CORDIS Project
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This project focuses on developing a gene therapy for Huntington's disease by using engineered proteins to reduce harmful gene products while preserving normal protein levels. It employs viral vectors for targeted delivery to the brain, aiming for a long-term therapeutic solution.
Huntington’s disease (HD) is a lethal inherited neurodegenerative disorder which currently has no cure.
Patients develop a devastating loss of muscle control and brain function, with symptoms typically developing at 35 - 45 years of age, although they can start much earlier.
Suffering is high for both patients and carers, with death generally occurring within 10 - 15 years of diagnosis.
We are working on a novel, curative gene therapy approach based on reducing the levels of the HTT disease gene…
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