This program utilised functional genomics to enhance children’s screening, diagnosis, and personalised clinical care. It introduced innovative treatments like gene and cell therapies for various conditions, such as inherited retinal diseases, mitochondrial disorders, telomere-related blood disorders, and cancers.
Furthermore, it strengthened stem cell capabilities in New South Wales, establishing a facility for stem cell production, organoid and cell-based disease modeling, and genetic modification. Additionally, it expanded genome editing capacity through the Vector and Genome Engineering Facility.
The program also supported the establishment of a unique laboratory in the southern hemisphere offering a precision medicine platform based on stem cell biology for children with cystic fibrosis. This platform predicted therapy responsiveness and assessed potential repurposing of existing treatments for patients with rare mutations.
- 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