Scientists have slowed the progression of an incurable disease.
Researchers from University College London have achieved a 75% reduction in the progression of a fatal disease of the nervous system using gene therapy.
Scientists from University College London have made a breakthrough in the treatment of Huntington’s disease, a hereditary disease that gradually destroys the nervous system. With the help of a new gene therapy, they managed to slow the development of the disease by 75%, The Guardian and BBC report.
The cause of the disease is a mutation in the HTT gene, which leads to the formation of a toxic protein called huntingtin. It destroys brain cells and causes a gradual deterioration of the condition – from mood swings and uncontrolled movements to dementia and paralysis.
To block the production of the harmful protein, scientists have developed a special drug based on a modified virus. It delivers a “corrective” DNA strand to neurons. The procedure takes 12–20 hours and is administered through a microcatheter into two areas of the brain.
The trial involved 29 patients who received high doses of the drug. Three years after the therapy, they showed a significant slowdown in disease progression, better cognitive and motor performance, and signs of neuronal preservation.
“We now have a treatment for one of the most devastating diseases in the world. This is a truly huge breakthrough. I am really excited,” said Professor Sarah Tabrizi, director of the Huntington’s Disease Centre at University College London.
According to the scientist, the new therapy gives hope that patients will be able to maintain independence and a high quality of life for longer.
It has previously been reported that stem cell transplantation can repair brain damage after a stroke.