Safety assessment of biolistic DNA vaccination

Methods in Molecular Biology

Volumn 940, Issue , 2013, Pages 371-388

  Langer, Barbara ^a   Renner, Matthias ^a   Scherer, Jürgen ^a   Schüle, Silke ^a   Cichutek, Klaus ^a  

^a PAUL EHRLICH INSTITUT   (Germany)

Author keywords

Biodistribution; Biolistic DNA delivery; Biosafety; Mutagenesis

Indexed keywords

DNA VACCINE;

ANIMAL; ARTICLE; AUTOIMMUNITY; BIOLISTIC TRANSFORMATION; GENETICS; HUMAN; IMMUNOLOGICAL TOLERANCE; IMMUNOLOGY; INSTRUMENTATION; SAFETY; VACCINATION;

ANIMALS; AUTOIMMUNITY; BIOLISTICS; HUMANS; IMMUNE TOLERANCE; SAFETY; VACCINATION; VACCINES, DNA;

EID: 84870531992     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-62703-110-3_27     Document Type: Article

References (73)

1. U.S. National Library of Medicine, National Institutes of Health, Clinical Trials database < http://clinicaltrials.gov/ct2/search >.
2. European Medicines Agency, EU Clinical Trials register < https://www.clinicaltrialsregister. eu/contacts.html >.
3. Liu MA (2011) DNA vaccines: an historical perspective and view to the future. Immunol Rev 239:62-84
4. Kendall M (2006) Engineering of needle-free physical methods to target epidermal cells for DNA vaccination. Vaccine 24:4651-4656
5. Banchereau J, Steinman RM (1998) Dendritic cells and the control of immunity. Nature 392:245-252
6. Albert ML, Sauter B, Bhardwaj N (1998) Dendritic cells acquire antigen from apoptotic cells and induce class I-restricted CTLs. Nature 392:86-89
7. Donnelly JJ, Wahren B, Liu MA (2005) DNA vaccines: progress and challenges. J Immunol 175:633-639
8. Feltquate DM et al (1997) Different T helper cell types and antibody isotypes generated by saline and gene gun DNA immunization. J Immunol 158:2278-2284
9. Ferraro B et al (2011) Clinical applications of DNA vaccines: current progress. Clin Infect Dis 53:296-302
10. Otten G et al (2004) Enhancement of DNA vaccine potency in rhesus macaques by electroporation. Vaccine 22:2489-2493
11. Satkauskas S et al (2001) Slow accumulation of plasmid in muscle cells: supporting evidence for a mechanism of DNA uptake by receptormediated endocytosis. Mol Ther 4:317-323
12. Barry ME et al (1999) Role of endogenous endonucleases and tissue site in transfection and CpG-mediated immune activation after naked DNA injection. Hum Gene Ther 10:2461-2480
13. Dale CJ et al (2006) Prime-boost strategies in DNA vaccines. Methods Mol Med 127:171-197
14. Gurunathan S, Klinman DM, Seder RA (2000) DNA vaccines: immunology, application, and optimization. Annu Rev Immunol 18:927-974
15. Schüle S et al (2010) Regulatory requirements for clinical trial and marketing authorisation application for gene therapy medicinal products. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 53:30-37
16. 2009/120/EC, The Commission of the European communities. Off J Eur Comm L 242 15(9)2009, p. 3-12. < http://ec.europa.eu/health/files/eudralex/vol-1/ dir-2009-120/dir-2009-120-en.pdf>.
17. 2001/83/EC. The European Parliament and the Council of the European Union. Off. J Eur Comm. L311, 67-126 (2001). < http://ec.europa.eu/health/fi les/eudralex/vol-1/dir-2001-83-cons2009/2001-83-cons2009-en.pdf >.
18. Gaspar HB et al (2004) Gene therapy of X-linked severe combined immunode ficiency by use of a pseudotyped gammaretroviral vector. Lancet 364:2181-2187
19. Cavazzana-Calvo M et al (2000) Gene therapy of human severe combined immunode ficiency (SCID)-X1 disease. Science 288:669-672
20. Fischer A, Hacein-Bey-Abina S, Cavazzana- Calvo M (2010) 20 years of gene therapy for SCID. Nat Immunol 11:457-460
21. Wang Z et al (2004) Detection of integration of plasmid DNA into host genomic DNA following intramuscular injection and electroporation. Gene Ther 11:711-721
22. Ledwith BJ et al (2000) Plasmid DNA vaccines: investigation of integration into host cellular DNA following intramuscular injection in mice. Intervirology 43:258-272
23. Kutzler MA, Weiner DB (2008) DNA vaccines: ready for prime time? Nat Rev Genet 9:776-788
