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Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway modelsMutation-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.
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Psychometric properties of the Perth emotional labour scale: Preliminary support for a new measure with theoretical implicationsEmotional labour is the process by which people regulate emotions congruently with occupational requirements. Research consistently links emotional labour to greater levels of burnout. However, we argue this literature is potentially confounded by measurement error.
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Two-year post-treatment outcomes following peanut oral immunotherapy in the Probiotic and Peanut Oral Immunotherapy-003 Long-Term (PPOIT-003LT) studyFew studies have examined long-term outcomes following oral immunotherapy; none have examined long-term risks and benefits associated with distinct clinical outcomes (desensitization, remission).
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Study of pediatric appendicitis scores and management strategies: A prospective observational feasibility studyThe objective was to investigate the feasibility of prospectively validating multiple clinical prediction scores for pediatric appendicitis in an Australian pediatric emergency department.
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Improved Glycemic Outcomes With Diabetes Technology Use Independent of Socioeconomic Status in Youth With Type 1 DiabetesTechnology use in type 1 diabetes (T1D) is impacted by socioeconomic status (SES). This analysis explored relationships between SES, glycemic outcomes, and technology use.
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Strep A: challenges, opportunities, vaccine-based solutions and economicsStreptococcus pyogenes (Strep A) is a leading cause of morbidity and mortality across the globe, annually causing hundreds of millions of cases of disease.
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Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AMLHematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to relapse. To improve overall survival, we propose a combined strategy based on cord blood (CB)-HCT with the application of AML-specific T cell receptor (TCR)-engineered T cell therapy derived from the same CB graft.
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Stratification of Sepsis Patients on Admission into the Intensive Care Unit According to Differential Plasma Metabolic PhenotypesDelayed diagnosis of patients with sepsis or septic shock is associated with increased mortality and morbidity. UPLC-MS and NMR spectroscopy were used to measure panels of lipoproteins, lipids, biogenic amines, amino acids, and tryptophan pathway metabolites in blood plasma samples collected from 152 patients within 48 h of admission into the Intensive Care Unit (ICU) where 62 patients had no sepsis, 71 patients had sepsis, and 19 patients had septic shock.
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Mutational rescue of the activity of high-fidelity Cas9 enzymesProgrammable DNA endonucleases derived from bacterial genetic defense systems, exemplified by CRISPR-Cas9, have made it significantly easier to perform genomic modifications in living cells. However, unprogrammed, off-target modifications can have serious consequences, as they often disrupt the function or regulation of non-targeted genes and compromise the safety of therapeutic gene editing applications.
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Correlating Quantitative and Genomic SARS-CoV-2 Wastewater Data with Clinical Metrics in Metropolitan Perth, Western AustraliaWastewater-based epidemiology (WBE) has emerged as a key method for the continuous monitoring of COVID-19 prevalence including circulating SARS-CoV-2 lineages. WBE addresses the limitations of traditional clinical COVID-19 surveillance such as clinical test availability, fluctuating testing rates, and increased reliance on rapid antigen tests.