Functional Genomics

Project Overview

This initiative will see the establishment of a translational stem cell biology team and enhancements to existing infrastructure to support functional genomics capability. This will allow for the capacity for stem cell production, organoid and cell-based disease modelling and genetic modifications for applied genomics. It will mark a significant future investment for New South Wales in clinical management and research discovery in cancers and rare genetic diseases. The initial treatment focus will be on metabolic liver disease, blinding eye disease, mitochondrial disorders, telomere and cancer disorders and cystic fibrosis.

Collaborations

Kids Research Institute (Australia)
City of Hope Center for Gene Technology (USA)
Sydney Children’s Hospitals Network (Australia)
University of Adelaide (Australia)
Children’s Medical Research Institute (Australia)
University of Lisbon (Portugal)
Children’s Cancer Institute (Australia)
University Medical Center Utrecht (Netherlands)
Australian Functional Genomics Network (https://www.functionalgenomics.org.au/)
Vertex Pharmaceutical
Royal Price Alfred Hospital (Australia)
Loxegen Pharmaceutical
University of Technology Sydney (Australia)
Biospecialist Ltd
University of Sydney (Australia)
CF Australia

Summary

The activities of the translational stem cell biology team and the core facilities for functional genomic research will provide transformative capacity in disease modelling. It will also facilitate preclinical investigations of novel treatment paradigms for cancers and rare genetic diseases.

Lead Investigators

Professor Ian Alexander
Head, Gene Therapy Unit, Children’s Medical Research Institute (Australia)
Professor Patrick Tam
Head, Embryology Unit, Children’s Medical Research Institute (Australia)
Professor Adam Jaffe
Head, School of Women’s and Children’s Health, University of New South Wales Sydney (Australia)
Dr Shafagh Waters
Lead Scientist, Australasian Centre for Personalised Cystic Fibrosis Medicine (ACPCFM), School of Women’s and Children’s Health, University of New South Wales Sydney (Australia)