Search
Research
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway modelsMutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout to assess the comparative effectiveness of CFTR modulators and lentiviral vector-mediated gene therapy.
Research
Aboriginal and Torres Strait Islander Attitudes to Organ Donation in Central Australia: A Qualitative Pilot StudyOrgan transplantation is a well-established intervention but is reliant on the donation of organs and tissues, mostly from deceased donors. The proportion of Australians proceeding to organ donation (OD) has increased, but the proportion of Indigenous Australians proceeding remains two-thirds that of non-Indigenous Australians. We sought to explore perceived barriers and enablers for the involvement of Indigenous peoples in the OD process.
Research
Clinically Relevant Genes Identified in Cerebral Palsy Cohorts Following Evaluation of the Clinical Description and Phenotype: A Systematic ReviewA growing number of genes have been identified in individuals with cerebral palsy; however, many of these studies have poor compliance with the cerebral palsy clinical description. This systematic review aimed to assess the quality of the cerebral palsy clinical description/phenotype in cerebral palsy genetic studies published between 2010 and 2024 and report clinically relevant genes based on the quality of the cerebral palsy phenotype.
Research
Two-year post-treatment outcomes following peanut oral immunotherapy in the Probiotic and Peanut Oral Immunotherapy-003 Long-Term (PPOIT-003LT) studyFew studies have examined long-term outcomes following oral immunotherapy; none have examined long-term risks and benefits associated with distinct clinical outcomes (desensitization, remission).
News & Events
Kings Birthday Honour for Children's Diabetes Centre Co-directorCongratulations to the Rio Tinto Children's Diabetes Centre Co-Director and Perth Children's Hospital Director of Research, Professor Timothy Jones, awarded a Member of the Order of Australia (AM) in the King's Birthday Honours List.
News & Events
Celebrating International Day of Women and Girls in ScienceTo celebrate International Day of Women and Girls in Science, we asked some of the research team to share why science is the key to success and their tips for encouraging girls who want to pursue a career in STEM.
