Results 221 to 230 of about 993,526 (301)

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
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

Characteristics and Outcomes of Male Participants in a Multicenter Longitudinal Australian Study Cohort

open access: yesArthritis Care &Research, EarlyView.
Objective The aim of this study was to determine the differences in demographic, serologic, and clinical characteristics between male and female patients with systemic sclerosis (SSc) in an Australian cohort. Methods This was a retrospective observational study using data from the Australian Scleroderma Cohort Study.
Emily Lin   +14 more
wiley   +1 more source

From Bioinformatic Modeling to Clinical Observation: Potential Implications of Ribosomal RNA Folding in <i>Blastocystis</i> sp. Isolates from Symptomatic and Asymptomatic Carriers. [PDF]

open access: yesPathogens
Martínez-Hernández F   +8 more
europepmc   +1 more source

Association of Elevated Platelets and C‐Reactive Protein With Severe Disease and Poor Survival in Systemic Sclerosis

open access: yesArthritis Care &Research, EarlyView.
Objective Elevated C‐reactive protein (CRP) levels in systemic sclerosis (SSc) have been linked with severe disease and worse survival, but the role of platelet levels remains unclear. This study examined whether elevated platelet levels, CRP levels, or both are associated with disease severity, progression, and survival in SSc.
Brian S. Lee   +4 more
wiley   +1 more source

Fusogenic RNA Nanomodules for Fusion‐Mediated and Multiplexed siRNA Delivery

open access: yesAdvanced Functional Materials, EarlyView.
A fusogenic lipid‐layered RNA nanomodules (L‐CRAMs) enable high‐capacity and long‐lasting siRNA delivery through membrane fusion. These nanomodules carry exceptionally large siRNA payloads, avoid conventional endosomal uptake, and release multiple functional siRNAs through Dicer‐mediated processing.
Sunghyun Moon   +5 more
wiley   +1 more source

Multifunctional Neutrophil Nanovesicles Induce Remodeling of Tumor Immune Microenvironment and Synergistic Multimodal Tumor Therapy

open access: yesAdvanced Functional Materials, EarlyView.
Schematic illustration of the engineered RN@FDT for multimodal tumor therapy. a) Fabrication of FD&DOX/TFA‐loaded RGD‐NNV (RN@FDT) and the light‐triggered disassemble of RN@FDT. b) After internalized by tumor cells, RN@FDT are disintegrated under NIR‐II laser irradiation to boost PTT and induce cell apoptosis.
Jiahui Zhang   +16 more
wiley   +1 more source

Myogenic Fusogen‐Engineered Lipid Nanoparticles Enhance mRNA Delivery in Skeletal Muscle

open access: yesAdvanced Functional Materials, EarlyView.
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

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