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GSK, University of Dundee Partner on Huntington’s Disease

Illustration of brain (NIDA)

(National Institute of Drug Abuse)

University of Dundee in Scotland and the pharmaceutical company GlaxoSmithKline (GSK) will collaborate in research to tackle Huntington’s disease, an inherited brain disorder. The joint project is valued at more than £1 million ($US 1.6 million).

Dundee medical professor Susann Schweiger, who has discovered a mechanism that controls production of the disease-causing protein involved in Huntington’s Disease, will lead the research team. The project is expected to bring together expertise in molecular genetics, behavior, brain physiology, and drug development from Dundee with their counterpart researchers at GSK.

Huntington’s disease is an hereditary disorder in which nerve cells in certain parts of the brain waste away, or degenerate. It is caused by defect in a chromosome that stimulates a part of DNA to occur many more times than normal. This section of DNA is normally repeated 10 to 28 times, but with Huntington’s disease, it is repeated 36 to 120 times.

The most common form of Huntington’s disease affects adults, where symptoms appear in people in their 30s and 40s. As the gene is passed down through families, the number of repeats tend to get larger, with a greater chance of developing symptoms at an earlier age. Thus, as the disease is passed along in families, symptoms develop at younger and younger ages.

Because Huntington’s Disease affects a relatively small number of people — as few as 1 in 5,000 to 10,000 — it is considered an orphan disease, which usually attracts little attention from the pharmaceutical industry. The U.S. Food and Drug Administration has given orphan designation to drugs treating Huntington’s disease symptoms.

GSK and Dundee are collaborating on research involving another rare disease, epidermolysis bullosa, also an inherited disorder that results in painful, debilitating and lifelong skin blistering.

Read more: Max Planck Institute, GSK to Collaborate on Diabetes Drugs

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