According to a recent report on the UK website Nature, the latest data from UniQure, a gene therapy company based in Amsterdam, the Netherlands, shows that in a trial involving 29 early-stage Huntington's disease patients, participants who received high-dose gene therapy directly injected into the brain experienced a disease progression reduction of about 75% over 3 years compared to the control group. This is the first time that gene therapy has successfully delayed the development of Huntington's disease. Although this therapy is still in its early stages, it is expected to provide new ideas for other neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease. Huntington's disease is a rare genetic neurodegenerative disease caused by excessive DNA repeat sequences in the Huntington protein gene, leading to abnormal protein accumulation in the brain. At present, the disease cannot be cured, and existing drugs can only alleviate symptoms. The gene therapy AMT-130 developed by UniQure delivers a microRNA sequence (microRNA) to affected areas of the brain through harmless viruses. This type of microRNA can 'shut down' defective genes and prevent the production of abnormal proteins. AMT-130 therapy targets the two brain regions where Huntington's disease first invades - the caudate nucleus and the putamen. Doctors need to use real-time brain scans and fine catheters for precise injections, and the entire process takes 12-18 hours. One injection may permanently reduce the level of mutant protein. Preliminary results show that the gene therapy slows down disease progression by about 75%, indicating that AMT-130 has the potential to fundamentally change the treatment status of Huntington's disease. At present, this therapy has good safety, with common side effects of headache and confusion, most of which can be self relieved or controlled through steroids. The company is expected to submit an application to the US Food and Drug Administration early next year. If approved, the relevant drugs are expected to be launched before 2027. (New Society)