Background

Many forms of MND and other neurological conditions cause the deterioration and loss of the lower motor neurones (the nerve cells connecting the spinal cord to muscles), such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). Therefore, the ability to slow or halt this deterioration offers a potentially powerful new treatment. 

Previous work has shown that some motor neurones possess a protective characteristic which enables them to resist deterioration during MND. It is now known that one major difference between vulnerable* and disease-resistant* motor neurones is their ability to generate and handle energy.  ‘Energy’ means the ability to generate the 'power' that is required for cells to live and function properly.

*Vulnerable motor neurones: Motor neurones that are lost in MND
*Disease-resistant motor neurones: Motor neurones that are protected from breaking down in MND 

The project

This project will use a combination of different MND models to test whether modifying ‘energy-related genes’ will help protect motor neurones from deteriorating in SMA and ALS. 

The project has three specific aims that will:

• establish whether targeting one ‘key’ energy-related gene (known as PGK1) with a known and approved drug has therapeutic benefits in model of SMA;
• identify other potential ‘key’ energy related genes that could be therapeutically targeted to prevent motor neurones against MND, and;
• establish whether the energy-related genes researchers have found to protect motor neurones in SMA are also capable of protecting motor neurons in models of two distinct forms of ALS (caused by defects in genes for C9orf72 and TDP-43).

Find out more

For more information please contact:

Email: T.Gillingwater@ed.ac.uk
Twitter: @GillingwaterLab

Project title: Identifying and targeting neuroprotective pathways in motor neurons. Please see MND Scotland's Animal Testing Policy.