Nanomedicine: Nanotechnology, Biology and Medicine
Volume 4, Issue 2 , Pages 89-97 , June 2008

Nonviral gene transfection nanoparticles: function and applications in the brain

  • Indrajit Roy, PhD

      Affiliations

    • Department of Chemistry, Institute for Lasers, Photonics, and Biophotonics, State University of New York, Buffalo, New York, USA
  • ,
  • Michal K. Stachowiak, PhD

      Affiliations

    • Department of Chemistry, Institute for Lasers, Photonics, and Biophotonics, State University of New York, Buffalo, New York, USA
    • Molecular and Structural Neurobiology and Gene Therapy Program, Department of Pathology and Anatomical Sciences, State University of New York, Buffalo, New York, USA
  • ,
  • Earl J. Bergey, PhD

      Affiliations

    • Department of Chemistry, Institute for Lasers, Photonics, and Biophotonics, State University of New York, Buffalo, New York, USA
    • Corresponding Author InformationCorresponding author. Department of Chemistry, Institute for Lasers, Photonics, and Biophotonics, State University of New York, Buffalo, New York 14260-3000, USA.

Received 20 April 2007 ,Accepted 28 January 2008.

References 

  1. Verma IM, Somia N. Gene therapy—promises, problems and prospects. Nature. 1997;389:239–242
  2. Prasad PN. Introduction to biophotonics. New York: Wiley-Interscience; 2004;
  3. Austin CP. The impact of the completed human genome sequence on the development of novel therapeutics for human disease. Annu Rev Med. 2004;55:1–13
  4. Vogelstein B, Kinzler KW. Cancer genes and the pathways they control. Nat Med. 2004;10:789–799
  5. Cullen R, Marshall S. Genetic research and genetic information: a health information professional's perspective on the benefits and risks. Health Info Libr J. 2006;23:275–282
  6. Davis SS. Biomedical applications of nanotechnology—implications for drug targeting and gene therapy. Trends Biotechnol. 1997;15:217–224
  7. Medina-Kauwe LK, Xie J, Hamm-Alvarez S. Intracellular trafficking of nonviral vectors. Gene Ther. 2005;12:1734–1751
  8. Rolland A. Nuclear gene delivery: the Trojan horse approach. Expert Opin Drug Deliv. 2006;3:1–10
  9. Bowers W, Federoff H. Gene therapy for neurological diseases. In:  Templeton NS editors. Gene and cell therapy: therapeutic mechanisms and strategies. 2nd ed.. New York: Marcel Dekker; 2004;p. 601–627
  10. Karpati G, Lochmuller H, Nalbantoglu J, Durham H. The principles of gene therapy for the nervous system. Trends Neurosci. 1996;19:49–54
  11. Anderson WF. Human gene therapy. Nature. 1998;392(6679 Suppl):25–30
  12. Uren AG, Kool J, Berns A, van Lohuizen M. Retroviral insertional mutagenesis: past, present and future. Oncogene. 2005;24:7656–7672
  13. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003;302:415–419
  14. Davis S. Biomedical applications of nanotechnology. Trends Biotechnol. 1997;15:217–224
  15. Check E. Cancer risk prompts US to curb gene therapy. Nature. 2003;422:6927
  16. Marwick C. FDA halts gene therapy trials after leukaemia case in France. BMJ. 2003;326:181
  17. Lehrman S. Virus treatment questioned after gene therapy death. Nature. 1999;517–518
  18. Deglon N, Hantraye P. Viral vectors as tools to model and treat neurodegenerative disorders. J Gene Med. 2005;7:530–539
  19. Mancheno-Corvo P, Martin-Duque P. Viral gene therapy. Clin Transl Oncol. 2006;8:858–867
  20. Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther. 2001;12:861–870
  21. Bhakta G, Mitra S, Maitra A. DNA encapsulated magnesium and manganous phosphate nanoparticles: potential non-viral vectors for gene delivery. Biomaterials. 2005;26:2157–2163
  22. Kam NW, Liu Z, Dai H. Functionalization of carbon nanotubes via cleavable disulfide bonds for efficient intracellular delivery of siRNA and potent gene silencing. J Am Chem Soc. 2005;127:12492–12493
  23. Roy I, Mitra S, Maitra A, Mozumdar S. Calcium phosphate nanoparticles as novel non-viral vectors for targeted gene delivery. Int J Pharm. 2003;250:25–33
  24. Roy I, Ohulchanskyy TY, Bharali DJ, Pudavar HE, Mistretta RA, 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. 2005;102:279–284
  25. Salem AK, Searson PC, Leong KW. Multifunctional nanorods for gene delivery. Nat Mater. 2003;2:668–671
  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. 2000;11:926–932
  27. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, 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. 1995;92:7297–7301
  28. Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci U S A. 1987;84:7413–7417
