Lung Gene Therapy: As Director of the HSC Lung Gene Based Therapy Programme, I am coordinating research and clinical activities to establish an effective and experimentally evaluted Gene Therapy Programme for patients with Cystic Fibrosis and for patients with lung diseases that may be initiated or exacerbated by the generation of free oxygen radicals. The overall approach is to use a liposome-mediated delivery of a plasmid expression cassette. The goal is to treat patients with Cystic Fibrosis on a monthly basis with an aerosolized pharmaceutical product. In contrast to the repetitive treatment required for Cystic Fibrosis the other major focus of the Gene Therapy programme is to target acute lung diseases such as neonatal and adult respiratory distress syndrome and by obtaining appropriate anti-oxidant transgene expression, amilorate the disease by opposing free-radical damage. There are four scientists, a major commitment to the Lung-Gene Therapy programme in addition to other collaborating HSC scientists.
FUTURE RESEARCH: A sensitive assay to evalute sodium channel expression in humans with lung disease is presently being developed, this will enable appropriate clinical studies to determine the role of inadquate or dysfunctional sodium channel function in the pathogenesis of Hyaline Membrane Disease and in patients with pulmonary edema and adult respiratory distress syndrome.
Research is continuing into the development of optimal delivery vehicles for plasmid expression cassettes in addition to optimizing transgene expression by avoiding endosomal destruction in enhanced nuclear targetting in addition to the use of human epithelial specific promotor and LCR constructs.