Nonviral delivery of self-amplifying RNA vaccines

Proceedings of the National Academy of Sciences of the United States of America

Volumn 109, Issue 36, 2012, Pages 14604-14609

  Geall, Andrew J ^a   Verma, Ayush ^a   Otten, Gillis R ^a   Shaw, Christine A ^a   Hekele, Armin ^a   Banerjee, Kaustuv ^a   Cu, Yen ^a   Beard, Clayton W ^a   Brito, Luis A ^a   Krucker, Thomas ^b   O'Hagan, Derek T ^a   Singh, Manmohan ^a   Mason, Peter W ^a   Valiante, Nicholas M ^a   Dormitzer, Philip R ^a   Barnett, Susan W ^a   Rappuoli, Rino ^a   Ulmer, Jeffrey B ^a   Mandl, Christian W ^a  

^a NOVARTIS VACCINES AND DIAGNOSTICS   (United States)

^b NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

Author keywords

HIV; Respiratory syncytial virus; SAM vaccine; Vaccine platform

Indexed keywords

DNA VACCINE; LIPID NANOPARTICLE; NAKED SELF AMPLIFYING RNA; NANOPARTICLE; PLASMID DNA; PLASMID DNA LAUNCHED SELF AMPLIFYING RNA; RESPIRATORY SYNCYTIAL VIRUS FUSION GLYCOPROTIEN; RESPIRATORY SYNCYTIAL VIRUS VACCINE; RNA VACCINE; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; DRUG POTENCY; FEMALE; IMMUNE RESPONSE; IMMUNOGENICITY; MOUSE; NANOENCAPSULATION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; RESPIRATORY SYNCYTIAL VIRUS INFECTION;

ALPHAVIRUS; ANALYSIS OF VARIANCE; ANIMALS; DRUG DELIVERY SYSTEMS; ELECTROPHORESIS, AGAR GEL; ESCHERICHIA COLI; FEMALE; FLUORESCENT ANTIBODY TECHNIQUE; HUMANS; LIPIDS; NANOPARTICLES; RATS; RNA, SMALL INTERFERING; STATISTICS, NONPARAMETRIC; VACCINES, DNA;

RESPIRATORY SYNCYTIAL VIRUS;

EID: 84865994357     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1209367109     Document Type: Article

References (40)

