Scientists look at genetic results


Professor Eric Alton
Academic co-lead
+44 (0)20 7594 7937

Professor Uta Griesenbach
Academic co-lead
+44 (0)20 
7594 7927

MSc Genes Drugs and Stem Cells

What we do

For over 20 year we have worked on developing a gene therapy-based treatment for patients with cystic fibrosis (CF). As part of this work we have recently completed the world’s largest CF gene therapy trial and have shown for the first time that non-viral gene transfer can stabilise lung disease progression in CF patients. In parallel, we have shown that a novel lentiviral vector (rSIV.F/HN) can transduce lungs over 100 fold more efficiently than this leading non-viral formulation, and are now progressing into first-in-man clinical trials. In addition, we are establishing proof-of-concept showing that rSIV.F/HN is a suitable vector for a range of other diseases including alpha 1 anti-trypsin deficiency, pulmonary alveolar proteinosis and haemophilia. 

Why it is important

Cystic fibrosis (CF) patients suffer from chronic lung infection and inflammation which leads to premature death. The median age of death in the UK is currently 28 years. New treatments that improve survival and decrease the significant treatment burden are, therefore, urgently required.

How it can benefit patients

We are a member of the UK Cystic Fibrosis Gene Therapy Consortium (GTC). The translational research performed by the GTC has already shown that gene therapy can stabilise cystic fibrosis lung disease, and we are further building on this success by bringing even more potent gene transfer agents into first-in-man clinical trials. Over 2000 different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can cause CF. Importantly, gene therapy is mutation-type independent and will be suitable for the treatment of patients with all types of CFTR mutations.  We are closely working with Imperial Innovations to establish early interaction with pharmaceutical companies, which is necessary to ensure rapid progression of successful compounds into Phase 3 clinical trials and pave the route to market.

Summary of current research

  • The Gene Therapy Section is a member of the UK Cystic Fibrosis Gene Therapy Consortium and has a long track record in translating gene therapy for cystic fibrosis into the clinic. 
  • We have recently completed the world’s largest CF Phase 2b CF gene therapy trial and shown that gene therapy can stabilise CF lung disease.
  • We have shown that a novel lentiviral vector, which is specifically pseudotyped to transduce lung epithelium is suitable for CF gene therapy and  progression into first-in-man studies.
  • Gene therapy vectors developed as part of our CF programme are also being applied to other congenital and acquired lung diseases.



Related Centres and Facilities


Our researchers

The members of this section have now joined the Respiratory Infection Section following a restructure.