Motor Neurone Disease Laboratory

Neurodegenerative diseases have a devastating impact on quality of life and impose a tremendous burden on the health care system.  Among neurodegenerative conditions, motor neurodegenerative diseases are the among the most rapidly fatal, with increasing disability and death within 2-3 years from symptom onset.  Our laboratory is primarily focused on understanding the molecular basis of motor neuron disease (MND), also called amyotrophic lateral sclerosis (ALS). We are also interested in other motor neurodegenerative disorders, including spinal muscular atrophy (SMA) and spinal bulbar muscular atrophy (SBMA), more commonly known as Kennedy's disease.

Our team employs a combination of cell and molecular biology to unravel MND pathogenesis in patient-derived samples, cell culture and animal models.  We seek to identify and understand the primary mechanisms underlying motor neuron vulnerability, stress and injury in MND, while translating our discoveries into relevant biomarkers and targets for effective intervention. Our group is particularly interested in the molecular determinants of protein misfolding and accumulation within neurons, and harnessing endogenous cellular protective mechanisms to combat protein misfolding.  We also investigate mechanisms of misfolded protein propagation in the central nervous system in MND with the goal to arrest disease spread.  

A further area of investigation is the therapeutic action of survival motor neuron (SMN) protein for SMA and MND.  We have discovered that SMN restoration slows disease progression and improves motor neuron survival in mouse models of MND, in addition to SMA mouse models. This has led to development of motor neuron targeted SMN gene therapy approaches for both SMA and MND.

   Motor neurons stained for Hb9 (red) Tuj-1 (green)



Prof Dame Kay Davies and Prof Kevin Talbot, University of Oxford

Dr Severine Boillee, Brain and Spine Institute, Paris

Prof Neil Cashman, University of British Columbia

Prof Uri Saragovi, McGill University



Prof Steve Vucic, University of Sydney

Dr Mary-Louise Rogers and Dr Hakan Muyderman, Flinders University

Dr Justin Yerbury, University of Wollongong

Prof Julie Atkin, Macquarie University

Assoc Prof Danny Hatters, University of Melbourne

Prof Philip Beart and Prof Malcolm Horne, University of Melbourne



Australian National Health and Medical Research Council

Bethlehem Griffiths Research Foundation

Cure for MND Foundation

Motor Neuron Disease Research Institute of Australia

Nick Baldi Construction Pty Ltd

Pratt Foundation

SciOpen Research Group

Stafford Fox Medical Research Foundation

Vowell Foundation

A glimpse at our research

Stimulating autophagy to improve intracellular proteostasis in MNDTargeting exosome-mediated propagation of protein misfolding in MNDDeveloping SMN gene therapy for SMA and MNDGenerating novel mouse models of Kennedy's disease


Scientists in the Neurodegeneration division interrogate how neurones live, die and can be rescued to improve brain function in degenerative conditions such as Parkinson’s and Motor Neuron Diseases. There is no effective treatment for Motor Neurone Disease and the incidence of Parkinson’s Disease is rising alarmingly in our aging community. Gene abnormalities, energy deprivation, toxic rubbish accumulation and inflammation all contribute to a toxic environment for brain cells. Our teams study these events in animal models and cultured cells, with a view to translating knowledge into new therapies for human patients.

All Labs that operate in this Division

Atomic Pathology LaboratoryBiophysics LaboratoryCellular Neurodegeneration LaboratoryCreutzfeldt Jakob Disease Clinical Research GroupMolecular Gerontology LaboratoryMotor Neurone Disease LaboratoryNational Dementia Diagnostics LaboratoryNeurochemistry of Metal IonsNeurogenesis and Neural Transplantation LaboratoryNeuropathology and Neurodegeneration LaboratoryNeuroproteomics and Metalloproteomics LaboratoryNeurotherapeutics LaboratoryParkinson's Disease LaboratoryPrana LaboratoryPre-clinical Parkinson’s Disease Research LaboratoryPresynaptic Physiology Stem Cells and Neural Development LaboratorySteroid Neurobiology LaboratorySynaptic Neurobiology LaboratoryThe Australian Imaging Biomarker and Lifestyle Study (AIBL)