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
Synaptic dysfunction in FUS MND: A novel role for FUS in synaptic vesicle biology
Dr Caroline Vance
Identifying causes of neuron cell death in C9orf72 MND
Dr Teresa Niccoli
Molecular mechanisms of disease onset in TDP-43 related MND
Dr Frank Hirth/Tiziana Ercolani
Investigating the role of TMEM106B in synapses in MND/FTD
Prof Kurt De Vos
Developing CCDC146 as a therapeutic target for MND
Dr Johnathan Cooper-Knock/Connie Treanor
Investigating whether gut-microbes contribute to MND
Prof Chris McDermott
Investigating changes in lipids in cell models of MND
Dr Agnes Nishimura
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
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
RNA Binding Proteins in MND: From molecular mechanisms to target identification
Dr Matthew White
Exploring differences between male and female motor neurons in MND
Dr Jemeen Sreedharan
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
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 faulty cell communication in human neurons and ways to restore it in MND/FTD
Prof Juan Burrone
Investigating the role of extracellular vesicles in MND and FTD
Dr Alex Thompson
Understanding the role of changes in the TBK1 gene in MND
Dr Sarah Langley/Sophie Shaw
Investigating faulty protein methylation in MND
Dr Judith Sleeman/ Emily Day
Investigating the effects of changes in the ATXN2 gene on cells in MND
Dr Katja Burk
Investigating the role of Annexin A11 gene changes in faulty cell signalling in MND
Dr Bradley Smith
Investigating synaptic dysfunction in MND
Dr Richard Mead/Dr Alannah Mole
Investigating links between changes in the TBK1 gene and inflammation in MND
Dr Owen Peters
Investigating the role of FUS in the early stages of ALS
Dr Caroline Vance/Cara Hatton
Characterising the effects of repeat expansions in brain cells in MND
Dr Russell McLaughlin
Exploring early changes in human motor neuron axons in MND
Dr Andrea Serio
Understanding the role of mitochondria in the faulty communication between neurons in MND
Dr Ruxandra Dafinca