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Research

Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient's genetic mutation, in particular those lesions that compromise normal pre-mRNA processing. Antisense oligonucleotides can alter gene expression through a variety of mechanisms as determined by the chemistry and antisense oligomer design.

Research

The identification of a COPD etiotype associated with preterm birth (COPD-developmental) has expedited calls for intervention strategies that may improve health outcomes for survivors of preterm birth (<37 weeks' gestation). Pulmonary-rehabilitation style training interventions achieve physiological and symptom improvement in older people with COPD, but whether similar training interventions are suitable for young people is unclear. We sought to understand the perceived need and requirements of an exercise training intervention for children, adolescents and adults born preterm.

Research

Jane Pillow BMedSci (Dist) MBBS, PhD (Dist) FRACP Research Theme Head, Early Environment; Team Lead, Chronobiology jane.pillow@thekids.org.au

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Nasal epithelial cells from young adults with a history of very preterm birth show delayed closure following scratch-wounding. Repair correlated with lung function, suggesting epithelial barrier integrity may play a role in preterm-associated lung disease.

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Septo-optic dysplasia (SOD) is a major cause of congenital hypopituitarism and is known to be associated with overweight and obesity in up to 44% of children. Given the role of the hypothalamus in hormonal regulation, we sought to assess the association of resting energy expenditure (REE), appetite and physical activity with SOD. 

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Worldwide, tuberculosis (TB) remains the leading cause of death from infectious diseases. Africa is the second most-affected region, accounting for a quarter of the global TB burden, but there is limited evidence whether there is subnational variation of TB prevalence across the continent. Therefore, this study aimed to estimate sub-national and local TB prevalence across Africa.

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Parents of children and adolescents with chronic conditions have an increased risk of stress-related mental health problems, and reduced quality of life. Third wave Cognitive Behavioral Therapy interventions have been shown to reduce stress in this parent population. Studies demonstrate that this efficacy endures when these therapies are delivered online.

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One third of Australian children diagnosed with type 1 diabetes present with life-threatening diabetic ketoacidosis (DKA) at diagnosis. Screening for early-stage, presymptomatic type 1 diabetes, with ongoing follow-up, can substantially reduce this risk (<5% risk). Several screening models are being trialled internationally, without consensus on the optimal approach. This pilot study aims to assess three models for a routine, population-wide screening programme in Australia.

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nfants with frequent viral and bacterial respiratory infections exhibit compromised immunity to routine immunizations. They are also more likely to develop chronic respiratory diseases in later childhood. This study investigated the feasibility of epigenetic profiling to reveal endotype-specific molecular pathways with potential for early identification and immuno-modulation. 

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This study identified alignment of indicators across different initiatives and data collection instruments as a foundation for future harmonization of adolescent health measurement.