ANTISENSE THERAPY: A PROMISING NEW WAY TO TREAT NEUROLOGICAL DISEASE

A new treatment for Huntington’s disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took part in the trial.
Huntington’s disease is caused by a mutation in the gene that makes a protein called huntingtin. The mutation makes the protein bigger and more likely to clump together. It is this increased stickiness that scientists believe causes brain cells to die in people with Huntington’s disease.
As the disease progresses, it affects movement, learning, thinking and emotions.
At the moment, there are no treatments for Huntington’s. Most people die within 15 to 20 years of receiving a diagnosis. But now, at least, there is hope. The new drug, developed by scientists at University College London working with a pharmaceutical company called Ionis, seems to be able to stop the production of the mutant huntingtin protein by silencing the mutated gene. The next stage is to show that this leads to a slowing in progression of the disease./dailyaccord
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