Chitosan-plasmid nanoparticle formulations for IM and SC delivery of recombinant FGF-2 and PDGF-BB or generation of antibodies

Gene Therapy

Volumn 16, Issue 9, 2009, Pages 1097-1110

  Jean, M ^a   Smaoui, F ^a   Lavertu, M ^a   Methot S ^a,b   Bouhdoud, L ^a   Buschmann, M D ^a   Merzouki, A ^a  

^a ÉCOLE POLYTECHNIQUE DE MONTRÉAL   (Canada)

^b BioSyntech Canada Inc   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AMINO ACID; CHITOSAN; DNA VACCINE; NANOPARTICLE; NEUTRALIZING ANTIBODY; PLASMID DNA; PLATELET DERIVED GROWTH FACTOR BB; RECOMBINANT FIBROBLAST GROWTH FACTOR; SIGNAL PEPTIDE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ANTIBODY SPECIFICITY; ANTIBODY TITER; ARTICLE; CONTROLLED STUDY; DEACETYLATION; DRUG EFFICACY; DRUG SAFETY; ENZYME LINKED IMMUNOSORBENT ASSAY; EXPRESSION VECTOR; GENE EXPRESSION; HISTOPATHOLOGY; MOLECULAR WEIGHT; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN SECRETION; RECOMBINANT PLASMID; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION;

ANIMALS; ANTIBODY FORMATION; BASE SEQUENCE; CHITOSAN; FEMALE; FIBROBLAST GROWTH FACTOR 2; GENE TRANSFER TECHNIQUES; HUMANS; INJECTIONS, INTRAMUSCULAR; INJECTIONS, SUBCUTANEOUS; MICE; MICE, INBRED BALB C; MICROSCOPY, ELECTRON, SCANNING; MOLECULAR SEQUENCE DATA; MUSCLE, SKELETAL; NANOPARTICLES; PLASMIDS; PLATELET-DERIVED GROWTH FACTOR; RECOMBINANT PROTEINS; SKIN;

MUS;

EID: 70249127578     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.60     Document Type: Article

References (60)

