What is the issue for NSW?
Frontotemporal Dementia and Motor Neurone Disease, both devastating neurodegenerative disorders, stand as formidable challenges in modern healthcare. These conditions ruin the quality of life for those affected and they also burden the families and healthcare systems. The World Alzheimer Report 2022 estimates that over 46.8 million people worldwide and 400,000 Australians are currently living with dementia. This number is expected to rise to over 800,000 by 2058 due to an aging population. Estimates suggest that Frontotemporal Dementia accounts for approximately 10-20% of early-onset dementias, affecting individuals predominantly between the ages of 40 and 65. Although less common, Motor Neurone Disease still impacts tens of thousands of people globally. Recent statistics estimate there are over 2,000 people in Australia currently diagnosed with Motor Neurone Disease and every day two2 Australians are diagnosed.
What does the research aim to do and how?
This research will accelerate a new experimental gene therapy towards clinical trials for patients with Frontotemporal Dementia and Motor Neuron Disease by tackling current barriers in the use of gene therapy. This research will improve an experimental gene therapy for these diseases. It will increase its cell-type specificity and transduction efficiency. It will also reduce unwanted immune responses in patients. The project will use the latest disease models and patient-derived systems. The project aims to improve a novel gene therapy’s safety and efficacy for rapid patient use.