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