degenerative brain disorder
UK researchers achieve first successful treatment for Huntington’s disease
For the first time, Huntington’s disease, a hereditary, degenerative brain disorder – has been successfully treated, offering hope to thousands of patients and families affected by the devastating condition, according to UK researchers.
The disease, which destroys brain cells and combines symptoms of dementia, Parkinson’s, and motor neuron disease, has now been slowed by an average of 75% in a clinical trial. Experts say this could extend the decline normally seen in a year to four years, giving patients decades of improved quality of life.
The breakthrough involves an advanced gene therapy administered through 12 to 18 hours of delicate brain surgery. A harmless virus carrying specially designed DNA is infused into key brain regions using MRI-guided microcatheters. This prompts brain cells to produce microRNA, a fragment that silences the mutant huntingtin gene responsible for neuron death, lowering levels of the toxic protein in the brain, reports BBC.
2 months ago