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. published online 04 March 2008.

Abstract 

In vivo transfer and expression of foreign genes allows for the elucidation of functions of genes in living organisms and generation of disease models in animals that more closely resemble the etiology of human diseases. Gene therapy holds promise for the cure of a number of diseases at the fundamental level. Synthetic “nonviral” materials are fast gaining popularity as safe and efficient vectors for delivering genes to target organs. Not only can nanoparticles function as efficient gene carriers, they also can simultaneously carry diagnostic probes for direct “real-time” visualization of gene transfer and downstream processes. This review has focused on the central nervous system (CNS) as the target for nonviral gene transfer, with special emphasis on organically modified silica (ORMOSIL) nanoparticles developed in our laboratory. These nanoparticles have shown robust gene transfer efficiency in brain cells in vivo and allowed to investigate mechanisms that control neurogenesis as well as neurodegenerative disorders.

Key words: Gene therapy, Nonviral vectors, Nanoparticles, Central nervous system, ORMOSIL

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 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

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