New Frontiers in Childhood Dementia Research: From Gene Therapy to Biomarkers

This webinar presents interim findings from 4 research projects funded through the world's first competitive research funding specifically directed to childhood dementia.

In March 2023, Australia's Federal Minister for Health, Mark Butler, announced the recipients of funding from the Medical Research Future Fund (MRFF) for childhood dementia. Now, two years later, we will hear about the progress of this research, which spans three different approaches to gene therapy and one project to identify biomarkers. 

With half of children affected by childhood dementia not surviving beyond age ten due to a lack of effective treatments, more research is urgently needed. These projects represent a critical step towards improving the outlook for children with dementia. The researchers will present an update on the research and there will be opportunities to ask questions and discuss these exciting projects.

Although the presentations will contain some technical information, every effort will be made to make the webinar understandable to non-scientists, including families of children with dementia. A simple overview of the science will be given at the beginning of the webinar.

Speakers:

• Developing a comprehensive CSF and plasma biomarker panel for childhood dementia therapies
  Professor Michelle Farrar, Sydney Children's Hospital Network and UNSW
• Developing an mRNA-based gene therapy strategy for Niemann-Pick Disease Type C1: a blueprint to treat childhood dementia
  Dr Ya Hui Hung, The Florey, Melbourne
• Developing Nanoparticle Mediated Gene Transfer for Childhood Dementia
  Dr Nicholas Smith, University of Adelaide and the Women's and Children's Hospital and Dr Adeline Lau, Flinders Health and Medical Research Institute (FHMRI)
• A new substrate reduction strategy to treat multiple childhood dementias: Glucosylceramide synthase-targeting antisense oligonucleotides
  Associate Professor Anthony Cook, Wicking Dementia Research & Education Centre at the University of Tasmania.

To accommodate those who are based overseas, this webinar will be recorded and distributed.