Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan +7 more
wiley +1 more source
ABSTRACT Objective Neuromyelitis optica spectrum disorder (NMOSD) is a devastating neurological disease that lacks serological biomarkers that can accurately reflect disease activity. We established a live cell‐based assay (LCBA) using serum with endogenous complement to quantify the overall cytotoxicity, offering a novel functional tool for monitoring
Xiaona Xu +10 more
wiley +1 more source
Case Report: Bilateral visual loss due to endogenous endophthalmitis following total pancreatectomy with duodenectomy and splenectomy. [PDF]
Gao M +6 more
europepmc +1 more source
From Adult to Adolescent: Alignment in Clinical Trials and Outcomes in Axial Spondyloarthritis
Spondyloarthritis (SpA) is a group of chronic inflammatory diseases encompassing axial and peripheral forms, with up to 20% of patients developing symptoms before age 16. Despite this substantial pediatric burden, treatment options for juvenile‐onset SpA (JSpA), particularly those with axial disease (axJSpA), remain limited.
Pamela F. Weiss +9 more
wiley +1 more source
Treatment Outcomes of Excisional Versus Conservative Surgery for Endogenous Cesarean Scar Pregnancy: A Retrospective Cohort Study. [PDF]
Wang Z +9 more
europepmc +1 more source
Reversible Tuning of Multiple Molecular Kinking for Stem Cell Regulation In Vivo
In this work, we introduce the novel concept of background component‐free “multiple kinks” material platform composed solely of liganded multiple kink‐bearing molecules as a strategy for dynamic biomaterial design. Tri‐kink molecules enable effective near‐infrared light‐triggered ligand masking and visible light‐triggered ligand exposure, thereby ...
Kanghyeon Kim +28 more
wiley +1 more source
Retraction: Long Noncoding RNA MEG3 Suppresses Glioma Cell Proliferation, Migration, and Invasion by Acting as a Competing Endogenous RNA of miR-19a. [PDF]
Oncology Research Editorial Office.
europepmc +1 more source
Myogenic Fusogen‐Engineered Lipid Nanoparticles Enhance mRNA Delivery in Skeletal Muscle
This study reports a biomimetic strategy of engineering full‐length Myomaker, a muscle‐specific fusogen, into lipid nanoparticles (LNPs) to harness the native myoblast fusion capability for skeletal muscle mRNA delivery. The resulting Mymk‐LNPs enhance transfection in differentiating myocytes and enable Cre‐mediated reporter activation in injured ...
Fangyu Zhang +18 more
wiley +1 more source
Characterization of endogenous pararetroviruses in yam (Dioscorea spp.) genomes revealed four pararetrovirus groups, including a dioscovirus-like lineage: implications for diagnostics and yam germplasm exchange. [PDF]
Osundahunsi BO +4 more
europepmc +1 more source

