The embryonic field of gene therapy may soon progress to foetal through the utilisation of potent new methods of treatment. Hoping to one day solve many genetically-linked diseases such as Parkinson’s disease, cystic fibrosis, hereditary cancers and multiple system atrophy, gene therapy promises to manipulate the genetic material expressed by patients with these disorders (Yurek, D, 2007). Up until recently, the DNA coding for a desired protein or hormone was introduced to cells via viral vector through a process known as transduction. This extra-chromosomal DNA is then transcribed and translated in a similar way to the cell’s own nucleic chromatin. Unfortunately, the immune response provoked by components of these viruses can lead to swelling and inflammation of the treated tissue: an undesirable side-effect which is potentially complicating in the case of neurological disorders (SFN, 2009).
New research promises to alleviate these worries and vastly increase the efficacy of gene treatment by providing a novel uptake mechanism: DNA nanoparticles. These are incredibly dense rods, toroids or globules of DNA 20-40nm in length that form as plasmids ‘condense’ onto a variety of substrates, including gold and polyethylene glycol polymers (Merkin et al., 2003). The beauty of transfection with nanoparticles is that their effects persist far longer than conventional viral or electroporation techniques with minimal inflammation (Behr et al., 2005).These nanoscopic particles promise to see the fledgling gene therapy field through to adulthood, and will also play integral roles in gene detection and characterisation in future genetic studies.
References
Behr et. al (2005) Monomolecular DNA Nanoparticles for Intravenous Delivery of Genes (Internet) Journal of the American Chemical Society. Available http://pubs.acs.org/doi/abs/10.1021/ja0522332 (accessed 16th March 2009)
Karey et al. (2008) Brain Facts: a Primer on the Brain and Nervous System (Internet), Society for Neuroscience. Available http://www.sfn.org/index.aspx?pagename=brainfacts (Accessed 16th March 2009)
Merkins et. al (2003) Bio/Nanomaterials Subgroup, Northwestern University, Available at
http://chemgroups.northwestern.edu/mirkingroup/BioNanomaterials2003rev1.htm (accessed 16th March 2009)
Yurek, D. (2008) Nanoparticle Therapy for Parkinson's Disease (Internet) UK College of Medicine. Available http://www.nanotechwire.com/news.asp?nid=4393 (accessed 16th March 2009)
Pictures accessed:
