Nonviral gene transfection nanoparticles: function and applications in the brain

Nanomedicine: Nanotechnology, Biology, and Medicine

Volumn 4, Issue 2, 2008, Pages 89-97

  Roy, Indrajit ^a   Stachowiak, Michal K ^a,b   Bergey, Earl J ^a  

^a UNIVERSITY AT BUFFALO   (United States)

^b UNIVERSITY AT BUFFALO   (United States)

Author keywords

Central nervous system; Gene therapy; Nanoparticles; Nonviral vectors; ORMOSIL

Indexed keywords

BRAIN; CELLS; DISEASES; GENE EXPRESSION; GENE TRANSFER; SILICA;

CENTRAL NERVOUS SYSTEM; ETIOLOGY; GENE THERAPY; NONVIRAL VECTORS; ORMOSIL;

NANOPARTICLES;

CHOLESTEROL; GREEN FLUORESCENT PROTEIN; LIPOFECTIN; LIPOSOME; MACROGOL; NAKED DNA; NANOPARTICLE; PLASMID DNA; POLYETHYLENEIMINE; SILICON DIOXIDE; TRANSFERRIN;

BRAIN; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; MATERIALS TESTING; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; REVIEW;

ANIMALS; BRAIN; DNA, VIRAL; DRUG CARRIERS; GENETIC VECTORS; HUMANS; NANOPARTICLES; NANOTECHNOLOGY; TRANSFECTION;

ANIMALIA;

EID: 44649111835     PISSN: 15499634     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nano.2008.01.002     Document Type: Review

References (58)

