Capturing cell-type specific miRNA deregulation in MND using human stem cell models
To investigate whether molecules known as miRNA are affected in MND and if they are a cause or a consequence of the disease.
Lead Investigator: Dr Hamish Crerar
Lead Institution: University College London and The Francis Crick Insitute
MND Association Funding: £150,000* - Junior Non-Clinical Fellowship
Funding dates: September 2022 - August 2024
*Supported by The Edith Wolfson Foundation Fellowship Programme
About the project
While research made great strides in discovering what is changing at a cellular level, we still have limited understanding as to why. One of these events is the incorrect placement and processing of a molecule known as RNA, which in its various forms is essential for the correct function of motor neurons, the cells most affected in MND. The main aim of this project is to investigate whether a class of small RNA molecules, known as miRNA, are affected in MND and, if they are, whether they are a cause or consequence of the disease. Promising recent studies have shown that pharmacologically manipulating these small RNA molecules can be used to treat disease. Identifying the molecules affected could allow this same technology to be applied to treat MND.
What does this mean for people living with MND?
This project is trying to understand why changes in the cells in MND happen. Further understanding of why things go wrong at a cellular level in MND will help to identify new potential therapeutic targets and help to treat the disease.
Project code: 974-799