UK scientists slow Huntington’s disease for first time

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.

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% 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 Center.

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