  29. Mounkes LC, Zhong W, Cipres-Palacin G, Heath TD, Debs RJ. Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo. J Biol Chem. 1998;273:26164–26170
  30. Foster BJ, Kern JA. HER2-targeted gene transfer. Hum Gene Ther. 1997;8:719–727
  31. Hood JD, Bednarski M, Frausto R, Guccione S, Reisfeld RA, Xiang R, et al. Tumor regression by targeted gene delivery to the neovasculature. Science. 2002;296:2404–2407
  32. Lee RJ, Huang L. Folate-targeted, anionic liposome-entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J Biol Chem. 1996;271:8481–8487
  33. Pirollo KF, Zon G, Rait A, Zhou Q, Yu W, Hogrefe R, et al. Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery. Hum Gene Ther. 2006;17:117–124
  34. Holt CE, Garlick N, Cornel E. Lipofection of cDNAs in the embryonic vertebrate central nervous system. Neuron. 1990;4:203–214
  35. Ono T, Fujino Y, Tsuchiya T, Tsuda M. Plasmid DNAs directly injected into mouse brain with lipofectin can be incorporated and expressed by brain cells. Neurosci Lett. 1990;117:259–263
  36. Imaoka T, Date I, Ohmoto T, 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. 1998;9:1093–1102
  37. Zou LL, Huang L, Hayes RL, Black C, Qiu YH, Perez-Polo JR, et al. Liposome-mediated NGF gene transfection following neuronal injury: potential therapeutic applications. Gene Ther. 1999;6:994–1005
  38. da Cruz MT, Cardoso AL, de Almeida LP, Simoes S, de Lima MC. Tf-lipoplex-mediated NGF gene transfer to the CNS: neuronal protection and recovery in an excitotoxic model of brain injury. Gene Ther. 2005;12:1242–1252
  39. Cao YJ, Shibata T, Rainov NG. Liposome-mediated transfer of the bcl-2 gene results in neuroprotection after in vivo transient focal cerebral ischemia in an animal model. Gene Ther. 2002;9:415–419
  40. Shi N, Pardridge WM. Noninvasive gene targeting to the brain. Proc Natl Acad Sci U S A. 2000;97:7567–7572
  41. Zhang Y, Schlachetzki F, Zhang YF, Boado RJ, Pardridge WM. 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. 2004;15:339–350
  42. Abdallah B, Hassan A, Benoist C, Goula D, Behr JP, Demeneix BA. A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine. Hum Gene Ther. 1996;7:1947–1954
  43. Goula D, Remy JS, 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. 1998;5:712–717
  44. Lemkine GF, 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. 2002;19:165–174
  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. 1993;43:372–376
  46. Peng H, Moffett J, Myers J, Fang X, Stachowiak EK, 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. 2001;12:449–462
  47. Peng H, Myers J, Fang X, Stachowiak EK, Maher PA, Martins GG, 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. 2002;81:506–524
  48. Corso TD, Torres G, Goulah C, Roy I, Gambino AS, 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. 2005;139:361–366
  49. Corso TD, Torres G, Goulah C, Roy I, Gambino AS, 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). 2005;64:130–144
  50. Fisher LJ, Gage FH. Radical directions in Parkinson's disease. Nat Med. 1995;1:201–203
  51. Wong AH, Van H, Tol H. Schizophrenia: from phenomenology to neurobiology. Neurosci Biobehav Rev. 2003;27:269–306
  52. Bharali DJ, Klejbor I, Stachowiak EK, 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. 2005;102:11539–11544
  53. Frielingsdorf H, Schwarz K, Brundin P, Mohapel P. No evidence for new dopaminergic neurons in the adult mammalian substantia nigra. Proc Natl Acad Sci U S A. 2004;101:10177–10182
  54. Tabbaa S, Goulah C, Tran RK, Lis A, Korody R, Stachowski B, et al. Gene transfer into the central nervous system using herpes simplex virus-1 vectors. Folia Morphol (Warsz). 2000;59:221–232
  55. Goldman J. Peripheral blood stem cells for allografting. Blood. 1995;85:1413–1415
  56. Lois C, 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. 1993;90:2074–2077
  57. Stachowiak MK, Fang X, Myers JM, Dunham SM, Berezney R, Maher PA, 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. 2003;90:662–691
  58. Klejbor I, Stachowiak EK, Bharali DJ, 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. 2007;165:230–243

 This study was supported by grants from the National Institutes of Health (NIH CA119397, NIH CA104492), the John R. Oishei Foundation, the Chemistry and Life Sciences Division of the Air Force Office of Scientific Research, and the University at Buffalo Interdisciplinary Research and Creative Activities Fund. Support from the Center of Excellence in Bioinformatics and Life Sciences is also acknowledged.

PII: S1549-9634(08)00003-8

doi: 10.1016/j.nano.2008.01.002

Nanomedicine: Nanotechnology, Biology and Medicine
Volume 4, Issue 2 , Pages 89-97 , June 2008