We have demonstrated that gene therapy can produce improvements in the lungs of CF patients. The key steps have been;
a) to identify methods by which genes can be delivered into the lungs, and
b) to understand that the new genes can secrete proteins both into the lungs and the bloodstream.
After securing a grant from the Wellcome Trusts Innovations funding stream we will apply our underpinning platform technology, based on the rSIV.F/HN lentiviral vector to five new projects:
Influenza A - a major worldwide health risk causing >500,000 deaths each year, mainly affecting vulnerable individals such as the young and elderly.
Pulmonary Fibrosis (PF) - a life-threatening condition with a variety of causes, it has an incidence of 2-30 cases per 100,000 person-years.
α1-Antitrypsin deficiency (AATD) – a rare genetic disease which is present in almost all ethnic groups. Worldwide there are estimated to be ~200,000 patients with AATD.
Haemophilia – a condition affecting around 400,000 individuals worldwide, of whom 85% have Haemophilia A (Factor VIII deficiency) and 15% Haemophilia B (Factor IX deficiency).
Surfactant Protein B (SP-B) deficiency - one of several rare interstitial lung diseases that cause respiratory distress syndrome (RDS) in newborn babies. The only treatment is lung transplantation, which is rarely attempted due to the lack of donor organs and the unstable state of the disease; the majority of individuals affected by SP-B deficiency succumb to the disease within the first few months of life.