Huntington's disease treated for first time
a day ago
- #gene therapy
- #Huntington's disease
- #medical breakthrough
- Huntington's disease, a devastating genetic disorder, has been successfully treated for the first time, slowing disease progression by 75%.
- The treatment involves a gene therapy delivered via a 12-18 hour brain surgery, targeting the faulty huntingtin gene to reduce toxic protein levels.
- Patients in the trial experienced significant improvements, with some returning to work and others maintaining mobility longer than expected.
- The therapy uses a virus to deliver DNA that helps brain cells produce a molecule to disable the mutant huntingtin protein, preserving neurons.
- Results show the treatment not only slows disease progression but also reduces brain cell death, offering hope for long-term benefits.
- The treatment is expected to be expensive and requires complex surgery, limiting its immediate availability to all patients.
- Researchers are now planning prevention trials for individuals with the gene but no symptoms, aiming to delay or stop the disease entirely.
- Huntington's disease affects approximately 75,000 people in the UK, US, and Europe, with many more carrying the mutation.