1. Coulier F, Pontarotti P, Roubin R, Hartung H, Goldfarb M, Birnbaum D. Of worms and men: An evolutionary perspective on the fibroblast growth factor (FGF) and FGF receptor families. J Mol Evol 1997; 44: 43-56.
2. Fernig DG, Gallagher JT. Fibroblast growth factors and their receptors: an information network controlling tissue growth, morphogenesis and repair. Prog Growth Factor Res 1994; 5: 353-377.
3. Mignatti P, Morimoto T, Rifkin DB. Basic fibroblast growth factor, a protein devoid of secretory signal sequence, is released by cells via a pathway independent of the endoplasmic reticulum-Golgi complex. J Cell Physiol 1992; 151: 81-93.
4. Sohn YD, Lim HJ, Hwang KC, Kwon JH, Park HY, Chung KH et al. A novel recombinant basic fibroblast growth factor and its secretion. Biochem Biophys Res Commun 2001; 284: 931-936.
5. Heldin CH, Ostman A, Ronnstrand L. Signal transduction via platelet-derived growth factor receptors. Biochim Biophys Acta 1998; 1378: F79-113.
6. Millette E, Rauch BH, Defawe O, Kenagy RD, Daum G, Clowes AW. Platelet-derived growth factor-BB-induced human smooth muscle cell proliferation depends on basic FGF release and FGFR-1 activation. Circ Res 2005; 96: 172-179.
7. Ross R, Glomset J, Kariya B, Harker L. A platelet-dependent serum factor that stimulates the proliferation of arterial smooth muscle cells in vitro. Proc Natl Acad Sci USA 1974; 71: 1207-1210.
8. Bennett SP, Griffiths GD, Schor AM, Leese GP, Schor SL. Growth factors in the treatment of diabetic foot ulcers. Br J Surg 2003; 90 133-146.
9. Heldin CH, Westermark B. Mechanism of action and in vivo role of platelet-derived growth factor. Physiol Rev 1999; 79: 1283-1316.
10. Ross R. Platelet-derived growth factor. Lancet 1989; 1: 1179-1182.
11. Nagai MK, Embil JM. Becaplermin: Recombinant platelet derived growth factor, a new treatment for healing diabetic foot ulcers. Expert Opin Biol Ther 2002; 2: 211-218.
12. Kitamura M, Nakashima K, Kowashi Y, Fujii T, Shimauchi H, Sasano T et al. Periodontal tissue regeneration using fibroblast growth factor-2: randomized controlled phase II clinical trial. PLoS ONE 2008; 3 e2611.
13. McGuire MK, Kao RT, Nevins M, Lynch SE. rhPDGF-BB promotes healing of periodontal defects: 24-month clinical and radiographic observations. Int J Periodontics Restorative Dent 2006; 26: 223-231.
14. Cao R, Brakenhielm E, Pawliuk R, Wariaro D, Post MJ, Wahlberg E et al Angiogenic synergism, vascular stability and improvement of hind-limb ischemia by a combination of PDGF-BB and FGF-2. Nat Med 2003; 9: 604-613.
15. MacLaughlin FC, Mumper RJ, Wang J, Tagliaferri JM, Gill I, Hinchcliffe M et al. Chitosan and depolymerized chitosan oligomers as condensing carriers for in vivo plasmid delivery. J Control Release 1998; 56: 259-272.
16. Mao HQ, Roy K, Troung-Le VL, Janes KA, Lin KY, Wang Y et al. Chitosan-DNA nanoparticles as gene carriers: Synthesis, characterization and transfection efficiency. J Control Release 2001; 70: 399-421.
17. Lavertu M, Methot S, Tran-Khanh N, Buschmann MD. High efficiency gene transfer using chitosan/DNA nanoparticles with specific combinations of molecular weight and degree of deacetylation. Biomaterials 2006; 27: 4815-4824.
18. Dodane V, Amin Khan M, Merwin JR. Effect of chitosan on epithelial permeability and structure. Int J Pharm 1999; 182: 21-32.
19. McNeela EA, O'Connor D, Jabbal-Gill I, Illum L, Davis SS, Pizza M et al. A mucosal vaccine against diphtheria: Formulation of cross reacting material (CRM(197)) of diphtheria toxin with chitosan enhances local and systemic antibody and Th2 responses following nasal delivery. Vaccine 2000; 19: 1188-1198.
