Messenger RNA-based therapeutics for the treatment of apoptosis-associated diseases

Scientific Reports

Volumn 5, Issue , 2015, Pages

  Matsui, Akitsugu ^a   Uchida, Satoshi ^a   Ishii, Takehiko ^b   Itaka, Keiji ^a   Kataoka, Kazunori ^a,b  

^a UNIVERSITY OF TOKYO   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; MESSENGER RNA; MICELLE;

ANIMAL; APOPTOSIS; BAGG ALBINO MOUSE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETICS; MICELLE; MOUSE; PLASMID; PROCEDURES;

ANIMALS; APOPTOSIS; DNA; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; MICE; MICE, INBRED BALB C; MICELLES; PLASMIDS; RNA, MESSENGER;

EID: 84945930982     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep15810     Document Type: Article

References (31)

1. Tavernier, G. et al. mRNA as gene therapeutic: how to control protein expression. J Control Release 150, 238-247 (2011).
2. Zangi, L. et al. Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction. Nat Biotechnol 31, 898-907 (2013).
3. Sahin, U., Kariko, K. and Tureci, O. mRNA-based therapeutics-developing a new class of drugs. Nat Rev Drug Discov 13, 759-780 (2014).
4. Zou, S., Scarfo, K., Nantz, M. H. and Hecker, J. G. Lipid-mediated delivery of RNA is more efficient than delivery of DNA in nondividing cells. Int J Pharm 389, 232-243 (2010).
5. Harui, A., Suzuki, S., Kochanek, S. and Mitani, K. Frequency and stability of chromosomal integration of adenovirus vectors. J Virol 73, 6141-6146 (1999).
6. Wang, Z. et al. Detection of integration of plasmid DNA into host genomic DNA following intramuscular injection and electroporation. Gene Ther 11, 711-721 (2004).
7. Kutschka, I. et al. Adenoviral human BCL-2 transgene expression attenuates early donor cell death after cardiomyoblast transplantation into ischemic rat hearts. Circulation 114, I174-180 (2006).
8. Ghavami, S. et al. Apoptosis in liver diseases-detection and therapeutic applications. Medical science monitor: international medical journal of experimental and clinical research 11, RA337-345 (2005).
9. Ichai, P. and Samuel, D. Etiology and prognosis of fulminant hepatitis in adults. Liver Transpl 14 Suppl 2, S67-79 (2008).
10. Uchida, S. et al. In vivo messenger RNA introduction into the central nervous system using polyplex nanomicelle. PLoS One 8, e56220 (2013).
11. Itaka, K. and Kataoka, K. Progress and prospects of polyplex nanomicelles for plasmid DNA delivery. Curr Gene Ther 11, 457-465 (2011).
12. Miyata, K., Nishiyama, N. and Kataoka, K. Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses. Chem Soc Rev 41, 2562-2574 (2012).
13. Kataoka, K., Harada, A. and Nagasaki, Y. Block copolymer micelles for drug delivery: design, characterization and biological significance. Adv Drug Deliv Rev 47, 113-131 (2001).
14. Baba, M., Itaka, K., Kondo, K., Yamasoba, T. and Kataoka, K. Treatment of neurological disorders by introducing mRNA in vivo using polyplex nanomicelles. J Control Release 201, 41-48 (2015).
15. Zhang, G., Budker, V. and Wolff, J. A. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 10, 1735-1737 (1999).
16. Suda, T. and Liu, D. Hydrodynamic gene delivery: its principles and applications. Mol Ther 15, 2063-2069 (2007).
17. Miao, C. H., Thompson, A. R., Loeb, K. and Ye, X. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol Ther 3, 947-957 (2001).
18. Chen, Q. et al. Homo-catiomer integration into PEGylated polyplex micelle from block-catiomer for systemic anti-angiogenic gene therapy for fibrotic pancreatic tumors. Biomaterials 33, 4722-4730 (2012).
19. Kanayama, N. et al. A PEG-based biocompatible block catiomer with high buffering capacity for the construction of polyplex micelles showing efficient gene transfer toward primary cells. ChemMedChem 1, 439-444 (2006).
20. Itaka, K., Ishii, T., Hasegawa, Y. and Kataoka, K. Biodegradable polyamino acid-based polycations as safe and effective gene carrier minimizing cumulative toxicity. Biomaterials 31, 3707-3714 (2010).
21. Warren, L. et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 7, 618-630 (2010).
22. Wang, Y., et al. Systemic delivery of modified mRNA encoding herpes simplex virus 1 thymidine kinase for targeted cancer gene therapy. Mol Ther 21, 358-367 (2013).
23. Kariko, K. et al. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 16, 1833-1840 (2008).
24. Suda, T., Gao, X., Stolz, D. B. and Liu, D. Structural impact of hydrodynamic injection on mouse liver. Gene Ther 14, 129-137 (2007).
25. Liu, F., Song, Y. and Liu, D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 6, 1258-1266 (1999).
26. Itaka, K. et al. Polyplex nanomicelle promotes hydrodynamic gene introduction to skeletal muscle. J Control Release 143, 112-119 (2010).
27. Uchida, S. et al. Combination of chondroitin sulfate and polyplex micelles from Poly(ethylene glycol)-poly{N-[N-(2-aminoethyl)- 2-aminoethyl]aspartamide} block copolymer for prolonged in vivo gene transfection with reduced toxicity. J Control Release 155, 296-302 (2011).
28. Nagata, K. et al. Muscle-targeted hydrodynamic gene introduction of insulin-like growth factor-1 using polyplex nanomicelle to treat peripheral nerve injury. J Control Release 183, 27-34 (2014).
29. Golde, W. T., Gollobin, P. and Rodriguez, L. L. A rapid, simple, and humane method for submandibular bleeding of mice using a lancet. Lab Anim (NY) 34, 39-43 (2005).
30. Song, E. et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 9, 347-351 (2003).
31. Mignon, A. et al. Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes. Nat Med 4, 1185-1188 (1998).