Breakthrough Gene Therapy Offers Hope for Huntington’s Disease Patients

A major breakthrough in the fight against Huntington’s disease has been reported by researchers at University College London (UCL) and University College London Hospitals (UCLH).

In a groundbreaking clinical trial, a new gene therapy (AMT-130) slowed the progression of Huntington’s disease by up to 75% over three years in patients receiving a high dose. Delivered as a one-time surgical treatment, the therapy works by targeting the faulty gene that causes Huntington’s, reducing the production of the toxic protein that damages brain cells.

Professor Sarah Tabrizi, Director of the UCL Huntington’s Disease Centre, described the results as “a milestone in our search for a disease-modifying treatment”. For the first time, there is strong evidence that gene therapy can significantly delay the advance of this devastating condition.

https://www.theguardian.com/science/2025/sep/24/huntingtons-disease-treated-successfully-for-first-time-in-gene-therapy-trial?utm_source=chatgpt.com