UK researchers said on Wednesday they have slowed the progression of the fatal neural condition Huntington’s disease for the first time with a groundbreaking new gene therapy.
Huntington’s disease causes nerve cells in the brain to decay over time. The disease affects a person’s movements, thinking ability and mental health. Huntington’s disease is rare and it’s often passed down through a changed gene from a parent. Medicines are presently available to help manage the symptoms of Huntington’s disease. However, these current medicines can’t prevent the physical, mental and behavioural decline caused by the disease.
The new 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. Some of the patients who took part in early-stage clinical trials at University College London (UCL) saw the rate 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 new treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery.
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