20. Iqbal M, Lin W, Jabbal-Gill I, Davis SS, Steward MW, Illum L. Nasal delivery of chitosan-DNA plasmid expressing epitopes of respiratory syncytial virus (RSV) induces protective CTL responses in BALB/c mice. Vaccine 2003; 21: 1478-1485.
21. Khatri K, Goyal AK, Gupta PN, Mishra N, Vyas SP. Plasmid DNA loaded chitosan nanoparticles for nasal mucosal immunization against hepatitis B. Int J Pharm 2008; 354: 235-241.
22. Vila A, Sanchez A, Janes K, Behrens I, Kissel T, Vila Jato JL et al Low molecular weight chitosan nanoparticles as new carriers for nasal vaccine delivery in mice. Eur J Pharm Biopharm 2004; 57 123-131.
23. Chew JL, Wolfowicz CB, Mao HQ, Leong KW, Chua KY. Chitosan nanoparticles containing plasmid DNA encoding house dust mite allergen, Der p 1 for oral vaccination in mice. Vaccine 2003; 21: 2720-2729.
24. Roy K, Mao HQ, Huang SK, Leong KW. Oral gene delivery with chitosan-DNA nanoparticles generates immunologic protection in a murine model of peanut allergy. Nat Med 1999; 5: 387-391.
25. Ghendon Y, Markushin S, Krivtsov G, Akopova I. Chitosan as an adjuvant for parenterally administered inactivated influenza vaccines. Arch Virol 2008; 153: 831-837.
26. Lee MK, Chun SK, Choi WJ, Kim JK, Choi SH, Kim A et al. The use of chitosan as a condensing agent to enhance emulsion-mediated gene transfer. Biomaterials 2005; 26: 2147-2156.
27. Yoo HS, Lee JE, Chung H, Kwon IC, Jeong SY. Self-assembled nanoparticles containing hydrophobically modified glycol chitosan for gene delivery. J Control Release 2005; 103: 235-243.
28. Issa MM, Koping-Hoggard M, Tommeraas K, Varum KM, Christensen BE, Strand SP et al. Targeted gene delivery with trisaccharide-substituted chitosan oligomers in vitro and after lung administration in vivo. J Control Release 2006; 115: 103-112.
29. Koping-Hoggard M, Tubulekas I, Guan H, Edwards K, Nilsson M, Varum KM et al. Chitosan as a nonviral gene delivery system. Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo. Gene Therapy 2001; 8: 1108-1121.
30. Koping-Hoggard M, Varum KM, Issa M, Danielsen S, Christensen BE, Stokke BT et al. Improved chitosan-mediated gene delivery based on easily dissociated chitosan polyplexes of highly defined chitosan oligomers. Gene Therapy 2004; 11: 1441-1452.
31. Chen J, Yang WL, Li G, Qian J, Xue JL, Fu SK et al. Transfection of mEpo gene to intestinal epithelium in vivo mediated by oral delivery of chitosan-DNA nanoparticles. World J Gastroenterol 2004; 10: 112-116.
32. de la Fuente M, Seijo B, Alonso MJ. Bioadhesive hyaluron-anchitosan nanoparticles can transport genes across the ocular mucosa and transfect ocular tissue. Gene Therapy 2008; 15: 668-676.
33. Nelea M, Lavertu M, MA PL, Buschman MD. Chitosan-plasmid DNA nanoparticle structure by ESEM and AFM depends on molecular weight and concentrations of plasmid and chitosan. (in preparation).
34. Kang BC, Kang KS, Lee YS. Biocompatibility and long-term toxicity of InnoPol implant, a biodegradable polymer scaffold. Exp Anim 2005; 54: 37-52.
35. Bos GW, Kanellos T, Crommelin DJ, Hennink WE, Howard CR. Cationic polymers that enhance the performance of HbsAg DNA in vivo. Vaccine 2004; 23: 460-469.
36. Bramwell VW, Eyles JE, Somavarapu S, Alpar HO. Liposome/DNA complexes coated with biodegradable PLA improve immune responses to plasmid encoding hepatitis B surface antigen. Immunology 2002; 106: 412-418.
37. Ma PL, Lavertu M, Winnik FM, Buschmann MD. New Insights into chitosan-DNA interactions using isothermal titration microcalorimetry. Biomolecules 2009; (in press).
38. Southwood LL, Frisbie DD, Kawcak CE, McIlwraith CW. Delivery of growth factors using gene therapy to enhance bone healing. Vet Surg 2004; 33: 565-578.
39. Ota T, Maeda M, Tatsuka M. Cationic liposomes with plasmid DNA influence cancer metastatic capability. Anticancer Res 2002; 22: 4049-4052.
