Centre of Gene and Cell Therapy

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.

Scientists 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, spinal muscular atrophy and neurofibromatosis, 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.

The following are recent achievements of the scientists at the CGCT:

• Microdystrophin gene therapy in a canine model, with a clinical trial planned
• Gene therapy for oculopharyngeal muscular dystrophy
• Exon skipping for Duchenne, Alzheimer and motor neuron disease
• Genome editing in models of Duchenne, immunodeficiency and ataxia telangiectasia
  
• Regulation of self-renewal in muscle stem cells
• In utero gene delivery to spinal motor neurons

For details you can use the "Researchers", "Publications" and "Projects" tabs above to browse the relevant information.

The research activity in the CGCT has been funded by numerous charitable and public organisations including Action for A-T, the Association Française contre les Myopathies, the Daphne Jackson Trust, the European Union, the Gilbert Family Foundation, Jesse's Journey, Muscular Dystrophy UK, Spinal Muscular Atrophy UK, Spinal Research, Rosetrees Trust, and others. Research has also been supported by Biotech companies like Sarepta Therapeutics, Benitec Biopharma and SynPromics.

You can watch CGCT Director Prof Yáñez-Muñoz in a 5-minute, plain-language videoclip on the Ataxia telangiectasia research: