European Society of Gene and Cell Therapy Conference, Bruges, 2008
The UK Cystic Fibrosis Gene Therapy Consortium is the combined research programme of the three UK gene therapy research groups that have performed cystic fibrosis gene transfer clinical studies. In support of our next phase of clinical studies we have undertaken an extensive pre-clinical research programme to identify a non-viral gene transfer formulation that:-
During this evaluation we identified plasmid derived CG dinucleotides as the major source of host inflammation in response to non-viral gene delivery to the lung and thus developed a range of CG dinucleotide free expression vectors that are capable of sustained lung transgene expression in the absence of host inflammation.