Carriers of Huntington’s can begin to display symptoms from 30 – 50 years old, with the disease progressing and symptoms worsening over the next 10 – 30 years. However, scientists at University College London have found a way to slow the onset of Huntington’s by a whole 75%.
Huntington’s disease occurs as a result of the mutation of the huntingtin (HTT) gene, causing an abnormally long repeat of the CAG sequence which ends up producing a faulty huntingtin protein. The faulty protein is what causes the damage nerve cells and the symptoms of this condition.
This new therapy works by introducing a safe virus, across 12 to 18 hours of brain surgery, with a specifically designed DNA sequence, into the brain via a microcatheter using real-time MRI scanning to guide it.
This new therapy works by introducing a safe virus, across 12 to 18 hours of brain surgery, with a specifically designed DNA sequence, into the brain via a microcatheter using real-time MRI scanning to guide it. The safe virus will deliver the designed DNA, which will activate once it reaches brain cells. This causes neurones to avert their own death and intercept signals being sent from the growing mutant huntingtin protein. The end goal of this is to lower the levels of toxic protein in the brain and resulting in a slowing of 75% of the disease.
In terms of the future of the treatment of Huntington’s, this is a significant development from a previously incurable disease. This new method of treatment can give families affected by Huntington’s a better lifestyle and quality of life. The full results of the trial are not yet published to all, but UniQure, the company producing the drug, aim to apply for its US licence in early 2026, to then launch the drug later that year.