1. Rappuoli R, Mandl CW, Black S, De Gregorio E (2011) Vaccines for the twenty-first century society. Nat Rev Immunol 11:865-872.
2. Kutzler MA, Weiner DB (2008) DNA vaccines: Ready for prime time? Nat Rev Genet 9: 776-788.
3. Kofler RM, et al. (2004) Mimicking live flavivirus immunization with a noninfectious RNA vaccine. Proc Natl Acad Sci USA 101:1951-1956. (Pubitemid 38228996)
4. Smerdou C, Liljeström P (1999) Non-viral amplification systems for gene transfer: Vectors based on alphaviruses. Curr Opin Mol Ther 1:244-251.
5. Wolff JA, et al. (1990) Direct gene transfer into mouse muscle in vivo. Science 247: 1465-1468.
6. Pascolo S (2008) Vaccination with messenger RNA (mRNA). Handb Exp Pharmacol (183):221-235.
7. Weiss R, Scheiblhofer S, Roesler E, Weinberger E, Thalhamer J (2012) mRNA vaccination as a safe approach for specifi c protection from type I allergy. Expert Rev Vaccines 11:55-67.
8. Tavernier G, et al. (2011) mRNA as gene therapeutic: How to control protein expression. J Control Release 150:238-247.
9. Pascolo S (2004) Messenger RNA-based vaccines. Expert Opin Biol Ther 4:1285-1294.
10. Perri S, et al. (2003) An alphavirus replicon particle chimera derived from venezuelan equine encephalitis and sindbis viruses is a potent gene-based vaccine delivery vector. J Virol 77:10394-10403. (Pubitemid 37129683)
11. Rayner JO, Dryga SA, Kamrud KI (2002) Alphavirus vectors and vaccination. Rev Med Virol 12:279-296.
12. Fleeton MN, et al. (2001) Self-replicative RNA vaccines elicit protection against influenza A virus, respiratory syncytial virus, and a tickborne encephalitis virus. J Infect Dis 183:1395-1398. (Pubitemid 32337123)
13. Johanning FW, et al. (1995) A Sindbis virus mRNA polynucleotide vector achieves prolonged and high level heterologous gene expression in vivo. Nucleic Acids Res 23: 1495-1501.
14. Johansson DX, Ljungberg K, Kakoulidou M, Liljeström P (2012) Intradermal electroporation of naked replicon RNA elicits strong immune responses. PLoS ONE 7:e29732.
15. Probst J, et al. (2006) Characterization of the ribonuclease activity on the skin surface. Genet Vaccines Ther 4:4.
16. Martinon F, et al. (1993) Induction of virus-specific cytotoxic T lymphocytes in vivo by liposome-entrapped mRNA. Eur J Immunol 23:1719-1722. (Pubitemid 23218089)
17. Fotin-Mleczek M, et al. (2011) Messenger RNA-based vaccines with dual activity induce balanced TLR-7 dependent adaptive immune responses and provide antitumor activity. J Immunother 34:1-15.
18. Piggott JM, Sheahan BJ, Soden DM, O'Sullivan GC, Atkins GJ (2009) Electroporation of RNA stimulates immunity to an encoded reporter gene in mice. Mol Med Report 2: 753-756.
19. Semple SC, et al. (2010) Rational design of cationic lipids for siRNA delivery. Nat Biotechnol 28:172-176.
20. Whitehead KA, Langer R, Anderson DG (2009) Knocking down barriers: Advances in siRNA delivery. Nat Rev Drug Discov 8:129- 138.
21. Atkins GJ, Fleeton MN, Sheahan BJ (2008) Therapeutic and prophylactic applications of alphavirus vectors. Expert Rev Mol Med 10:e33.
22. Barnett SW, et al. (2010) Antibody-mediated protection against mucosal simianhuman immunodeficiency virus challenge of macaques immunized with alphavirus replicon particles and boosted with trimeric envelope glycoprotein in MF59 adjuvant. J Virol 84:5975-5985.
23. Robert-Guroff M (2007) Replicating and non-replicating viral vectors for vaccine development. Curr Opin Biotechnol 18:546-556.
24. Zimmer G (2010) RNA replicons - a new approach for influenza virus immunoprophylaxis. Viruses 2:413-434.
25. Bernstein DI, et al. (2009) Randomized, double-blind, Phase 1 trial of an alphavirus replicon vaccine for cytomegalovirus in CMV seronegative adult volunteers. Vaccine 28:484-493.
26. Sardesai NY, Weiner DB (2011) Electroporation delivery of DNA vaccines: Prospects for success. Curr Opin Immunol 23:421-429.
27. Heyes J, Palmer L, Bremner K, MacLachlan I (2005) Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids. J Control Release 107:276-287. (Pubitemid 41316290)
28. Jeffs LB, et al. (2005) A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA. Pharm Res 22:362-372. (Pubitemid 40558494)
29. Swanson KA, et al. (2011) Structural basis for immunization with postfusion respiratory syncytial virus fusion F glycoprotein (RSV F) to elicit high neutralizing antibody titers. Proc Natl Acad Sci USA 108:9619-9624.
30. Prince GA, Horswood RL, Chanock RM (1985) Quantitative aspects of passive immunity to respiratory syncytial virus infection in infant cotton rats. J Virol 55:517-520. (Pubitemid 16249655)
31. Groothuis JR, et al.; The Respiratory Syncytial Virus Immune Globulin Study Group (1993) Prophylactic administration of respiratory syncytial virus immune globulin to high-risk infants and young children. N Engl J Med 329:1524-1530. (Pubitemid 23332437)
32. Piedra PA, Jewell AM, Cron SG, Atmar RL, Glezen WP (2003) Correlates of immunity to respiratory syncytial virus (RSV) associated-hospitalization: Establishment of minimum protective threshold levels of serum neutralizing antibodies. Vaccine 21:3479-3482. (Pubitemid 36792107)
33. Graham BS (2011) Biological challenges and technological opportunities for respiratory syncytial virus vaccine development. Immunol Rev 239:149-166.
34. Dupuis M, et al. (2000) Distribution of DNA vaccines determines their immunogenicity after intramuscular injection in mice. J Immunol 165:2850-2858. (Pubitemid 30660737)
35. Tao W, et al. (2011) Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses. Mol Ther 19:567-575.
36. Wilson KD, et al. (2007) Effects of intravenous and subcutaneous administration on the pharmacokinetics, biodistribution, cellular uptake and immunostimulatory activity of CpG ODN encapsulated in liposomal nanoparticles. Int Immunopharmacol 7: 1064-1075. (Pubitemid 46891326)
37. Rittig SM, et al. (2011) Intradermal vaccinations with RNA coding for TAA generate CD8+ and CD4+ immune responses and induce clinical benefit in vaccinated patients. Mol Ther 19:990-999.
38. Weide B, et al. (2008) Results of the first phase I/II clinical vaccination trial with direct injection of mRNA. J Immunother 31:180-188.
39. Weide B, et al. (2009) Direct injection of protamine-protected mRNA: Results of a phase 1/2 vaccination trial in metastatic melanoma patients. J Immunother 32: 498-507.
40. Jones KL, Drane D, Gowans EJ (2007) Long-term storage of DNA-free RNA for use in vaccine studies. Biotechniques 43:675-681. (Pubitemid 350174918)