University of Wollongong (UOW) scientists have developed a breakthrough therapy that clears toxic proteins from nerve cells—a discovery that advances the work of the late Professor Justin Yerbury and could transform the treatment of motor neuron disease (MND).
The proof-of-concept study, published in Nature Communications and led by Dr. Christen Chisholm from UOW’s Molecular Horizons, unveils a therapeutic designer molecule, MisfoldUbL, that targets and removes toxic misfolded SOD1 (superoxide dismutase 1) proteins from cells. SOD1 is an antioxidant enzyme that plays a crucial role in protecting cells from damage caused by superoxide radicals. About 35% of people with inherited MND in Australia have SOD1 gene mutations that cause more frequent misfolding.
“In MND, proteins misfold more frequently and the cell’s degradation systems become overwhelmed and stop working properly. The misfolded protein can then accumulate, forming clumps or ‘aggregates’ and over time, this accumulation damages and eventually kills motor neurons, leading to gradual muscle weakness, paralysis and death,” Dr. Chisholm said.









