Lowe syndrome (LS) is a very rare disorder of phosphatidylinositol metabolism, which manifests with a complex phenotype comprising a clinical triad encompassing major abnormalities of the eyes, the kidneys, and the central nervous system. We are reporting a 23-year-old Egyptian male with a severe phenotype of LS with a minimal kidney disease. Direct sequencing of the OCRL gene detected a p.His375Arg mutation in the catalytic domain of the protein. The patient suffered from bilateral congenital cataracts and glaucoma, striking growth deficiency, severe psychomotor disability, a severe osteopathy, and seizures, but only minimal renal dysfunction. Although the biological mechanisms underlying the pathophysiology of LS manifestations is yet unclear, it has been proposed that growth delay and osteopathy are linked to a renal dysfunction. This report, however, argues this association and suggests that kidney dysfunction may partially explain the growth deficiency and bone abnormalities, but other still undefined factors might have a potential impact.
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http://dx.doi.org/10.1002/ajmg.a.38572 | DOI Listing |
Dev Psychopathol
January 2025
Department of Psychology, University of Wisconsin-Madison, Madison, WI, USA.
Polygenic scores (PGSs) have garnered increasing attention in the clinical sciences due to their robust prediction signals for psychopathology, including externalizing (EXT) behaviors. However, studies leveraging PGSs have rarely accounted for the phenotypic and developmental heterogeneity in EXT outcomes. We used the National Longitudinal Study of Adolescent to Adult Health (analytic = 4,416), spanning ages 13 to 41, to examine associations between EXT PGSs and trajectories of antisocial behaviors (ASB) and substance use behaviors (SUB) identified via growth mixture modeling.
View Article and Find Full Text PDFResearch (Wash D C)
January 2025
Division of Biotechnology, Dalian Institute of Chemical Physics, Chinese Academy of Sciences, Dalian 116023, China.
Sepsis-associated encephalopathy (SAE) is a severe and frequent septic complication, characterized by neuronal damage as key pathological features. The astrocyte-microglia crosstalk in the central nervous system (CNS) plays important roles in various neurological diseases. However, how astrocytes interact with microglia to regulate neuronal injury in SAE is poorly defined.
View Article and Find Full Text PDFAlzheimers Dement (Amst)
January 2025
Weill Institute for Neurosciences, Department of Neurology, Memory and Aging Center University of California, San Francisco San Francisco California USA.
Introduction: Plasma amyloid beta/amyloid beta (Aβ/Aβ) and phosphorylated tau217 (p-tau217) identify individuals with primary Alzheimer's disease (AD). They may detect AD co-pathology in the setting of other primary neurodegenerative diseases, but this has not been systematically studied.
Methods: We compared the clinical, neuroimaging, and neuropathological associations of plasma Aβ/Aβ (mass spectrometry), p-tau217 (electrochemiluminescence), and neurofilament light ([NfL], single molecule array [Simoa]), as markers of AD co-pathology, in a sporadic frontotemporal dementia (FTD) cohort ( = 620).
J Transl Med
January 2025
Department of Urology, First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi Province, China.
Background: The progression of bladder cancer (BC) from non-muscle-invasive bladder cancer (NMIBC) to muscle-invasive bladder cancer (MIBC) significantly increases disease severity. Although the tumor microenvironment (TME) plays a pivotal role in this process, the heterogeneity of tumor cells and TME components remains underexplored.
Methods: We characterized the transcriptomes of single cells from 11 BC samples, including 4 NMIBC, 4 MIBC, and 3 adjacent normal tissues.
J Transl Med
January 2025
Department of Endocrinology, Diabetology and Metabolism, Lausanne University Hospital, Avenue de la Sallaz 8, CH-1011, Lausanne, Switzerland.
Background: Obesity is associated with varying degrees of metabolic dysfunction. In this study, we aimed to discover markers of the severity of metabolic impairment in men with obesity via a multiomics approach.
Methods: Thirty-two morbidly men with obesity who were candidates for Roux-en-Y gastric bypass (RYGB) surgery were prospectively followed.
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