Gene Therapy Targeting Proteostasis for MND/ALS.

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There is a vital need to develop effective treatments for Amyotrophic Lateral Sclerosis (ALS), the most common Motor Neuron Disease (MND). Disrupted ubiquitin homeostasis - a key component of proteostasis - is a hallmark of ALS and restoring the capacity of the ubiquitin system in motor neurons is a promising therapeutic strategy in ALS. The proposed research will develop and validate novel viral and nanoparticle-based gene therapy targeting proteostasis in cell and animal models of ALS. Complementary work using cutting-edge ubiquitomics techniques and will be used to monitor biochemical response and understand disease mechanism.

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Supervisory team
Darren
Saunders

Medicine
Medical Sciences
Justin
Yerbury

Uni of Wollongong
External to UNSW
Kara
Perrow (nee Vine)

External to UNSW
external to UNSW
d.saunders@unsw.edu.au

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