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Randomised controlled trials in cystic fibrosis: What, when and how?

The major morbidity and mortality from cystic fibrosis (CF) comes from progressive lung disease with bronchiectasis leading to respiratory failure

Bile acids in the lower airways is associated with airway microbiota changes in chronic obstructive pulmonary disease: an observational study

Chronic obstructive pulmonary disease (COPD) is a complex disorder with a high degree of interindividual variability. Gastrointestinal dysfunction is common in patients with COPD and has been proposed to influence the clinical progression of the disease. Using the presence of bile acid(s) (BA) in bronchoalveolar lavage (BAL) fluid as a marker of gastric aspiration, we evaluated the relationships between BAs, clinical outcomes and bacterial lung colonisation.

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models

Mutation-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. 

Inhaled nebulised adrenaline delivery in children and adults: A simulation study

Sometimes, there is an urgent need to administer inhaled adrenaline to children, awake, sedated or anaesthetised to treat asthma, bronchospasm, croup, and suspected laryngeal/pharyngeal oedema or stridor, which can become severe or even life-threatening. To better inform emergency dosing and administration guidelines, we aimed to quantify the amount of adrenaline delivered for inhalation from a nebuliser, in a simulated experimental delivery set-up for spontaneously breathing children and adults, either via an anaesthetic face mask, a Laryngeal Mask Airway or an Endotracheal tube.

Conservation of gene expression patterns between the amniotic and nasal epithelium at birth

Amniotic epithelial cells are fetal-derived stem cells, capable of differentiating into all three germ layers, including mature epithelial cell populations. Here, we hypothesised that the amniotic epithelium might serve as a surrogate tissue source for investigating transcriptional profiles in the respiratory epithelium of newborns.

Personal network inference identifies children at risk of recurrent wheezing and asthma

Wheezing and asthma exacerbations are leading causes of pediatric hospital admissions. Predicting which children will experience persistent exacerbations remains challenging. Prior research has identified immune endotypes in the nasal epithelium of children with acute asthma and wheezing, characterized by varying balances of interferons and inflammatory markers. Notably, children exhibiting low interferon responses coupled with high inflammation are at an increased risk for recurrent respiratory exacerbations.

Regulating the Irregular: Phage Therapy and the Case for a Regulatory Sandbox Approach in Australia

Bacteriophages (phages) are viruses that selectively kill bacteria and offer a promising option to address the growing global pandemic of antimicrobial-resistant infections. However, phage therapy does not easily align with traditional regulatory pathways designed for fixed-composition chemical drugs or biologics with fixed non-evolving compositions.

Advancing bioinformatics capacity through Nextflow and nf-core: lessons from an early-to mid-career researchers-focused program at The Kids Research Institute Australia

he increasing adoption of high-throughput "omics" technologies has heightened the demand for standardized, scalable, and reproducible bioinformatics workflows. Nextflow and nf-core provide a robust framework for researchers, particularly early- and mid-career researchers (EMCRs), to navigate complex data analysis.

Transcriptomic analysis of primary nasal epithelial cells reveals altered interferon signalling in preterm birth survivors at one year of age

Many survivors of preterm birth (<37 weeks gestation) have lifelong respiratory deficits, the drivers of which remain unknown. Influencers of pathophysiological outcomes are often detectable at the gene level and pinpointing these differences can help guide targeted research and interventions. This study provides the first transcriptomic analysis of primary nasal airway epithelial cells in survivors of preterm birth at approximately 1 year of age.

Phage therapy to treat cystic fibrosis Burkholderia cepacia complex lung infections: perspectives and challengesge

Burkholderia cepacia complex is a cause of serious lung infections in people with cystic fibrosis, exhibiting extremely high levels of antimicrobial resistance. These infections are difficult to treat and are associated with high morbidity and mortality.