24. Faurez F et al (2010) Biosafety of DNA vaccines: new generation of DNA vectors and current knowledge on the fate of plasmids after injection. Vaccine 28:3888-3895
25. WHO. World Health Organization: Guidelines for assuring the quality and nonclinical safety evaluation of DNA vaccines; WHO Technical Report Series No 941, 2007, Annex 1. < http://www.who.int/biologicals/publications/trs/areas/ vaccines/dna/Annex%201-DNA%20vaccines.pdf >.
26. Food and Drug Administration: Guidance for Industry: Considerations for plasmid DNA vaccines for infectious disease indications, 2007 < http://www.fda.gov/biologicsbloodvaccines/ guidancecomplianceregulatoryinformation/guidances/vaccines/ucm074770. htm >
27. Committee for the Medicinal Product for Human Use (CHMP): Guideline on safety and ef ficacy follow-up - risk management of advanced therapy medicinal products (EMEA/149995/2008) < http://www.ema. europa.eu/docs/en-GB/document- library/Regulator y-And-procedural-guideline/2009/10/WC500006326.pdf >.
28. Committee for Medicinal Products for Human Use CHMP/GTWP/60436/07. Follow-up of patients administered with gene therapy medicinal products (Draft). EMEA, London. 2008. < http://www.emea.europa.eu/pdfs/human/genetherapy/ 6043607endraft.pdf >.
29. Manam S et al (2000) Plasmid DNA vaccines: tissue distribution and effects of DNA sequence, adjuvants and delivery method on integration into host DNA. Intervirology 43:273-281
30. Bureau MF et al (2004) Intramuscular plasmid DNA electrotransfer: biodistribution and degradation. Biochim Biophys Acta 1676:138-148
31. Gallot D et al (2002) Systemic diffusion including germ cells after plasmidic in utero gene transfer in the rat. Fetal Diagn Ther 17:157-162
32. Schalk JA et al (2006) Preclinical and clinical safety studies on DNA vaccines. Hum Vaccin 2:45-53
33. 273974/2005. EMEA/CHMP: Guideline on non-clinical testing for inadvertent germline transmission of gene transfer vectors < http://www.emea.europa.eu/ pdfs/human/swp/27397405en fi n.pdf >.
34. Donnelly JJ et al (1997) DNA vaccines. Annu Rev Immunol 15:617-648
35. Mor G, Eliza M (2001) Plasmid DNA vaccines. Immunology, tolerance, and autoimmunity. Mol Biotechnol 19:245-250
36. Pavlovic M et al (2010) Pathogenic and epiphenomenal antI-DNA antibodies in SLE. Autoimmune Dis 2011:462841
37. Manson JJ, Isenberg DA (2006) The origin and pathogenic consequences of antI-dsDNA antibodies in systemic lupus erythematosus. Expert Rev Clin Immunol 2:377-385
38. Isenberg DA et al (2007) Fifty years of antI-ds DNA antibodies: are we approaching journey's end? Rheumatology (Oxford) 46:1052-1056
39. Ichino M et al (1999) Factors associated with the development of neonatal tolerance after the administration of a plasmid DNA vaccine. J Immunol 162:3814-3818
40. Fioretti D et al (2010) DNA vaccines: developing new strategies against cancer. J Biomed Biotechnol 2010:174378
41. Mor G et al (1996) Induction of neonatal tolerance by plasmid DNA vaccination of mice. J Clin Invest 98:2700-2705
42. van Drunen Littel-van den Hurk S, Hannaman D (2010) Electroporation for DNA immunization: clinical application. Expert Rev Vaccines 9, 503-517
43. Dean HJ (2005) Epidermal delivery of protein and DNA vaccines. Expert Opin Drug Deliv 2:227-236
44. Anliker B, Longhurst S, Buchholz CJ (2010) Environmental risk assessment for medicinal products containing genetically modi fied organisms. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz 53:52-57
45. ERA. Committee for the Medicinal Product for Human Use (CHMP). Guideline on scienti fic requirements for the environmental risk assessment of gene therapy medicinal products. Doc. Ref. EMEA/CHMP/GTWP/125491/2006. EMEA, London, 2008. < >.
46. Droge M, Puhler A, Selbitschka W (1998) Horizontal gene transfer as a biosafety issue: a natural phenomenon of public concern. J Biotechnol 64:75-90 5.14. European Pharmacopeia, 7th Edition, Supplement 6.3. Chapter 5.14. Gene Transfer Medicinal Products for Human Use. 01/2008 :54100, correct 6.0.
