Scientists at the University of Oxford, led by Somerville Fellow Professor Matthew Wood, have been awarded £1m as part of the BioMedical Catalyst funding programme of the Medical Research Council, to tackle the challenge of delivering gene therapy to Huntington’s disease patients.
Huntington’s is a devastating disorder caused by mutations in a single gene, which result in production of a toxic protein. This protein accumulates in the brain, resulting in the progressive degeneration of nerve and muscle cells leading to death, on average within 25 years of diagnosis. Around one in 10,000 people is affected by the condition, for which there is currently no cure.
One potential avenue for treating the disease is via gene therapy, which would target the mutated gene and suppress production of the toxic protein. However, the affected brain tissue is separated from the circulating blood supply, making it difficult to target with drugs.
The Oxford researchers hope to employ a type of naturally occurring cell transport method, called exosomes, to deliver the therapy directly to the brain. They will test their approach in a mouse model of the disease in the hope of proving that this could be a potential mechanism of delivering human therapy.