Cell & Gene Therapy

We aim to develop cell and gene therapies to treat inherited disorders, particularly those affecting the blood and liver of children and young people. Conditions we are currently investigating include the common blood disorder thalassaemia and the rare metabolic disorder methylmalonic aciduria.

Research overview

We are investigating possible ways of treating genetic disorders. One method involves gene therapy - introducing 'healthy' copies of genes into a patient's cells. This concept has proved harder to implement than previously thought. For example, the large size of most human genes has necessitated the use of 'stripped-down' versions of these genes. However, minimising the amount of genetic material used can exclude stretches of DNA that would normally control the gene's function.

Safer and more efficient ways of delivering these 'replacement' genes directly to their target cells need to be devised. Research is also required on how to keep the inserted DNA intact and retain its normal functions in the cell. We are very aware of the serious concerns about the safety and effectiveness of gene therapy, and are committed to addressing these issues.

We are also investigating treatments based on cell therapy, and the use of drugs to modify gene expression. In many genetic illnesses, it may even be possible to alter other genes pharmacologically so as to overcome the disease. Our group is developing a number of in vitro and in vivo model systems that can be used to identify and evaluate novel pharmacological agents that can alter the effects of specific gene mutations which cause disease.