Self-reported respiratory and gastrointestinal outcomes in children with isolated congenital diaphragmatic hernia: A prospective multicentre study.

Objective: To evaluate medium-term self-reported respiratory and gastrointestinal (GI) outcomes in children with congenital diaphragmatic hernia (CDH).

Design: Self-reported respiratory and GI outcomes correlated with prenatal severity indicators.

Setting: Prospective study at three fetal medicine units.

Setting the research agenda: involving parents in research on children who are HIV-free.

Introduction: There is growing interest in health, developmental and survival outcomes of children who are born HIV-free to women living with HIV (children born HIV-free). To date, the research agenda has been largely determined by researchers, funders and policy makers, with limited involvement of parents, who are key stakeholders. Researchers at UCL Great Ormond Street Institute of Child Health in partnership with community-based organisation 4M Network of Mentor Mothers conducted two workshops with parents in March 2022 to establish research priorities for children born HIV-free, and key considerations for methodological approaches both to research and engagement with the affected communities.

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8.

Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic diseases. Here, we report the bioengineering of a set of next-generation AAV vectors, named AAV-SYDs (where "SYD" stands for Sydney, Australia), with increased human hepato-tropism in a liver xenograft mouse model repopulated with primary human hepatocytes.

Child maltreatment and emergency department visits: a longitudinal birth cohort study from infancy to early adulthood.

Background: Child maltreatment (CM) is a serious global public health issue, with documented impacts on health.

Objective: To examine the association between different levels of CM concern, and Emergency Department (ED) visits from infancy to early adulthood.

Participants And Setting: Individuals born in Adelaide, South Australia from January 1986 to June 2017 (N = 443,754).

Identification of an Altered Matrix Signature in Kidney Aging and Disease.

Background: Accumulation of extracellular matrix in organs and tissues is a feature of both aging and disease. In the kidney, glomerulosclerosis and tubulointerstitial fibrosis accompany the decline in function, which current therapies cannot address, leading to organ failure. Although histologic and ultrastructural patterns of excess matrix form the basis of human disease classifications, a comprehensive molecular resolution of abnormal matrix is lacking.

Restoring the natural tropism of AAV2 vectors for human liver.

Recent clinical successes in gene therapy applications have intensified interest in using adeno-associated viruses (AAVs) as vectors for therapeutic gene delivery. Although prototypical AAV2 shows robust in vitro transduction of human hepatocyte-derived cell lines, it has not translated into an effective vector for liver-directed gene therapy in vivo. This is consistent with observations made in (FRG) mice with humanized livers, showing that AAV2 functions poorly in this xenograft model.

Hospitalizations among children involved in the child protection system: A long-term birth cohort study from infancy to adulthood using administrative data.

Background: Despite considerable health consequences of child abuse and neglect, there is limited evidence on hospitalizations in this population.

Objectives: To describe frequency and reasons for hospitalization by lifetime child protection system (CPS) involvement.

Participants: 608,540 children born from January 1, 1986 to June 30, 2017 in South Australia, Australia METHODS: Using linked administrative data on CPS involvement and hospitalizations, we descriptively examined cumulative incidence, cumulative count and reasons for hospitalization from infancy to early adulthood by CPS involvement.

Insufflation in minimally invasive surgery: Is there any advantage in staying low?

Aim: Minimally invasive repair of esophageal atresia with tracheoesophageal fistula (EA/TEF) and congenital diaphragmatic hernia (CDH) is feasible and confers benefits compared to thoracotomy or laparotomy. However, carbon dioxide (CO) insufflation can lead to hypercapnia and acidosis. We sought to determine the effect of lower insufflation pressures on patients' surrogate markers for CO absorption - arterial partial pressure of CO (PaCO), end tidal CO (EtCO) and pH.

Thoracoscopic aortopexy for symptomatic tracheobronchomalacia.

Aim: Symptomatic tracheobronchomalacia can be fatal. Successful treatment includes aortopexy. We report outcomes of the thoracoscopic approach in a single centre.

Vascular Instability and Neurological Morbidity in Sickle Cell Disease: An Integrative Framework.

It is well-established that patients with sickle cell disease (SCD) are at substantial risk of neurological complications, including overt and silent stroke, microstructural injury, and cognitive difficulties. Yet the underlying mechanisms remain poorly understood, partly because findings have largely been considered in isolation. Here, we review mechanistic pathways for which there is accumulating evidence and propose an integrative systems-biology framework for understanding neurological risk.

Using a Three-Dimensional Collagen Matrix to Deliver Respiratory Progenitor Cells to Decellularized Trachea In Vivo.

This article describes a method for engrafting epithelial progenitor cells to a revascularized scaffold in a protective and supportive collagen-rich environment. This method has the potential to overcome two key limitations of existing grafting techniques as epithelial cells are protected from mechanical shear and the relatively hypoxic phase that occurs while grafts revascularize, offering the opportunity to provide epithelial cells to decellularized allografts at the point of implantation. Advances in this area will improve the safety and efficacy of bioengineered organ transplantation.