• Paediatric Precision Medicine

Centralised capacity to develop functional genomics for Paediatric Precision Medicine – PPM 1

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 treatments paradigms for cancers and rare genetic diseases.

Lead Investigators

  • Ian_Alexander@2x

    Professor Ian Alexander

    Head, Gene Therapy Unit, Children’s Medical Research Institute (Australia)
  • Patrick_Tam@2x

    Professor Patrick Tam

    Head, Embryology Unit, Children’s Medical Research Institute (Australia)
  • Adam_Jaffe@2x

    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)

Team

Research Groups

Dr Anai Gonzalez Cordero
Children’s Medical Research Institute (Australia)

Professor Robyn Jamieson
Children’s Medical Research Institute (Australia)

Professor Ian Alexander
Children’s Medical Research Institute (Australia)

Professor Tracy Bryan
Children’s Medicial Research Institute (Australia)

Professor Adam Jaffe, Dr Shafagh Waters
School of Women’s and Children’s Health, University of NSW (Australia)

Professor Patrick Tam
Children’s Medical Research Institute (Australia)

Professor Roger Reddel
Children’s Medical Research Institute (Australia)

Precision Medicine Facilities

Stem Cell and Organoid Facility  

Dr Anai Gonzalez Cordero, Children’s Medical Research Institute (Australia)

 

Advanced Microscopy and the ACRF Telomere Analysis Centre (ATAC)

Dr Scott Page and Joshua Studdert, Children’s Medical Research Institute (Australia)

 

Single Cell Analytics Facility

Joshua Studdert, Children’s Medical Research Institute (Australia)

 

Vectorology and Genome Engineering

Dr Leszek Lisowski and Dr Predrag Kalajdzi, Children’s Medical Research Institute (Australia)
Rare Disease Functional Genomics Laboratory