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Research

Hyperactive Nickase Activity Improves Adenine Base Editing

Base editing technologies enable programmable single-nucleotide changes in target DNA without double-stranded DNA breaks. Adenine base editors (ABEs) allow precise conversion of adenine to guanine. However, limited availability of optimized deaminases as well as their variable efficiencies across different target sequences can limit the ability of ABEs to achieve effective adenine editing.

Research

Investigating the Feasibility and Acceptability of a Facebook Delivered, Parent Mediated, Physical Activity Intervention for Children with Developmental Coordination Disorder

Children with Developmental Coordination Disorder (DCD) experience difficulties performing fundamental movement skills, resulting in reduced physical activity (PA). Given low PA can impact mental and physical health, improving PA in DCD appears imperative. This study investigates the feasibility of a Facebook delivered, parent mediated, PA intervention for children with DCD.

Research

A pilot randomised controlled trial of a telehealth-delivered brief ‘Sleeping Sound Autism’ intervention for autistic children

Access to behavioural sleep intervention is beneficial for autistic children, yet many families face barriers to access associated with location and time. Preliminary evidence supports telehealth-delivered sleep intervention. However, no studies have evaluated brief telehealth sleep intervention.

Research

Patient-derived xenografts and single-cell sequencing identifies three subtypes of tumor-reactive lymphocytes in uveal melanoma metastases

Uveal melanoma is a rare melanoma originating in the eye's uvea, with 50% of patients experiencing metastasis predominantly in the liver. In contrast to cutaneous melanoma, there is only a limited effectiveness of combined immune checkpoint therapies, and half of patients with uveal melanoma metastases succumb to disease within 2 years.

Research

Rationale for use for monoclonal antibody and anti-cytokine therapy for multisystem inflammatory syndrome in children: A systematic review

Objective: To review systematically the rationale for choice and use of monoclonal antibody and anti-cytokine therapy in multisystem inflammatory syndrome in children.

Research

An Anti-VEGF-B Antibody Reduces Abnormal Tumor Vasculature and Enhances the Effects of Chemotherapy

The vascular endothelial growth factors and their receptors are key regulators of blood vessel formation, including in tumors, where their deregulated function can promote the production of aberrant, leaky blood vessels, supporting tumor development. 

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High-flow nasal oxygen for children's airway surgery to reduce hypoxaemic events: a randomised controlled trial

Tubeless upper airway surgery in children is a complex procedure in which surgeons and anaesthetists share the same operating field. These procedures are often interrupted for rescue oxygen therapy. 

Research

Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial

Bronchiectasis is a worldwide chronic lung disorder where exacerbations are common. It affects people of all ages, but especially Indigenous populations in high-income nations. Despite being a major contributor to chronic lung disease, there are no licensed therapies for bronchiectasis and there remain relatively few randomised controlled trials (RCTs) conducted in children and adults.

Research

Patient preferences for prophylactic regimens requiring regular injections in children and adolescents: A systematic review and thematic analysis

At present, limited literature exists exploring patient preferences for prophylactic treatment of acute rheumatic fever and rheumatic heart disease. Given low treatment completion rates to this treatment in Australia, where the burden of disease predominantly affects Aboriginal and Torres Strait Islander people, an improved understanding of factors driving patient preference is required to improve outcomes.