Development of pulmonary gene therapy approaches to treat cystic fibrosis and other genetic diseases Section: Genetic and Experimental Medicine Image Dr Chris Boyd - Reader Research in a Nutshell Cystic fibrosis (CF) is a common genetic disease that affects around 10,500 people in the UK. It results from a mutated version of the CFTR gene causing the build-up of thick sticky mucus in the lungs which become repeatedly infected and inflamed, leading to respiratory failure. In 2001, research groups in London, Oxford and Edinburgh, including the CF Gene Therapy Group in CGEM, formed the UK CF Gene Therapy Consortium (www.cfgenetherapy.org.uk) to develop gene therapy for CF lung disease. There are two main research fronts. In Wave 1, our nonviral product (pGM169/GL67A) has been tested in a Phase IIb clinical trial conducted in London and Edinburgh, and was shown to arrest the decline in lung function (Alton et al, 2015, Lancet Respiratory Medicine, 3:684-691). In Wave 2, we are developing a lentiviral vector (rSIV.F/HN) which is more efficient at delivering transgenes to the lung. Wave 2 research is in the preclinical stage, and my Group's main roles are to assess how safe the use of rSIV.F/HN in people is likely to be by studying how the integrated viral DNA interacts with the genome of its target cells, and to monitor CFTR expression from transduced cells. Research Programme: Development of Pulmonary Gene Therapy Research in a Nutshell Image People Dr Chris Boyd Group Leader Ann Doherty Clinical Trial Lab Manager Paul Dickinson Research Fellow Contact Chris.Boyd@ed.ac.uk Collaborations Dr Gerry McLachlan, University of Edinburgh Dr David Collie, University of Edinburgh Prof Eric Alton, Imperial College London Prof Jane Davies, Imperial College London Prof Uta Griesenbach, Imperial College London Dr Deborah Gill, University of Oxford Dr Steve Hyde, University of Oxford Prof David Sheppard, University of Bristol Dr Kevin Dhalewal, University of Edinburgh Dr Pleasantine Mill, University of Edinburgh Prof Donald Salter, University of Edinburgh Partners and Funders Cystic Fibrosis Trust Imperial College of Science Technology and Medicine Just Gene Therapy, Flutterby Fundraisers and other charities Scientific Themes Translational Medicine, Gene Therapy, Synthetic Biology, Recombinant DNA Technology, Airway Cell Biology, Immunohistochemistry, Genotoxicity Technology Expertise Pre-clinical development of gene therapy, vectorology, genomic engineering and synthetic biology This article was published on 2024-09-23
