An online scientific seminar series focused on motor neurone disease (MND) Research. Hear from national experts and emerging leaders in MND research including the latest clinical trials, drug developments, pre-clinical models, discovery research and collaborative platforms. Learn, connect and share with the Australian MND research community.
For enquiries related to the seminar series, please contact: Dr Sarah Bennett
MND-associated SOD1 mutations increase the propensity of the SOD1 protein to misfold and aggregate, leading to motor neuron degeneration. The SOD1 antisense oligonucleuotide, Tofersen provides evidence that specifically targeting SOD1 is clinically beneficial. However, Tofersen may only be the first step in the pathway to treat SOD1-associated MND. In this seminar I will demonstrated that therapeutically addressing multiple steps in the SOD1 cycle including translation, folding and degradation may provide further therapeutic opportunities. Specifically, I will showcase research demonstrating improved SOD1 ASO delivery using nanoparticle focused ultrasound technology, the prevention of SOD1 misfolding with the use of small molecules and aiding of misfolded SOD1 degradation with bioengineered proteins to provide neuroprotection in SOD1-MND models.
Jeremy Lum is a Bill Gole MND Postdoctoral Fellow in the School of Medical, Health and Indigenous Science at the University of Wollongong. He received his PhD in cellular neuroscience and neuropharmacology in 2019. He currently leads an arm of the late Prof. Justin Yerbury’s research team utilising cell and mouse models of MND to advance the investigation and assessment of therapeutic avenues.
The Australian Research Data Commons, March 2025
Dr Anne Hogden, Feb2024