Simon Waddington leads a team, comprising Drs Suzy Buckley and Ahad Rahim, which is focused upon preclinical models of gene and stem cells transfer, particularly for in utero and neonatal gene delivery and for monogenetic disorders of the coagulation cascade. As gene therapy is now being used for therapeutic benefit we are investigating its potential application for lethal, early diseases such as factor X deficiency and neuronopathic Gaucher’s disease. For Gaucher disease we have recently obtained an MRC grant in collaboration with Dr Jon Cooper (KCL), Professor Gennadij Raivich (IfWH, UCL) and Professor Phil Bennett (Imperial College London). Our expertise in mouse models of in utero intervention has enabled us to maintain excellent collaborations in areas such as preterm labour and perinatal brain injury with Professors Donald Peebles and Gennadij Raivich (IfWH, UCL) and Professor Phil Bennett (Imperial College London). Moreover we have developed an application of gene transfer whereby signalling pathways in diseased organs and tumours can be quantified continually and non-invasively. In collaboration with Professor Andy Baker (University of Glasgow) and Professor John McVey (Thrombosis Research Institute, London) we have elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells.