Results 71 to 80 of about 2,687,044 (278)

Intranasal Delivery of Gallium–Quercetin Nanoparticles for Multi‐Target Ferroptosis Inhibition in Parkinson's Disease

open access: yesAdvanced Science, EarlyView.
Leveraging the chemical similarity between Ga3+ and Fe3+ as well as the antioxidant properties of quercetin, gallium–quercetin nanoparticles (GQNPs) were prepared to integrate iron homeostasis regulation, oxidative stress suppression, and mitochondrial protection for multi‐target ferroptosis inhibition in Parkinson's Disease.
Keyang Xu   +12 more
wiley   +1 more source

RNA Promotes Synapsin Condensates That Organize Synaptic Vesicles in Live Neurons and Enable Localized Translation in Reconstituted System

open access: yesAdvanced Science, EarlyView.
Condensates at synapses organize synaptic vesicles (SVs) and are crucial for efficient neurotransmitter release, yet how RNA contributes to this mesoscale architecture remains poorly understood. Here we uncover that RNA modulates synapsin–driven condensates.
Branislava Rankovic   +21 more
wiley   +1 more source

Mitochondrial stress markers associate with phenotypic variability in Fabry disease

open access: yesOrphanet Journal of Rare Diseases
Background Fabry disease (FD) exhibits marked clinical heterogeneity that cannot be fully explained by residual α-galactosidase A activity. Mitochondrial dysfunction has been reported in FD, but the role of mitochondrial stress remains unexplored ...
Lucia Lavalle   +5 more
doaj   +1 more source

A Multidimensional Engineering Strategy Reprograms Microglia via Targeted and Sustained‐Release Extracellular Vesicles for Spinal Cord Injury Repair

open access: yesAdvanced Science, EarlyView.
A multifunctional EV‐based nanoplatform (Ang‐TEVs@Gel) was engineered via preconditioning, surface targeting, and ROS‐responsive hydrogel encapsulation to reprogram microglia. This system restored autophagy via miR‐664a‐3p/PIK3CA axis, cleared myelin debris, resolved neuroinflammation, and promoted axon remyelination, ultimately achieving robust motor ...
Wu Xiong   +17 more
wiley   +1 more source

Nucleoside‐Modified mRNA Encoding Alpha‐Galactosidase A Ameliorates Fabry Disease Phenotypes in Human IPSC‐Derived Cardiomyocytes

open access: yesAdvanced Science, EarlyView.
Human iPSC‐derived Fabry cardiomyocytes exhibited broad transcriptional dysregulation, apoptosis, mitochondrial dysfunction, impaired reactive oxygen species handling, altered contractility, and abnormal calcium transient decay, potentially mediated by phospholamban hyperphosphorylation.
Malte Juchem   +24 more
wiley   +1 more source

Types and Genetic Evaluation of Lysosomal Storage Diseases in Kurdistan Region

open access: yesمجلة الكوفة الطبية
Background and objectives: Lysosomal storage diseases are a set of single-gene disorders that is attributed to insufficient certain lysosomal hydrolase activity or non-enzymatic proteins vital for typical lysosomal functions.
Lana Ahmed Mohammed
doaj   +1 more source

Lysosome

open access: yesJournal of Inborn Errors of Metabolism and Screening, 2016
Since Christian de Duve first described the lysosome in the 1950s, it has been generally presented as a membrane-bound compartment containing acid hydrolases that enables the cell to degrade molecules without being digested by autolysis.
Ursula Matte BSc, PhD   +1 more
doaj   +1 more source

Lysosomal storage disorders: emerging therapeutic options require early diagnosis

open access: yes, 2003
Lysosomal storage disorders have been recognised as one of the major groups of genetic disorders affecting children and adults. With over 40 different disorders and a combined prevalence of up to 1:5000 births, this group of disorders is a major public ...
Hopwood, J., Meikle, P.
core   +1 more source

A Decoy‐Receptor‐Armed Biomimetic Nanotherapeutic With Inherent Tropism for Conserved Pathogenic Macrophages for Treating Osteoarthritis and Intervertebral Disc Degeneration

open access: yesAdvanced Science, EarlyView.
A biomimetic doppelgänger nanosystem neutralizes extracellular inflammatory cytokines and silences intracellular pyroptosis, reprogramming pathogenic macrophages to attenuate both joint and spine degeneration. ABSTRACT Osteoarthritis (OA) and intervertebral disc degeneration (IVDD) are debilitating musculoskeletal disorders driven by shared ...
Fudong Li   +9 more
wiley   +1 more source

Induced pluripotent stem cell models of lysosomal storage disorders

open access: yes, 2017
Induced pluripotent stem cells (iPSCs) have provided new opportunities to explore the cell biology and pathophysiology of human diseases, and the lysosomal storage disorder research community has been quick to adopt this technology.
Tamanna Roshan Lal   +5 more
core   +2 more sources

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