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Research
Hyperactive Nickase Activity Improves Adenine Base EditingBase 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.
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A pilot randomised controlled trial of a telehealth-delivered brief ‘Sleeping Sound Autism’ intervention for autistic childrenAccess 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.
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Patient-derived xenografts and single-cell sequencing identifies three subtypes of tumor-reactive lymphocytes in uveal melanoma metastasesUveal 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.
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Rationale for use for monoclonal antibody and anti-cytokine therapy for multisystem inflammatory syndrome in children: A systematic reviewObjective: To review systematically the rationale for choice and use of monoclonal antibody and anti-cytokine therapy in multisystem inflammatory syndrome in children.
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Perturbation of the T Cell Compartment Underlies Rheumatic Fever PathogenesisThis prospective cohort study, which recruited participants with definite ARF in Australia and Aotearoa New Zealand, profiled circulating immune molecules and cells to inform disease mechanisms and future druggable pathways.
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Parents’ Disclosure of Their Child’s Health and Neurodevelopmental Conditions: A Systematic Review and Qualitative MetasynthesisParents of children with physical/mental health and/or neurodevelopmental conditions often need to make disclosure decisions for their child. Disclosure can bring benefits (e.g., support) but can also risk harm (e.g., stigma). This systematic review aimed to consolidate research regarding parents' disclosure experiences to better understand how to support parents during this process.
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The parent empowerment scale: development and psychometric propertiesParents of trans and gender diverse (herein ‘trans’) children experience additional challenges in their parenting role relative to parents of cisgender children. Understanding and enhancing parents’ empowerment is a promising approach to support both parents and children. We aimed to develop an empowerment scale specific to parents of trans children, grounded in parents’ lived experiences.
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Glycemic and Psychosocial Outcomes of Advanced Hybrid Closed-Loop Therapy in Youth With High HbA1c: A Randomized Clinical TrialTo determine the efficacy of advanced hybrid closed-loop therapy in a high-risk cohort of youth on continuous subcutaneous insulin infusion with or without continuous glucose monitoring with suboptimal glycemia.
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Influences of bilingual input on English vocabulary size and academic outcomes: a large-scale longitudinal study following children in Australia from five to ten yearsResearch from large population-based studies investigating the language and academic outcomes for bilingual children is rare. The current study aimed to investigate the influence of dual language exposure on (i) English vocabulary outcomes at 5 years (126 bilinguals, 1675 monolinguals), and 10 years (vocabulary: 92 bilinguals, 1413 monolinguals:), and (ii) academic outcomes at 10 years (107 bilinguals, 1746 monolinguals).
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Which emerging autism features at 12 months of age are associated with later parent-child interaction?Parent-child interactions (PCI) in infants with an elevated likelihood (EL) of autism start to diverge from other infants toward the end of the first year. This divergence is often attributed to emerging features of autism impacting infant social interactions in ways that become increasingly amplified. The aim was to identify which, if any, 12-month autism features were associated with later PCI qualities.