More than 40 different human disorders and diseases, including Huntington’s disease and myotonic dystrophy, are caused by an increase in number of repeated DNA sequence within the associated gene. The DNA sequence is usually less than 30 repeats in the general population. But is more than 40 repeats in affected patients. The more DNA repeats a person inherits, the earlier the age at onset of symptoms and the worse the disease. Most interestingly, the repeat also grows in number throughout the lifetime of the individual, accelerating the disease process. Stopping the repeat from growing during an individual’s lifetime thus presents as a novel therapeutic approach.
In this new project, the three teams will work together to develop novel technologies. It is to monitor how the number of repeats changes in cells grown in the laboratory. This system will then be used to identify new drugs that slow the rate at which the repeat grows. The hope is that one such drug could then be used to treat multiple inherited disorders, including Huntington’s disease and myotonic dystrophy.
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