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Our Research

 Asset 214

The five research groups work synergistically to address various aspects of dementia, as illustrated in the figure above. Our work is streamlined into three research themes: RNA mechanisms (RNA and RNA-binding proteins), therapeutics, and neurophysiology. 

Our core scientific aims are

1. Elucidation of the physiological and pathophysiological roles of RNA-binding proteins in ALS/FTD and other neurodegenerative diseases at the atomic, molecular, cellular, tissue and organismal level.

2. Uncovering the molecular mechanisms maintaining synapse homeostasis and early synapse weakening that precedes synaptic loss and neurodegeneration

3. Development of novel tools and technologies for the investigation of disease mechanisms, including: 

Generation and characterisation of novel, consistent and robust physiological models of neurodegenerative disease 

Development of innovative diagnostic and prognostic tools and methodologies for monitoring protein behaviour at the single molecule and whole tissue level 

4. Translation and application of novel findings into therapeutic approaches, particularly, by:

Targetting shared pathomechanisms across genetic and sporadic forms of disease

Preventing aberrant protein-protein and RNA-protein interactions

Enhancing endogenous neuroprotective pathways

5. Advancement of gene therapy approaches for the treatment of neurodegenerative disease by:

Establishing an AAV Core Facility for the design and production of clinical grade AAV for pre-clinica and clinial studies

Optimising the delivery and distribution of AAVs to the brain and spinal cord

Generating novel AAV therapeutics and testing them in pre-clinical animal models


To learn more about the individual group leaders and their research: 


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