NUI Galway leads new research project on neurological diseases

Professor Bob Lahue, of the Centre for Chromosome Biology and the Galway Neuroscience Centre at NUI Galway. Credit: NUI Galway.
May 03 2022 Posted: 11:00 IST

Researchers at three universities in Ireland, Scotland and Wales are joining forces to develop new tools for identifying drugs to treat multiple, currently incurable, neurological diseases.

More than 40 different human disorders, 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 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.

Professor Bob Lahue, of the Centre for Chromosome Biology and the Galway Neuroscience Centre at NUI Galway, said: “There are several related neurological diseases, such as Huntington’s, for which we do not have treatments that can alter the course of the disease. The focus of the research in this project is to develop new technologies which allow us to better understand what causes these diseases.”

Professor Lahue is leading the research and working with research groups at the University of Glasgow, led by Professor Darren Monckton, and at the UK Dementia Research Institute at Cardiff University, led by Professor Vincent Dion.

“We are very fortunate that Professor Monckton at the University of Glasgow and Professor Dion at Cardiff University are part of the efforts to learn more about these neurological diseases. The three of us have complementary strengths in a common cause to understand and combat disease-causing genetic mutations known as triplet repeat expansions.”

The project is titled TRXassay - Development of a novel pre-clinical assay to detect triplet repeat expansions and is co-funded by the European Joint Programme on Rare Diseases (EJP-RD) and by LoQus23 Therapeutics Ltd of Cambridge, UK.  

Speaking on behalf of the EJP-RD, Dr Christine Fetro of the Foundation for Rare Diseases said: “The TRXassay was one of three projects selected for funding within the Rare Diseases Research (RDR) Challenges call led by Fondation Maladies Rares and EJP-RD. This collaborative project brings together academic innovators with an industry partner to drive rare disease research towards effective treatments, which is at the very heart of this European funding initiative.”

Dr David Reynolds, chief executive of LoQus23, said: “We are delighted to be involved in this exciting project with leading academics in the field. The project has potential to deliver assays suitable for discovering therapeutics to treat Huntington’s and other triplet repeat diseases.”


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