UK researchers said Wednesday they had slowed the progression of the fatal neural condition Huntington’s disease for the first time with a groundbreaking new gene therapy.
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Some patients who took part in early-stage clinical trials at University College London (UCL) saw the speed at which their condition developed reduced by 75 per cent after three years, according to uniQure, a gene therapy company based in the Netherlands and the United States.
The study tested a new gene therapy, AMT-130, which is delivered through an injection directly into the brain.
Researchers said that AMT-130 works by permanently introducing new functional DNA into a patient’s cells.
“This result changes everything,” said the lead researcher, Ed Wild of UCL’s Huntington’s Disease Centre. “On the basis of these results it seems likely AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff,” he added.
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Gene therapy which could slow human ageing found by Chinese researchers in studies on mice
Gene therapy which could slow human ageing found by Chinese researchers in studies on mice
Some 29 patients took part in the trial with the 12 who were given the highest dose experiencing the greatest slowing of the disease.
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