Cystic fibrosis (CF) is a genetic disease that affects over 8000 people in the UK. It results from an inherited genetic mistake in a gene called CFTR causing the build up of thick sticky mucus in the lungs. Eventually the lungs become repeatedly infected and inflamed, leading to lung failure and premature death.  In 2001, research groups from three UK universities, whose Edinburgh component now comprises my CGEM group and that of my colleague Gerry McLachlan at the Roslin Institute, formed the CF Gene Therapy Consortium (GTC) to develop new kinds of treatment based on introducing working copies of the CFTR gene into the lungs of people with CF.

My pulmonary gene therapy group has contributed in many ways to the GTC’s aim to translate gene therapy for CF to the clinic, from immunological detection of the CFTR protein to analysing the effects of viral insertion into human DNA. The Edinburgh groups played a role in preclinical testing of GL67/pGM169, our nonviral gene therapy product for CF, which led in 2012-14 to a major UK-wide double-blind, placebo-controlled Phase IIb clinical trial whose Scottish arm was carried out at the Western General Hospital in Edinburgh. The trial met its primary endpoint and demonstrated significant stabilisation of lung function, galvanising our quest for even greater efficacy. To this end, the GTC has prioritised the development of rSIV.F/HN, a genetically modified lentiviral product -- which is more efficient at delivering genes such as CFTR to the lung -- through conventional funding sources and a partnership with Boehringer Ingelheim and Oxford Biomedica. In parallel, we are pursuing gene therapy approaches for other lung diseases with Wellcome Trust Portfolio funding: my group’s main contribution to this work is the development and testing of lentiviral vectors aimed at treating pulmonary fibrosis.  

I'm a scientist working at the Centre for Genomic and Experimental Medicine. My background is in prokaryotic molecular biology, with a particular interest in plasmids which over the years has evolved into a career in human gene therapy, a research field impinging on many aspects of biology from genome structure to physiology. I lead the Centre’s pulmonary gene therapy group, which since the early 2000s has formed part of the UK CF Gene Therapy Consortium (www.cfgenetherapy.org.uk), a collaboration between Imperial College London, and the universities of Edinburgh and Oxford. I am a long-standing member of the GTC’s strategy group.

My primary research interest is in the pre-clinical development of lung gene therapy based on my genomic engineering expertise, focusing on pulmonary fibrosis, vectorology and insertional genotoxicity.

My group works in CGEM within the Institute of Genetics and Cancer, but we also work closely on transgene expression and related studies with Gerry McLachlan at the Roslin Institute. I am affiliated to the Centre for Inflammation Research.