47. 5.14. European Pharmacopeia, 7th Edition, Supplement 6.3. Chapter 5.14. Gene Transfer Medicinal Products for Human Use. 01/2008:54100, correct 6.0. 48. Degryse E (1991) Stability of a host-vector system based on complementation of an essential gene in Escherichia coli. J Biotechnol 18:29-39
48. Mairhofer J et al (2008) A novel antibiotic free plasmid selection system: advances in safe and ef ficient DNA therapy. Biotechnol J 3:83-89
49. Cranenburgh RM et al (2001) Escherichia coli strains that allow antibiotic-free plasmid selection and maintenance by repressor titration. Nucleic Acids Res 29:e26
50. Garmory HS et al (2005) Antibiotic-free plasmid stabilization by operator-repressor titration for vaccine delivery by using live Salmonella enterica Serovar typhimurium. Infect Immun 73:2005-2011
51. Williams SG et al (1998) Repressor titration: a novel system for selection and stable maintenance of recombinant plasmids. Nucleic Acids Res 26:2120-2124
52. Hanke T, McMichael AJ (2000) Design and construction of an experimental HIV-1 vaccine for a year-2000 clinical trial in Kenya. Nat Med 6:951-955
53. Hanke T et al (2002) Lack of toxicity and persistence in the mouse associated with administration of candidate DNA- and modi fied vaccinia virus Ankara (MVA)-based HIV vaccines for Kenya. Vaccine 21:108-114
54. Darquet AM et al (1997) A new DNA vehicle for nonviral gene delivery: supercoiled minicircle. Gene Ther 4:1341-1349
55. Kay MA, He CY, Chen ZY (2010) A robust system for production of minicircle DNA vectors. Nat Biotechnol 28:1287-1289
56. Schirmbeck R et al (2001) Priming of immune responses to hepatitis B surface antigen with minimal DNA expression constructs modi fied with a nuclear localization signal peptide. J Mol Med (Berl) 79:343-350
57. Lopez-Fuertes L et al (2002) DNA vaccination with linear minimalistic (MIDGE) vectors confers protection against Leishmania major infection in mice. Vaccine 21:247-257
58. Stacey KJ, Sweet MJ, Hume DA (1996) Macrophages ingest and are activated by bacterial DNA. J Immunol 157:2116-2122
59. Krieg AM et al (1995) CpG motifs in bacterial DNA trigger direct B-cell activation. Nature 374:546-549
60. Jakob T et al (1998) Activation of cutaneous dendritic cells by CpG-containing oligodeoxynucleotides: a role for dendritic cells in the augmentation of Th1 responses by immunostimulatory DNA. J Immunol 161:3042-3049
61. Landrigan A, Wong MT, Utz PJ (2011) CpG and non-CpG oligodeoxynucleotides directly costimulate mouse and human CD4+ T cells through a TLR9- and MyD88- independent mechanism. J Immunol 187:3033-3043
62. Sato Y et al (1996) Immunostimulatory DNA sequences necessary for effective intradermal gene immunization. Science 273:352-354
63. Lindau D et al (2011) Nucleosome-induced neutrophil activation occurs independently of TLR9 and endosomal acidi fication: implications for systemic lupus erythematosus. Eur J Immunol 41:669-681
64. Yasuda K et al (2009) Requirement for DNA CpG content in TLR9-dependent dendritic cell activation induced by DNA-containing immune complexes. J Immunol 183:3109-3117
65. Ada G, Ramshaw I (2003) DNA vaccination. Expert Opin Emerg Drugs 8:27-35
66. Yamada H et al (2002) Effect of suppressive DNA on CpG-induced immune activation. J Immunol 169:5590-5594
67. Krieg AM et al (1998) Sequence motifs in adenoviral DNA block immune activation by stimulatory CpG motifs. Proc Natl Acad Sci U S A 95:12631-12636
68. Tolmachov O (2009) Designing plasmid vectors. Methods Mol Biol 542:117-129
69. Wooddell CI et al (2011) Muscle damage after delivery of naked plasmid DNA into skeletal muscles is batch dependent. Hum Gene Ther 22:225-235
70. Magalhaes PO et al (2007) Methods of endotoxin removal from biological preparations: a review. J Pharm Pharm Sci 10:388-404
71. Schleef M et al (2010) Production of non viral DNA vectors. Curr Gene Ther 10:487-507
72. MacGregor RR et al (1998) First human trial of a DNA-based vaccine for treatment of human immunode ficiency virus type 1 infection: safety and host response. J Infect Dis 178:92-100
73. Lu S, Wang S, Grimes-Serrano JM (2008) Current progress of DNA vaccine studies in humans. Expert Rev Vaccines 7:175-191