Huntington’s disease, one of the most destructive inherited brain disorders, was successfully treated for the first time. The study of gene therapy led from the UK showed dramatic results and after three years slowed the progress of the disease by three quarters.
The disease that is passed on by families is triggered by a single defective gene. As soon as it is active, it constantly destroys brain cells, leading to mood changes, uncontrolled movements, dementia and to death. Until now, there has not been a treatment that could slow it down.
“That’s absolutely huge. I’m really excited,” said prof. Sarah Tabrizi, directed by a court at the University College London’s Huntington’s Center’s Center. “Now we have the treatment of one of the more terrible diseases in the world,” Guardian said.
How does the therapy work
The treatment, known as the AMT-130, is supplied by a long surgery, which lasts 12 to 20 hours. Doctors use a microcatter to slowly infusion of a modified virus directly into two areas of the brain. Once inside it is inside, the virus carries a spring of DNA, which turns off the production of toxic protein “Huntingtin”, the main cause of the disease.
Patients need only one shot. However, the complexity of the procedure means that it will be expensive, if it is widely introduced, the BBC reported.
Also read: Kissing Bug In Us: Symptoms, Causes and Risks – What do you need to know about Chagas cosih
The results of an attempt
The study conducted by the Dutch biotechnology company Uniqure was followed by 29 patients in the UK and the US. According to their study, three years after treatment, patients who received a high dose of therapy had 75% slowing the disease progression compared to the control group. Motor skills, memory and everyday functions have all decreased much less than expected.
The brands in the brain also told the same story. Neurofilament levels, cell death signs, were significantly lower in the levels that were administered by a drug.
“These findings strengthen our beliefs that AMT-130 has the potential to transform the treatment landscape for Huntington’s disease,” Dr. Walid Abi-Saab, Chief doctor Uniqure, in a press release issued by a company.
What does it mean for families
Approximately 6,000-10,000 people live with Huntington’s disease in the UK, while at least 20,000 others bears a defective gene. At present, only a fraction is testing, because until now there has been no hope of slowing down the disease.
“Now I think many other people will apply for a genetic test because there is treatment,” Tabrizi Guardian said.
Uniqure said he intended to present the results of the US regulatory organs at the beginning of 2026. For now, scientists and groups of patients have described findings as the most convincing progress yet against illness, which has long been considered incurable.
Frequent
What is Huntington’s disease?
It is a rare genetic brain disorder that causes problems with movement, memory and mood, which eventually leads to dementia and death.
What did the British court show?
The study found that one -off gene therapy slowed the progression of the disease by about 75% after three years.
Who led the study?
The court was led by prof. Sarah Tabrizi, Director of University College London Huntington’s Center’s Center.
What are the new treatment called?
Therapy is known as AMT-130 and has been developed by the biotechnology company Uniqure.
(Tagstotranslate) Huntington’s disease
