Development of pulmonary gene therapy approaches to treat cystic fibrosis and other genetic diseases Section: Genetic and Experimental Medicine Dr Chris Boyd Reader Contact details Email: Chris.Boyd@ed.ac.uk Research in a NutshellCystic 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 People NameRole Dr Chris BoydGroup LeaderAnn DohertyClinical Trial Lab ManagerPaul DickinsonResearch FellowGiulia BartolomucciResearch Assistant Key Publications Chris Boyd Research Explorer Profile Collaborations Dr Gerry McLachlan, University of EdinburghDr David Collie, University of EdinburghProf Eric Alton, Imperial College LondonProf Jane Davies, Imperial College LondonProf Uta Griesenbach, Imperial College LondonDr Deborah Gill, University of OxfordDr Steve Hyde, University of OxfordProf David Sheppard, University of BristolDr Kevin Dhalewal, University of EdinburghDr Pleasantine Mill, University of EdinburghProf Donald Salter, University of Edinburgh Partners and Funders Cystic Fibrosis TrustImperial College of Science Technology and MedicineJust 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