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

Understanding and correcting the mis-folding of SOD1 proteins in MND

Dr Gareth Wright

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

Understanding the role of NEAT1 in MND

Dr Tatyana Shelkovnikova

Investigating the role of NEK1 in MND

Dr Kurt de Vos/Natalie Pye

Investigating synaptic dysfunction in MND

Dr Richard Mead/Dr Alannah Mole

Mapping biological signatures of MND genes

Dr Bradley Smith

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

Investigating the role of adenosine deaminase in MND

Dr Scott Allen

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

The role of microglia in C9orf72-related MND

Prof Kevin Talbot

Dissecting FUS’ cytoplasmic toxic gain-of-function in MND

Dr Marc-David Ruepp/Juan Alcalde

Therapeutic modulation of TDP-43 mechanisms in MND

Prof Jernej Ule

Cystatin C in human MND

Dr Robin Highley

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

Targeting faulty intron retention in MND

Dr Yiran Wang

Investigating the mechanisms which lead to damage of synapses using models of MND

Prof Gareth Miles