First Possible Huntington's Disease Gene Therapy
- Seoyoung Kang
- Sep 27
- 1 min read
Sep 27, 2025
Seoyoung Kang
Recently, a small clinical experiment in the UK found that gene therapy slowed the progression of Huntington's disease by approximately 75% over three years.
Huntington's disease was reported to the academic world by American physician George Huntington in 1872. It is a hereditary brain disease in which nerve cells are gradually damaged. This disease is caused by a genetic mutation. Specifically, the problem arises when the CAG repeat sequence within the HTT gene on chromosome 4 becomes abnormally long. If one parent has Huntington's disease, there's a 50% chance their child will inherit. Unfortunately, it's estimated that there are 40,000 patients worldwide. Europe and the United States are particularly high, with around 1,000 in Japan. As of 2022, the number of patients in Korea is estimated at approximately 340.
The research team tested a novel approach to gene therapy on 29 patients living in the UK and the US. They used a safely modified virus to deliver a specially designed piece of DNA to brain cells, blocking the production of a toxic protein, a key cause of Huntington's disease.
Although there is still no cure, these new findings suggest that gene therapy may eventually open the door to more effective treatments, offering hope to patients and families worldwide.
