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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.
Research
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.
Research
Understanding variations in the built environment over time to inform longitudinal studies of young children's physical activity behaviour - The BEACHES projectWe know relatively little about the role the neighbourhood built environment plays in promoting young children's physical activity, particularly its longitudinal effect either through repeated exposure to the same environment or through change in exposure by moving from one neighbourhood to another.
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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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Insights into the Clinical, Biological and Therapeutic Impact of Copy Number Alteration in CancerCopy number alterations (CNAs), resulting from the gain or loss of genetic material from as little as 50 base pairs or as big as entire chromosome(s), have been associated with many congenital diseases, de novo syndromes and cancer. It is established that CNAs disturb the dosage of genomic regions including enhancers/promoters, long non-coding RNA and gene(s) among others, ultimately leading to an altered balance of key cellular functions.
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Towards Harmonized Adolescent Health Measurement: Assessing Alignment Between Current Recommendations and the Global Action for Measurement of Adolescent Health–Recommended IndicatorsThis study identified alignment of indicators across different initiatives and data collection instruments as a foundation for future harmonization of adolescent health measurement.
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Serological Responses to Streptococcus pyogenes Vaccine Candidate Antigens Suggests That Streptococcus dysgalactiae Is the Predominant Cause of Lower Limb CellulitisA future Streptococcus pyogenes (Strep A) vaccine will ideally prevent a significant burden of lower limb cellulitis; however, natural immune responses to proposed vaccine antigens following an episode of cellulitis remain uncharacterized.
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Outcomes of rheumatic fever in Uganda: a prospective cohort studyRheumatic heart disease is the largest contributor to cardiac-related mortality in children worldwide. Outcomes in endemic settings after its antecedent illness, acute rheumatic fever, are not well understood. We aimed to describe 3-5 year mortality, acute rheumatic fever recurrence, changes in carditis, and correlates of mortality after acute rheumatic fever.
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Impaired interferon response in plasmacytoid dendritic cells from children with persistent wheezeImpaired interferon response and allergic sensitization may contribute to virus-induced wheeze and asthma development in young children. Plasmacytoid dendritic cells play a key role in antiviral immunity as critical producers of type I interferons.