HUNTINGTON DISEASE TREATED SUCCESSFULLY BY DOCTORS FOR THE FIRST TIME
For the first time, Huntington's disease, a genetic degenerative illness, has been effectively treated in England. In the battle against Huntington's disease, this enormous discovery marks a sea change and gives thousands of families hope after years of dealing with the illness without any available treatments.
Huntington's disease is a hereditary disorder that alters neurological activity and gradually destroys brain cells. Typically, symptoms start to show up in people's 30s or 40s. Within 20 years after the onset of these symptoms, Huntington's disease usually progresses clinically to death. It is "a combination of dementia, Parkinson's, and motor neuron disease," according to BBC News.
According to researchers at the University College London Huntington's Disease Centre, their medication slowed the disease in patients by 75%, increasing the likelihood that the disease wouldn't even develop in the first place. The ground-breaking procedure involves 12–18 hours of brain surgery during which gene therapy and gene silencing are combined.
The huntingtin gene (HTT), which is essential for the proper operation of the human brain, is the source of the name for Huntington's illness. HTT becomes poisonous due to its mutation. The mutant protein destroys brain cells rather than supplying the HTT required for brain function.
A safe virus with a genetically modified DNA sequence is injected into the brain as part of the treatment developed by the University College London Huntington's Disease Centre. The putamen and the caudate nucleus are two distinct brain areas to which a microcatheter is directed. Both components are linked to execution, mobility, and learning.
The message included in the modified DNA sequence that has now been injected into the brain prevents cells from generating the mutant HTT. The amount of mutant HTT in the brain progressively decreases as a result of the DNA sequence's internal replication over time.
Another encouraging finding from this ground-breaking trial is that the therapy is protecting brain cells in addition to lowering mutant HTT. In actuality, the neurofilament levels in the spinal fluids of the patients were lower than they were at the start of the research. These levels rise in the case of Huntington's development. Patients with stage zero Huntington's disease, who carry the Huntington's gene but have not yet shown symptoms, are currently enrolled in related trials.
The findings represent a significant advancement in the battle against Huntington's disease, even though additional research is required before this treatment is made generally accessible. One day, a condition that was formerly thought to be incurable and untreatable might be prevented or even eliminated.