40. Kaiser S, Toborek M. Liposome-mediated high-efficiency transfection of human endothelial cells. J Vasc Res 2001; 38: 133-143.
41. Langer R, Cleland JL, Hanes J. New advances in microsphere-based single-dose vaccines. Adv Drug Deliv Rev 1997; 28: 97-119.
42. O'Hagan DT, Valiante NM. Recent advances in the discovery and delivery of vaccine adjuvants. Nat Rev Drug Discov 2003; 2: 727-735.
43. O'Hagan DT, Singh M, Ulmer JB. Microparticles for the delivery of DNA vaccines. Immunol Rev 2004; 199: 191-200.
44. Garcia F, Petry KU, Muderspach L, Gold MA, Braly P, Crum CP et al. ZYC101a for treatment of high-grade cervical intraepithelial neoplasia: a randomized controlled trial. Obstet Gynecol 2004; 103: 317-326.
45. Donnelly JJ, Liu MA, Ulmer JB. Antigen presentation and DNA vaccines. Am J Respir Crit Care Med 2000; 162: S190-S193.
46. Chow YH, Chiang BL, Lee YL, Chi WK, Lin WC, Chen YT et al. Development of Th1 and Th2 populations and the nature of immune responses to hepatitis B virus DNA vaccines can be modulated by codelivery of various cytokine genes. J Immunol 1998; 160: 1320-1329.
47. Schirmbeck R, Reimann J. Modulation of gene-gun-mediated Th2 immunity to hepatitis B surface antigen by bacterial CpG motifs or IL-12. Intervirology 2001; 44: 115-123.
48. Shedlock DJ, Weiner DB. DNA vaccination: Antigen presentation and the induction of immunity. J Leukoc Biol 2000; 68: 793-806.
49. Takashima A, Morita A. Dendritic cells in genetic immunization. J Leukoc Biol 1999; 66: 350-356.
50. Dupuis M, Denis-Mize K, Woo C, Goldbeck C, Selby MJ, Chen M et al. Distribution of DNA vaccines determines their immunogenicity after intramuscular injection in mice. J Immunol 2000; 165: 2850-2858.
51. Jiang ZL, Reay D, Kreppel F, Gambotto A, Feingold E, Kochanek S et al Local high-capacity adenovirus-mediated mCTLA4Ig and mCD40Ig expression prolongs recombinant gene expression in skeletal muscle. Mol Ther 2001; 3: 892-900.
52. Miller M, Rekas G, Dayball K, Wan YH, Bramson J. The efficacy of electroporated plasmid vaccines correlates with long-term antigen production in vivo. Vaccine 2004; 22: 2517-2523.
53. Varum KM, Holme HK, Izume M, Stokke BT, Smidsrod O. Determination of enzymatic hydrolysis specificity of partially N-acetylated chitosans. Biochim Biophys Acta 1996; 1291: 5-15.
54. Chou TC, Fu E, Shen EC. Chitosan inhibits prostaglandin E2 formation and cyclooxygenase-2 induction in lipopolysaccharide-treated RAW 264.7 macrophages. Biochem Biophys Res Commun 2003; 308: 403-407.
55. Huang M, Khor E, Lim LY. Uptake and cytotoxicity of chitosan molecules and nanoparticles: Effects of molecular weight and degree of deacetylation. Pharm Res 2004; 21: 344-353.
56. Otterlei M, Varum KM, Ryan L, Espevik T. Characterization of binding and TNF-alpha-inducing ability of chitosans on monocytes: The involvement of CD14. Vaccine 1994; 12: 825-832.
57. Meyer KB, Thompson MM, Levy MY, Barron LG, Szoka Jr FC. Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics. Gene Therapy 1995; 2: 450-460.
58. Tuomela M, Malm M, Wallen M, Stanescu I, Krohn K, Peterson P. Biodistribution and general safety of a naked DNA plasmid, GTU-MultiHIV, in a rat, using a quantitative PCR method. Vaccine 2005; 23: 890-896.
59. Nguyen S, Winnik F, Buschmann MD. Improved reproducibility in the determination of the molecular weight of chitosan by analytical size exclusion chromatography. Carbohydrate Polymers 2009; 75: 528-533.
60. Lavertu M, Xia Z, Serreqi AN, Berrada M, Rodrigues A, Wang D et al A validated 1H NMR method for the determination of the degree of deacetylation of chitosan. J Pharm Biomed Anal 2003; 32: 1149-1158.