The main thrust of my research is the development of novel treatment avenues for haematological disorders and cancer using gene transfer technology. I am in addition interested in stem cell biology
My group has developed a distinctive approach for gene therapy of haemophilia B using a self complementary adeno-associated viral vector (AAV), in collaboration with St Jude Children's Research Hospital in Memphis Tennessee. This vector is significantly more potent than conventional AAV vectors. We have demonstrated the preclinical safety and efficacy of this new vector system and are about to commence a Phase I/II study in subjects with severe haemophilia B. Other clinical targets that are currently being evaluated include: Haemophilia A, Congenital FVII and FX deficiency, urea cycle and lysosomal storage disorders.
Haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies. Immunotherapy strategies using engineered T cells are being evaluated for the treatment of chronic lymphocytic leukaemia, a disease that remains incurable. Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required and the role of gene therapy-mediated delivery of these agents is being evaluated.
Recently we have begun a program of research on the use of induced pluripotent stem (iPS) cells for the treatment of degenerative conditions as well as disease models. Through funding from the UKRMP/MRC we evaluating methods for the generation of iPS cells in a GMP environment. In addition, through EU framework support we are assessing the potential of iPS cells as a source of cells for the treatment of malignancies and or organ failure.  
Gene therapy for haematological malignancies: My haemato-oncology research is focused on the development of two emerging strategies: immunotherapy and anti-angiogenesis. Gene-therapy approaches are central to each of these strategies.

Immunotherapy strategies are directed against chronic lymphocytic leukaemia, a disease that remains incurable. Current emphasis is on the adoptive transfer of bi-specific gene modified T cells which recognise CMV infected cells through their native receptor and CLL via a tumour antigen specific receptor. Our ultimate goal is to adoptively transfer bi-specific cells immediately after front line therapy in patients with poor prognosis CLL.
Angiogenesis is required for the growth of tumours including haematological malignancies thus making it an important target for anti-cancer strategies. Long-term expression of an angiogenesis inhibitor is likely to be required for the successful treatment of malignancies. Therefore, gene therapy-mediated delivery of these agents is an alternative way to provide long-term expression of these therapeutic proteins. Vectors expressing a number of different angiogenesis inhibitors are currently being designed and tested in vitro and in vivo in murine models of leukaemia and lymphoma.