UCL Institute for Women's Health (IfWH)

Gene Transfer Technology Group

Lead: Dr Simon Waddington, Reader in Gene Transfer Technology

Research Overview

Dr Simon Waddington leads a team, comprising Drs Suzy Buckley, Ahad Rahim and Natalie Ward, which is focused upon preclinical models of gene and stem cell transfer, particularly for in utero and neonatal gene delivery and for monogenetic disorders of the coagulation cascade. 

Somatotransgenic Bioimaging

We have developed an application of gene transfer whereby signalling pathways in diseased organs and tumours can be quantified continually and non-invasively. This project is being driven by Dr Suzy Buckley in collaboration with Dr Tristan McKay (Queen Mary University of London). More details can be found here.

Brain Disease

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). This is to develop new gene therapy strategies for Neuronopathic Gaucher Disease, a debilitating neurodegenerative disease affecting children. For more information see the Gauchers Association website at www.gaucher.org.uk.  Dr Ahad Rahim is directing this project using adeno-associated virus (AAV) vector. We are also collaborating with Dr Steve Howe (ICH, UCL) on the use of lentiviral vectors for this disease.

Preterm Brain Injury

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 Professors Phil Bennett and Mark Johnson (Imperial College London). Dr Bronwen Herbert is co-ordinating numerous projects while based at UCL. 

Inherited Diseases of Coagulation

As gene therapy is now being used for therapeutic benefit we are investigating its potential application for haemophilia A and B and lethal, early diseases such as factor X deficiency. Dr Natalie Ward, who gained a PhD on gene therapy for haemophilia A, is spearheading this initiative. This work is being performed in collaboration with Professor Adrian Thrasher (ICH, UCl) and Professor John McVey.

Adenovirus vectors

In collaboration with Professor Andy Baker and Dr Stu Nicklin (University of Glasgow) and Professor John McVey. We have elucidated one of the fundamental mechanisms by which adenovirus vectors transduce cells.

Advanced Preclinical Models

We collaborate closely with Dr Anna David (IfWH, UCL) who has developed technology for gene and stem cell transfer to the fetal sheep for potential treatment of fetal growth restriction and for inherited blood diseases. 

We also collaborate with Drs Jerry Chan and Citra Mattar (National University of Singapore) who are concentrating on characterisation of fetal stem cells and the development of NHP models of gene therapy for treatment of early clotting diseases and for thalassemia. 

Further information

There are many other resources which explain the concept of gene therapy for different diseases. The British Society for Gene Therapy represents the gene and cell therapy community in the UK. The European Society of Gene and Cell Therapy represents activities across the whole of Europe. There are also resources accessible to a lay audience such as project recently completed by Nowgen which examines the potential for gene therapy to treat diseases including cystic fibrosis.