-mediated RNA delivery: a novel approach for enhanced transfection of primary and post-mitotic cells. Nucleic Acids Res 29: 3882-3891.
25. Tseng, YC, Mozumdar, S and Huang, L (2009). Lipid-based systemic delivery of siRNA. Adv Drug Deliv Rev 61: 721-731.
26. Lam, AP and Dean, DA (2010). Progress and prospects: nuclear import of nonviral vectors. Gene Ther 17: 439-447.
27. Eckner, R, Ellmeier, W and Birnstiel, ML (1991). Mature mRNA 3' end formation stimulates RNA export from the nucleus. EMBO J 10: 3513-3522.
28. Löser, P, Jennings, GS, Strauss, M and Sandig, V (1998). Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkappaB. J Virol 72: 180-190.
29. Banks-Schlegel, SP, Gazdar, AF and Harris, CC (1985). Intermediate flament and cross-linked envelope expression in human lung tumor cell lines. Cancer Res 45: 1187-1197.
30. Diebold, SS, Kaisho, T, Hemmi, H, Akira, S and Reis e Sousa, C (2004). Innate antiviral responses by means of TLR7-mediated recognition of single-stranded RNA. Science 303: 1529-1531.
31. Nallagatla, SR, Toroney, R and Bevilacqua, PC (2008). A brilliant disguise for self RNA: 5'-end and internal modifcations of primary transcripts suppress elements of innate immunity. RNA Biol 5: 140-144.
32. Cantara, WA, Crain, PF, Rozenski, J, McCloskey, JA, Harris, KA, Zhang, X et al. (2011). The RNA Modifcation Database, RNAMDB: 2011 update. Nucleic Acids Res 39(Database issue): D195-D201.
33. Karikó, K, Muramatsu, H, Ludwig, J and Weissman, D (2011). Generating the optimal mRNA for therapy: HPLC purifcation eliminates immune activation and improves translation of nucleoside-modifed, protein-encoding mRNA. Nucleic Acids Res 39: e142.
34. Maeda, H, Wu, J, Sawa, T, Matsumura, Y and Hori, K (2000). Tumor vascular permeability and the EPR effect in macromolecular therapeutics: a review. J Control Release 65: 271-284.
35. Kirpotin, DB, Drummond, DC, Shao, Y, Shalaby, MR, Hong, K, Nielsen, UB et al. (2006). Antibody targeting of long-circulating lipidic nanoparticles does not increase tumor localization but does increase internalization in animal models. Cancer Res 66: 6732-6740.
36. Li, SD and Huang, L (2010). Stealth nanoparticles: high density but sheddable PEG is a key for tumor targeting. J Control Release 145: 178-181.
37. Tomicic, MT, Thust, R and Kaina, B (2002). Ganciclovir-induced apoptosis in HSV-1 thymidine kinase expressing cells: critical role of DNA breaks, Bcl-2 decline and caspase-9 activation. Oncogene 21: 2141-2153.
38. Bostanci, A (2002). Gene therapy. Blood test fags agent in death of Penn subject. Science 295: 604-605.
39. Hacein-Bey-Abina, S, von Kalle, C, Schmidt, M, Le Deist, F, Wulffraat, N, McIntyre, E et al. (2003). A serious adverse event after successful gene therapy for X-linked severe combined immunodefciency. N Engl J Med 348: 255-256.
40. Banerjee, R, Tyagi, P, Li, S and Huang, L (2004). Anisamide-targeted stealth liposomes: a potent carrier for targeting doxorubicin to human prostate cancer cells. Int J Cancer 112: 693-700.