Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice.

Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current approved treatments seek to restore IDUA levels via enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT). The effectiveness of these treatment strategies in preventing neurodegeneration is limited due to the inability of ERT to penetrate the blood-brain barrier (BBB) and HSCT's limited CNS reconstitution of IDUA levels.

Novel risk predictor of arrhythmias for patients with potassium channel-related congenital long QT syndrome.

Background: Congenital long QT syndrome (LQTS) is characterized by delayed ventricular repolarization, predisposing to potentially lethal ventricular arrhythmias. The variability in disease severity among patients remains largely unexplored, underscoring the limitations of current risk stratification methods.

Objective: We aimed to evaluate the potential utility of electrocardiographic markers from the exercise stress test (EST) in identifying patients with high-risk LQTS.

A Systematic Review and Meta-Analysis of Oncologic Liver Resections in Low- and Middle-Income Countries: Opportunities to Improve Evidence and Outcomes.

Background: Patients in low- and middle-income countries (LMICs) are disproportionately affected by liver cancers but there is a lack of understanding of their postoperative outcomes. This study aimed to review the current status of research in LMICs regarding outcomes after oncologic hepatectomy and synthesize the data reported in the literature.

Methods: The PubMed, Scopus, Embase, Web of Science, and World Health Organization (WHO) Global Index Medicus databases were searched from database inception to May 26th, 2022.