Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models.

Children with neurodegenerative disease often have debilitating gastrointestinal symptoms. We hypothesized that this may be due at least in part to underappreciated degeneration of neurons in the enteric nervous system (ENS), the master regulator of bowel function. To test this hypothesis, we evaluated mouse models of neuronal ceroid lipofuscinosis type 1 and 2 (CLN1 and CLN2 disease, respectively), neurodegenerative lysosomal storage disorders caused by deficiencies in palmitoyl protein thioesterase-1 and tripeptidyl peptidase-1, respectively.

Evaluating the feasibility, sensitivity, and specificity of next-generation molecular methods for pleural infection diagnosis.

Pleural infections are common and associated with substantial healthcare costs, morbidity, and mortality. Accurate diagnosis remains challenging due to low culture positivity rates, frequent polymicrobial involvement, and non-specific diagnostic biomarkers. Here, we undertook a prospective study examining the feasibility and performance of molecular methods for diagnosing suspected pleural infection.

General Endotracheal Anesthesia vs Total Intravenous Anesthesia for Children Undergoing Low-Risk Cardiac Catheterization.

Historically, children undergoing cardiac catheterization have been managed with general endotracheal anesthesia (GETA). However, recent practice has favored general anesthesia with total intravenous anesthesia (TIVA). This study compares procedural outcomes in the pediatric cardiac catheterization laboratory (PCCL) for children undergoing low-risk cardiac catheterization with GETA vs.

Dereplication of Natural Product Antifungals via Liquid Chromatography-Tandem Mass Spectrometry and Chemical Genomics.

Recently expanded reports of multidrug-resistant fungal infections underscore the need to develop new and more efficient methods for antifungal drug discovery. A ubiquitous problem in natural product drug discovery campaigns is the rediscovery of known compounds or their relatives; accordingly, we have integrated Liquid Chromatography-Tandem Mass Spectrometry (LC-MS/MS) for structural dereplication and Yeast Chemical Genomics for bioprocess evaluation into a screening platform to identify such compounds early in the screening process. We identified 450 fractions inhibiting and the resistant strains of and among more than 40,000 natural product fractions.

The Influence of Project ECHO and Integrated Behavioral Health in Primary Care on Emergency Department Visits Among Youth Diagnosed with Depression.

Rates of depression among youth and emergency department (ED) visits for un- or under-treated symptoms are on the rise. Early identification and treatment of depression is imperative at the patient, program, system, and population levels. This paper examines the individual and cumulative impact of Project ECHO and the inclusion of IBH services in pediatric primary care practices on mental health-related ED rates among youth diagnosed with depression for those practices.