Here you can find more information about the projects we are funding that are investigating therapeutic targets. Click on a project to find out more information and what the project means to a person living with MND.
These projects aim to understand the causes of MND and focus in on the pivotal biochemical processes involved in the disease that will provide a starting point for the development of new treatments.
Investigating the role of RNA binding proteins in FUS MND
Dr Nicol Birsa
Developing AAV-GRN gene therapy for MND
Dr Younbok Lee/Joe Reynolds
Investigating the role of miRNA in human stem cell models of MND
Dr Hamish Crerar
Role of axonal transport in cell models carrying MND mutations
Dr Andrew Tosolini
Determining the mechanisms behind astrocyte and microglia changes in MND
Dr Ben Clarke
Targeting ALS-dysregulated microRNAs in astrocytes
Prof Rob Layfield/Hannah Bailey
Glial engulfment of human synapses in MND
Dr Zsófia László
Investigating the mechanisms which lead to damage of synapses using models of MND
Prof Gareth Miles
Identifying the early biochemical signature of MND
Dr Alex Thompson
Characterising changes in ‘non-coding’ regions of DNA
Dr Jonathan Cooper-Knock/Calum Harvey
Using statistical models and machine learning to find MND subgroups
Dr Alfredo Iacoangeli
Molecular mechanisms of disease onset in TDP-43 related MND
Dr Frank Hirth/Tiziana Ercolani
Investigating the role of NEK1 in MND
Dr Kurt de Vos/Natalie Pye
Investigating synaptic dysfunction in MND
Dr Richard Mead/Dr Alannah Mole
Investigating whether gut-microbes contribute to MND
Prof Chris McDermott
Understanding RNA dysfunction using novel RNA-seq technologies
Prof Pietro Fratta/Samuel Bryce-Smith
Confirming possible subgroups of MND
Dr Alfredo Iacoangeli/Dr Heather Marriott
Dissecting the genes involved in cognitive and behavioural symptoms in MND
Dr Ross Byrne
Investigating the effects of multiple gene mutations in the onset of MND
Prof Majid Hafezparast/Sarah Gornall
The impact of TDP-43 on neuron damage in MND
Prof Pietro Fratta
Investigating the role of C9orf72/SMCR8 in motor neuron communication in MND
Prof Kurt De Vos
Investigating why protein movement is disrupted in C9orf72 MND
Dr Daniel Solomon
Exploring striatal neuron dysfunction in C9orf72 MND-FTD cells
Dr Matthew Livesey/Manpreet Atwal
Role of repetitive proteins in C9orf72-MND waste disposal dysfunction
Dr Sarah Mizielinska/Olivia Houghton
Genetic and environmental risk factors that combine with SARM1 in MND
Prof Michael Coleman/Adina Zhao
Role of transposable elements in MND development & progression
Prof John Quinn
Analysing axonal transport changes in MND
Prof Giampietro Schiavo/Alya Masoud Abdelhafid
Resolving the link between cell processes and axon length in a cell model of MND
Dr Andrea Serio
Understanding the role of NEAT1 in energy metabolism in MND
Dr Tatyana Shelkovnikova/Ruaridh Lang
Investigating mechanisms underlying the impaired hypoxia response in C9Orf72-related MND
Dr Ryan West/Ergita Balli
Investigating how C9orf72 MND may impact cell structure and shape
Dr Ryan West/Charlotte Gale
Dissecting FUS’ cytoplasmic toxic gain-of-function in MND
Dr Marc-David Ruepp/Juan Alcalde
Understanding how NEK1 and C21ORF2 mutations contribute to MND
Prof John Rouse
Analysing gene changes in different brain cells in MND
Dr Johnathan Cooper-Knock
Modelling MND and Frontotemporal Dementia (FTD) in cell cultures
Dr Fiona LeBeau
Investigating the role of TBK1 in microglia behaviour in MND
Dr Owen Peters
Exploring differences between male and female motor neurons in MND
Dr Jemeen Sreedharan
Investigating changes in lipids in cell models of MND
Dr Agnes Nishimura
Synaptic dysfunction in FUS MND: A novel role for FUS in synaptic vesicle biology
Dr Caroline Vance