1. Verma I.M., and Somia N. Gene therapy-promises, problems and prospects. Nature 389 (1997) 239-242
2. Prasad P.N. Introduction to biophotonics (2004), Wiley-Interscience, New York
3. Austin C.P. The impact of the completed human genome sequence on the development of novel therapeutics for human disease. Annu Rev Med 55 (2004) 1-13
4. Vogelstein B., and Kinzler K.W. Cancer genes and the pathways they control. Nat Med 10 (2004) 789-799
5. Cullen R., and Marshall S. Genetic research and genetic information: a health information professional's perspective on the benefits and risks. Health Info Libr J 23 (2006) 275-282
6. Davis S.S. Biomedical applications of nanotechnology-implications for drug targeting and gene therapy. Trends Biotechnol 15 (1997) 217-224
7. Medina-Kauwe L.K., Xie J., and Hamm-Alvarez S. Intracellular trafficking of nonviral vectors. Gene Ther 12 (2005) 1734-1751
8. Rolland A. Nuclear gene delivery: the Trojan horse approach. Expert Opin Drug Deliv 3 (2006) 1-10
9. Bowers W., and Federoff H. Gene therapy for neurological diseases. In: Templeton N.S. (Ed). Gene and cell therapy: therapeutic mechanisms and strategies. 2nd ed. (2004), Marcel Dekker, New York 601-627
10. Karpati G., Lochmuller H., Nalbantoglu J., and Durham H. The principles of gene therapy for the nervous system. Trends Neurosci 19 (1996) 49-54
11. Anderson W.F. Human gene therapy. Nature 392 6679 Suppl (1998) 25-30
12. Uren A.G., Kool J., Berns A., and van Lohuizen M. Retroviral insertional mutagenesis: past, present and future. Oncogene 24 (2005) 7656-7672
13. Hacein-Bey-Abina S., Von Kalle C., Schmidt M., McCormack M.P., Wulffraat N., Leboulch P., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
14. Davis S. Biomedical applications of nanotechnology. Trends Biotechnol 15 (1997) 217-224
15. Check E. Cancer risk prompts US to curb gene therapy. Nature 422 (2003) 6927
16. Marwick C. FDA halts gene therapy trials after leukaemia case in France. BMJ 326 (2003) 181
17. Lehrman S. Virus treatment questioned after gene therapy death. Nature (1999) 517-518
18. Deglon N., and Hantraye P. Viral vectors as tools to model and treat neurodegenerative disorders. J Gene Med 7 (2005) 530-539
19. Mancheno-Corvo P., and Martin-Duque P. Viral gene therapy. Clin Transl Oncol 8 (2006) 858-867
20. Nishikawa M., and Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 12 (2001) 861-870
21. Bhakta G., Mitra S., and Maitra A. DNA encapsulated magnesium and manganous phosphate nanoparticles: potential non-viral vectors for gene delivery. Biomaterials 26 (2005) 2157-2163
22. Kam N.W., Liu Z., and Dai H. Functionalization of carbon nanotubes via cleavable disulfide bonds for efficient intracellular delivery of siRNA and potent gene silencing. J Am Chem Soc 127 (2005) 12492-12493
23. Roy I., Mitra S., Maitra A., and Mozumdar S. Calcium phosphate nanoparticles as novel non-viral vectors for targeted gene delivery. Int J Pharm 250 (2003) 25-33
24. Roy I., Ohulchanskyy T.Y., Bharali D.J., Pudavar H.E., Mistretta R.A., Kaur N., et al. Optical tracking of organically modified silica nanoparticles as DNA carriers: a nonviral, nanomedicine approach for gene delivery. Proc Natl Acad Sci U S A 102 (2005) 279-284
25. Salem A.K., Searson P.C., and Leong K.W. Multifunctional nanorods for gene delivery. Nat Mater 2 (2003) 668-671
26. Kneuer C., Sameti M., Bakowsky U., Schiestel T., Schirra H., Schmidt H., et al. A nonviral DNA delivery system based on surface modified silica-nanoparticles can efficiently transfect cells in vitro. Bioconjug Chem 11 (2000) 926-932
27. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci U S A 92 (1995) 7297-7301
28. Felgner P.L., Gadek T.R., Holm M., Roman R., Chan H.W., Wenz M., et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci U S A 84 (1987) 7413-7417
29. Mounkes L.C., Zhong W., Cipres-Palacin G., Heath T.D., and Debs R.J. Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo. J Biol Chem 273 (1998) 26164-26170
30. Foster B.J., and Kern J.A. HER2-targeted gene transfer. Hum Gene Ther 8 (1997) 719-727
31. Hood J.D., Bednarski M., Frausto R., Guccione S., Reisfeld R.A., Xiang R., et al. Tumor regression by targeted gene delivery to the neovasculature. Science 296 (2002) 2404-2407
32. Lee R.J., and Huang L. Folate-targeted, anionic liposome-entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J Biol Chem 271 (1996) 8481-8487
33. Pirollo K.F., Zon G., Rait A., Zhou Q., Yu W., Hogrefe R., et al. Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery. Hum Gene Ther 17 (2006) 117-124
34. Holt C.E., Garlick N., and Cornel E. Lipofection of cDNAs in the embryonic vertebrate central nervous system. Neuron 4 (1990) 203-214
35. Ono T., Fujino Y., Tsuchiya T., and Tsuda M. Plasmid DNAs directly injected into mouse brain with lipofectin can be incorporated and expressed by brain cells. Neurosci Lett 117 (1990) 259-263
