Outcomes After Thoracoscopic and Open Repair of Esophageal Atresia With Tracheoesophageal Fistula at US Children's Hospitals.

Background: Thoracoscopic repair of esophageal atresia with tracheoesophageal fistula (EA/TEF) has been reported to have superior outcomes to the open approach. We sought to evaluate adoption of thoracoscopic repair at US children's hospitals and compare outcomes to open repair.

Methods: The Pediatric Health Information System database was used to identify patients with EA/TEF who underwent repair between 2016 and 2023.

A phase I clinical trial of intrahepatic artery delivery of TG6002 in combination with oral 5-fluorocytosine in patients with liver-dominant metastatic colorectal cancer.

Background: Effective treatment for patients with metastatic cancer is limited, particularly for colorectal cancer patients with metastatic liver lesions (mCRC), where accessibility to numerous tumours is essential for favourable clinical outcomes. Oncolytic viruses (OVs) selectively replicate in cancer cells; however, direct targeting of inaccessible lesions is limited when using conventional intravenous or intratumoural administration routes.

Methods: We conducted a multi-centre, dose-escalation, phase I study of vaccinia virus, TG6002, via intrahepatic artery (IHA) delivery in combination with the oral pro-drug 5-fluorocytosine to fifteen mCRC patients.

Lifestyle factors and other predictors of common mental disorders in diagnostic machine learning studies: A systematic review.

Background: Machine Learning (ML) models have been used to predict common mental disorders (CMDs) and may provide insights into the key modifiable factors that can identify and predict CMD risk and be targeted through interventions. This systematic review aimed to synthesise evidence from ML studies predicting CMDs, evaluate their performance, and establish the potential benefit of incorporating lifestyle data in ML models alongside biological and/or demographic-environmental factors.

Methods: This systematic review adheres to the PRISMA statement (Prospero CRD42023401194).

Ion mobility mass spectrometry unveils conformational effects of drug lead EPI-001 on the intrinsically disordered N-terminal domain of the androgen receptor.

Intrinsically disordered proteins (IDPs) are important drug targets as they are key actors within cell signaling networks. However, the conformational plasticity of IDPs renders them challenging to characterize, which is a bottleneck in developing small molecule drugs that bind to IDPs and modulate their behavior. In relation to this, ion mobility mass spectrometry (IM-MS) is a useful tool to investigate IDPs, as it can reveal their conformational preferences.

Mechanisms driving epigenetic and transcriptional responses of microglia in a neurodegenerative lysosomal storage disorder model.

Lysosomal dysfunction is causally linked to neurodegeneration in many lysosomal storage disorders (LSDs) and is associated with various age-related neurodegenerative diseases , but there is limited understanding of the mechanisms by which altered lysosomal function leads to changes in gene expression that drive pathogenic cellular phenotypes. To investigate this question, we performed systematic imaging, transcriptomic, and epigenetic studies of major brain cell types in null (KO) mice, a preclinical mouse model for Sanfilippo syndrome (Mucopolysaccharidosis Type IIIA, MPS-IIIA) . MPS-IIIA is a neurodegenerative LSD caused by homozygous loss-of-function (LoF) mutations in which results in severe early-onset developmental, behavioral, and neurocognitive impairment .