The Centre of Gene and Cell Therapy delivers research, teaching and outreach in gene and cell therapy, with a focus on rare diseases of the neuromuscular and central nervous systems.
Gene therapy is the deliberate alteration of the genome or its function to produce a therapeutic benefit. Sometimes cells are modified outside the body, resulting in gene cell therapy.
Academics from the Biological Sciences, Psychology and History departments work closely together and with external partners to develop novel therapeutics for rare diseases including ataxia telangiectasia, motor neuron disease (ALS), muscular dystrophies and atrophy, and spinal muscular atrophy, but also for more common ailments like spinal injuries, Parkinson Disease, Alzheimer disease and bacterial infections. They also explore public perceptions and understanding of gene therapies including genome editing, and the history of rare diseases.
Training and promotion of junior scientists in research, teaching and outreach activities are key aims for this centre. The outreach programme includes a yearly Rare Disease Day event, celebrated alongside the international day.
Here is a film about our annual Rare Disease Day event: