Search
Research
Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse ModelSeverity and disease progression in people with Cystic Fibrosis is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides. CFTR exon 9 is an in-frame exon and hence the exclusion of this exon would excise only 31 amino acids but not alter the reading frame of the remaining mRNA.
Research
Predicting disease progression in cystic fibrosisComprehensive scores incorporating clinical, lung function, imaging and laboratory data will become essential in the future for predicting disease progression and for use in clinical trials
Research
Prevalence of tracheobronchomalacia is higher than previously reported in children with cystic fibrosisTracheobronchomalacia (TBM) is estimated to be present in 1 in 2100 children. Previous reports suggest the prevalence is higher in children with cystic fibrosis (CF). This has clinical implications with potential to influence airway clearance and lung health.
Research
Airway macrophages display decreased expression of receptors mediating and regulating scavenging in early cystic fibrosis lung diseaseCystic fibrosis (CF) airway disease is characterized by chronic inflammation, featuring neutrophil influx to the lumen. Airway macrophages (AMs) can promote both inflammation and resolution, and are thus critical to maintaining and restoring homeostasis. CF AM functions, specifically scavenging activity and resolution of inflammation, have been shown to be impaired, yet underlying processes remain unknown.
We’ve heard from families that trustworthy information about preterm-associated lung disease is difficult to find. In response, we’ve created resources to empower families with the knowledge they need to manage these challenges.
Research
Pseudomonas aeruginosa Resistance to Bacteriophages and Its Prevention by Strategic Therapeutic Cocktail FormulationAntimicrobial resistance poses a significant threat to modern healthcare as it limits treatment options for bacterial infections, particularly impacting those with chronic conditions such as cystic fibrosis (CF). Viscous mucus accumulation in the lungs of individuals genetically predisposed to CF leads to recurrent bacterial infections, necessitating prolonged antimicrobial chemotherapy. Pseudomonas aeruginosa infections are the predominant driver of CF lung disease, and airway isolates are frequently resistant to multiple antimicrobials.
Research
Assessment of p.Phe508del-CFTR functional restoration in pediatric primary cystic fibrosis airway epithelial cellsThis study assessed an alternative approach, using a small scale halide assay that can be adapted for a personalized high throughput setting to analyze CFTR function of pAEC.
Research
Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimise new therapeutic interventions.
News & Events
$3.4mill to improve treatment management of cystic fibrosisThe Kids researchers have been awarded over $3.4 million for a new trial to pioneer improved ways for managing cystic fibrosis (‘CF’).
News & Events
Born with cystic fibrosis, now research is her life’s workWhen Ingrid Laing was born, the outlook for kids with cystic fibrosis was bleak. Her parents were told she might make it to 20 if she was lucky.