It’s been reported that the defect that causes Huntington’s(a neurodegenerative disease),has been successfully corrected for the first time. And it is believed that this Huntington’s disease breakthrough might help in stopping this disease.
According to a research team based in University College London, there is hope that the deadly disease could be put to an end.This could be the biggest breakthrough in the history of Neurodegenerative diseases something that has lasted for almost 50 years.
Huntington’s is a very devastating disease, it could be described as Parkinson’s, Alzheimer’s and motor neuron disease all combined in one.The uncontrollable death of brain cells in this deadly disease leaves patients in a state of permanent decline, this affects their movement, behavior, memory, and ability to think properly.
- Huntington’s mostly affects people in their late 30s and 40s
- Patients with Huntington’s die around 10 to 15 years after the symptoms are noticed
- In the UK about 8,500 people have this deadly disease and a further 25,000 will develop these once they reach the specified age.
- Huntington’s is a disease caused by an inherited defect in a single human gene.It’s autosomal dominant which implies that only one defective copy is enough to develop the disorder.
- The treatment is being designed the gene that causes the disease.
Professor Sarah Tabrizi who led the phase 1 trial in the University College London’s Huntington’s disease center said the results exceeded her expectations.
“The results of this trial are of ground-breaking importance for Huntington’s disease patients and families,”
“For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease,” she said.
The drug known as Ionis-HTTRx, works by blocking and destroying a messenger molecule before the harmful protein can be made, when this occurs, the effects of the mutant gene is silenced.
This could potentially be used in the treatment of other brain disorders such as Alzheimers and Parkinson’s.