Scientists give emotional celebration as Huntington’s disease successfully treated for first time

In a massive medical breakthrough, a research team has found a way to slow the progression of Huntington’s disease for the very first time using gene therapy.

Huntington’s disease is a genetically inherited, fatal neurological disorder that progressively damages nerve cells in the brain, causing problems with movement, cognition, and changes in behavior.

First classified in 1872 by American physician George Huntington, there has never been a cure for the condition. Existing treatments only aim to manage symptoms of Huntington’s, which tend to crop up in patients who are around 30 – 40 years of age.

Now, more than a century later, scientists have discovered a way to successfully hinder its progress, leaving the medical community stunned and hopeful.

Researchers slow Huntington’s disease by 75% with gene therapy

On September 24, 2025, a team of medical researchers shared the results of a trial they had conducted over the last three years, which found that a specific type of gene therapy can help slow the progression of Huntington’s disease.

The condition is caused by a mutation of the huntingtin protein in the brain, transforming it into a toxin that attacks and kills other brain cells over time.

Using a combination of gene therapy and gene splicing techniques, scientists were able to infuse a non-threatening ‘virus,’ a vehicle for the gene therapy that had been altered to contain a specific sequence of DNA, into several parts deep within patients’ brains.

After insertion, the DNA then activates, attaching itself to messenger RNA and disrupting the process by which the huntingtin gene’s code is translated into proteins.

The delicate surgical operation takes anywhere from 12-18 hours to complete. Surgeons use a catheter and MRI imaging to inject the therapy, making it equal parts grueling for everyone involved and likely incredibly expensive.

uniQure, a leading company in gene therapy, published the study’s results on September 24, showing that patients experienced an average 75% slowing of Huntington’s three years after undergoing treatment.

Speaking to the BBC, the director of the University College London Huntington’s Disease Centre, Prof Tabrizi, gushed over the trial’s “spectacular” results.

“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” she told the outlet.

Prof Ed Wild, a consultant neurologist at the National Hospital for Neurology and Neurosurgery at UCLH, said he got a “bit teary” at the news.

“There was every chance that we would never see a result like this, so to be living in a world where we know this is not only possible, but the actual magnitude of the effect is breathtaking, it’s very difficult to fully encapsulate the emotion,” he said.