Targeting paraspeckles
Dr Tatyana Shelkovnikova,Dr Fadi Soukarieh, Dr Michael Paradowski, Prof John R. Atack, The University of Sheffield and Cardiff University
Advancing Treatments Award
Accelerate new treatments
June 2025 - May 2027
£376,526.00
Summary
When cells become stressed, they present with small granules called “paraspeckles” which help cells survive. The main building block of these paraspeckles is a molecule called NEAT1, and these structures have been found in the intact motor neurons of people with MND. Therefore, paraspeckles may have a protective function and help neurons survive. In addition, several proteins which are affected by MND-associated mutations are known to regulate NEAT1-paraspeckles.
These and other studies by Dr Shelkovnikova’s team suggest that boosting NEAT1-paraspeckle production could have the potential to improve the survival of motor neurons in MND.
Supported by funding from UKRI, the team has looked at approximately 1 million compounds from AstraZeneca’s chemical collection to identify those that can bind to and make NEAT1 more stable, therefore increasing the number of these important paraspeckles. This project aims to take the most promising compounds and investigate their potential as treatments for MND.
Why this research is important
This project will identify whether targeting NEAT1 could be a promising treatment strategy for MND. We hope it will generate the data needed to attract follow-on funding for onward development of the most promising compounds.
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