Possible Cure for Neurodegenerative Diseases? Huntington’s Progress Slowed 75% by Gene Therapy

by Aarav Shah,

Biochem Associate; The Lawrenceville School, NJ

For an extensive amount of time, researchers have struggled to find an effective remedy for Huntington’s disease – one that could both slow down Huntington’s progression and associated symptoms. 

However, in the late days of September 2025, researchers part of the company UniQure produced promising results for finding Huntington’s potential cure. Huntington’s disease is a neurodegenerative disease, one that is caused by an inherited genetic change in the HTT gene. Specifically, this genetic mutation involves segments of the HTT gene to have a three-nucleotide long DNA pattern that recurs multiple times. As a result, the huntingtin protein forms incorrectly and gathers in a cavernous region of the brain known as the striatum. This consistent accumulation results in visible symptoms, including spasms of the muscle, sporadic movements, and a decline in the brain’s cognitive function. Huntington’s disease typically presents itself in an adult aged thirty to forty years.

Previously, researchers were centered around finding a treatment that could lessen the amount of incorrectly formed huntingtin proteins. One specific treatment, antisense oligonucleotides (ASOs), worked by removing mRNA strands responsible for the faulty huntingtin proteins through injections of genetic material into cerebrospinal fluid. However, in 2021, trials for ASOs ended when Roche, a pharmaceutical company, held a trial for its drug tominersen. The results of that trial showed that patients who administered the drug had no significant benefits compared to the control group, with some patients experiencing worsening symptoms. 

So then, how does this new breakthrough in Huntington’s research differ? The new treatment is a type of gene therapy medicine named AMT-130. While it is a one-time neurosurgical procedure, it is quite long and complex. The process involves placing catheters – flexible tubes – inside the brain so that they can deliver the AMT-130 drug into neurons. These neurons happen to be located in the striatum, where Huntington patients tend to have a large accumulation of the incorrectly formed huntingtin proteins. The AMT-130 drug uses adeno-associated viruses carrying genetic information that produces microRNA. This microRNA has the ability to target and destroy mRNA coding for faulty huntington that builds up in the striatum. According to the trials for this potential remedy for Huntington’s disease, those who received the procedure have seen a 75% slower development of Huntington’s as compared to the control group over a three-year period. 

Despite some success with the treatment, it is important to note that three patients in the study experienced intense swelling and headaches as a result of the strenuous surgery. In addition to these side effects is the gargantuan general cost that comes with gene therapy, with the “...NHS [paying] for a £2.6M-per-patient gene therapy for haemophilia B”. For context, haemophilia B is an inherited bleeding disease involving changes in blood clotting factors. As professor Edward Wild, a neurologist, stated, “we must now work no less diligently to turn this breakthrough into something that benefits everyone who needs it” (National Institute for Health and Care Research. 

What’s more, questions about the ethical aspect of this new treatment can arise, specifically on the issue of gene editing. On a note similar to the Baby KJ and CRISPR Gene Editing article published on TechTrek, there needs to be certain safeguards to prevent people from using gene editing for personal gains, either by altering physical appearance or other nonmedical conditions. While it may seem enticing, enhancements in heritable traits are not worth the potential health complications that they may bring, especially if the gene editing is not a medical necessity. Adding on, with the short-term quality of clinical trials, gene editing’s safety in the long term is not predictable for the patients, further raising questions about its proper usage.

In summary, with clinical trials proven to significantly slow progression of Huntington’s disease, the novel gene therapy treatment presented by UniQure has shown significant benefits in reducing the amount of faulty huntingtin protein produced. This treatment not only changes the way we think about mitigating the symptoms of the disease, but also provides new insight into the onset and causes for such illness. Important ethical and equitable questions must be considered, but it is only a matter of further research and persistence until this treatment is available to more Huntington disease patients. 

Works Cited---

Parshall, A. & Lewis, T. (2025, October 1). How Scientists Finally Found a Treatment That Slows Huntington’s Disease. Scientific American. Retrieved from https://www.scientificamerican.com/article/first-treatment-that-slows-huntingtons-disease-comes-after-years-of/

Gallagher, J. (2025, September 24). Huntington’s disease successfully treated for first time. British Broadcasting Corporation. Retrieved from https://www.bbc.com/news/articles/cevz13xkxpro

Kaiser, J. (2025, September 24). In a first, a gene therapy seems to slow Huntington disease. Science. Retrieved from https://www.science.org/content/article/first-gene-therapy-seems-slow-huntington-disease

Treatment for Huntington’s disease shows positive results. (2025, September 25). Retrieved from https://www.nihr.ac.uk/news/treatment-huntingtons-disease-shows-positive-results 

Hemophilia B. (2025, July 24). Retrieved from https://my.clevelandclinic.org/health/diseases/23912-hemophilia-b