By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news reporting out of Edinburgh, United Kingdom, by NewsRx editors, research stated, "For gene therapy to improve lung function in cystic fibrosis (CF) subjects, repeated administration of the gene transfer agent over the lifetime of patients is likely to be necessary. This requirement limits the utility of adenoviral and adeno-asociated viral vectors (both previously evaluated in CF gene therapy trials) because of induced adaptive immune responses that render repeated dosing ineffective."
Our news journalists obtained a quote from the research from the University of Edinburgh, "For CF gene therapy trials, non-viral vectors are currently the only viable option. We previously showed that the cationic lipid formulation GL67A is the most efficient of several non-viral vectors analysed for airway gene transfer. Here, we assessed the efficacy and safety of administering 12 inhaled doses of GL67A complexed with pGM169, a CpG-free plasmid encoding human CFTR complementary DNA, into mice. We show that repeated administration of pGM169/GL67A to murine lungs is feasible, safe and achieves reproducible, dose-related and persistent gene expression (>140 days after each dose) using an aerosol generated by a clinically relevant nebuliser."
According to the news editors, the research concluded: "This study supports progression into the first non-viral multidose lung trial in CF patients."
For more information on this research see: Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung. Gene Therapy, 2014;21(1):89-95. Gene Therapy can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group - www.nature.com/; Gene Therapy - www.nature.com/gt/)
Our news journalists report that additional information may be obtained by contacting E. Alton, University of Edinburgh, Roslin Inst, Edinburgh EH4 2XU, Midlothian, United Kingdom. Additional authors for this research include A.C. Boyd, S.H. Cheng, J.C. Davies, L.A. Davies, A. Dayan, D.R. Gill, U. Griesenbach, T. Higgins, S.C. Hyde, J.A. Innes, G. McLachlan, D. Porteous, I. Pringle, R.K. Scheule and S. Sumner-Jones (see also technology.html">Biotechnology).
Keywords for this news article include: Biotechnology, Viral, Virus, Europe, Edinburgh, DNA Research, Gene Therapy, United Kingdom, Bioengineering, Cystic Fibrosis
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