Huntington’s disease sees first treatment success in UK gene therapy study

Huntington’s disease , one of the most devastating inherited brain disorders , has been treated successfully for the first time. A gene therapy trial led out of the UK has shown dramatic results, slowing the disease’s progress by three-quarters after three years.

The illness , which is passed down through families, is triggered by a single faulty gene. Once active, it steadily destroys brain cells, leading to mood changes, uncontrolled movements, dementia, and, in time, death. Until now, there has been no treatment that could slow it down.

How the therapy works

The treatment, known as AMT-130, is delivered through a lengthy surgical procedure lasting 12 to 20 hours. Doctors use a micro-catheter to slowly infuse a modified virus directly into two areas of the brain. Once inside, th