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UBC examines effects of gene silencing in Huntington's disease

UBC examines effects of gene silencing in Huntington's disease
12th August 2015

Researchers at the University of British Columbia (UBC) have been looking into the effects of gene silencing therapy in Huntington's disease, in the hope to launch the first human clinical trial of a drug that targets the genetic source of the condition.

Developed by UBC's Centre for Molecular Medicine and Therapeutics and the Centre for Huntington Disease, the test aims to reduce the levels of a protein, known as huntingtin, in the brain that is produced via the genetic mutation responsible for the condition. Over time, this protein damages brain cells and scientists hope that lowering the levels of huntingtin will delay, or even prevent, symptoms.

The test, which has already shown positive results in animal models, is able to measure the protein levels in the brain by using a spinal tap to collect cerebrospinal fluid - it can even measure the smallest amounts of the protein. The next step will be to launch a human clinical trial, testing its safety, and it will actually be the first human study of any drug that targets huntingtin.

Blair Leavitt of the study said: "This is an important breakthrough for several promising gene silencing therapies in Huntington's disease that are now moving from the bench to the bedside. We can move forward with these clinical trials and accurately measure whether our treatments are working."

This comes just after a study by Cardiff University, which suggested that scientists are that little bit closer to developing a treatment that may be able to delay, or even prevent, symptoms of Huntington's disease. Led by Professor Lesley Jones, the team looked into the biological mechanisms underlying the illness, examining the DNA of 6,000 people with the disease. It soon emerged that the biological process in the body that controls the repair and manufacturing of DNA could actually influence the age at which a person will develop Huntington's.

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