36. Imaoka T., Date I., Ohmoto T., and Nagatsu T. Significant behavioral recovery in Parkinson's disease model by direct intracerebral gene transfer using continuous injection of a plasmid DNA-liposome complex. Hum Gene Ther 9 (1998) 1093-1102
37. Zou L.L., Huang L., Hayes R.L., Black C., Qiu Y.H., Perez-Polo J.R., et al. Liposome-mediated NGF gene transfection following neuronal injury: potential therapeutic applications. Gene Ther 6 (1999) 994-1005
38. da Cruz M.T., Cardoso A.L., de Almeida L.P., Simoes S., and de Lima M.C. Tf-lipoplex-mediated NGF gene transfer to the CNS: neuronal protection and recovery in an excitotoxic model of brain injury. Gene Ther 12 (2005) 1242-1252
39. Cao Y.J., Shibata T., and Rainov N.G. Liposome-mediated transfer of the bcl-2 gene results in neuroprotection after in vivo transient focal cerebral ischemia in an animal model. Gene Ther 9 (2002) 415-419
40. Shi N., and Pardridge W.M. Noninvasive gene targeting to the brain. Proc Natl Acad Sci U S A 97 (2000) 7567-7572
41. Zhang Y., Schlachetzki F., Zhang Y.F., Boado R.J., and Pardridge W.M. Normalization of striatal tyrosine hydroxylase and reversal of motor impairment in experimental parkinsonism with intravenous nonviral gene therapy and a brain-specific promoter. Hum Gene Ther 15 (2004) 339-350
42. Abdallah B., Hassan A., Benoist C., Goula D., Behr J.P., and Demeneix B.A. A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine. Hum Gene Ther 7 (1996) 1947-1954
43. Goula D., Remy J.S., Erbacher P., Wasowicz M., Levi G., Abdallah B., et al. Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervous system. Gene Ther 5 (1998) 712-717
44. Lemkine G.F., Mantero S., Migne C., Raji A., Goula D., Normandie P., et al. Preferential transfection of adult mouse neural stem cells and their immediate progeny in vivo with polyethylenimine. Mol Cell Neurosci 19 (2002) 165-174
45. Tooyama I., Kawamata T., Walker D., Yamada T., Hanai K., Kimura H., et al. Loss of basic fibroblast growth factor in substantia nigra neurons in Parkinson's disease. Neurology 43 (1993) 372-376
46. Peng H., Moffett J., Myers J., Fang X., Stachowiak E.K., Maher P., et al. Novel nuclear signaling pathway mediates activation of fibroblast growth factor-2 gene by type 1 and type 2 angiotensin II receptors. Mol Biol Cell 12 (2001) 449-462
47. Peng H., Myers J., Fang X., Stachowiak E.K., Maher P.A., Martins G.G., et al. Integrative nuclear FGFR1 signaling (INFS) pathway mediates activation of the tyrosine hydroxylase gene by angiotensin II, depolarization and protein kinase C. J Neurochem 81 (2002) 506-524
48. Corso T.D., Torres G., Goulah C., Roy I., Gambino A.S., Nayda J., et al. Transfection of tyrosine kinase deleted FGF receptor-1 into rat brain substantia nigra reduces the number of tyrosine hydroxylase expressing neurons and decreases concentration levels of striatal dopamine. Brain Res Mol Brain Res 139 (2005) 361-366
49. Corso T.D., Torres G., Goulah C., Roy I., Gambino A.S., Nayda J., et al. Assessment of viral and non-viral gene transfer into adult rat brains using HSV-1, calcium phosphate, and PEI-based methods. Folia Morphol (Warsz) 64 (2005) 130-144
50. Fisher L.J., and Gage F.H. Radical directions in Parkinson's disease. Nat Med 1 (1995) 201-203
51. Wong A.H., Van H., and Tol H. Schizophrenia: from phenomenology to neurobiology. Neurosci Biobehav Rev 27 (2003) 269-306
52. Bharali D.J., Klejbor I., Stachowiak E.K., Dutta P., Roy I., Kaur N., et al. Organically modified silica nanoparticles: a nonviral vector for in vivo gene delivery and expression in the brain. Proc Natl Acad Sci U S A 102 (2005) 11539-11544
53. Frielingsdorf H., Schwarz K., Brundin P., and Mohapel P. No evidence for new dopaminergic neurons in the adult mammalian substantia nigra. Proc Natl Acad Sci U S A 101 (2004) 10177-10182
54. Tabbaa S., Goulah C., Tran R.K., Lis A., Korody R., Stachowski B., et al. Gene transfer into the central nervous system using herpes simplex virus-1 vectors. Folia Morphol (Warsz) 59 (2000) 221-232
55. Goldman J. Peripheral blood stem cells for allografting. Blood 85 (1995) 1413-1415
56. Lois C., and Alvarez-Buylla A. Proliferating subventricular zone cells in the adult mammalian forebrain can differentiate into neurons and glia. Proc Natl Acad Sci U S A 90 (1993) 2074-2077
57. Stachowiak M.K., Fang X., Myers J.M., Dunham S.M., Berezney R., Maher P.A., et al. Integrative nuclear FGFR1 signaling (INFS) as a part of a universal "feed-forward-and-gate" signaling module that controls cell growth and differentiation. J Cell Biochem 90 (2003) 662-691
58. Klejbor I., Stachowiak E.K., Bharali D.J., Roy I., Spodnik I., Morys J., et al. ORMOSIL nanoparticles as a non-viral gene delivery vector for modeling polyglutamine induced brain pathology. J Neurosci Methods 165 (2007) 230-243