Results 141 to 150 of about 8,108 (242)

Material‐Encoded Synchronization of Immunogenic Cell Death With Adenosine A2A Receptor Blockade Reprograms the Tumor Microenvironment

open access: yesAdvanced Science, EarlyView.
A bismuth–copper diselenide–based nanoplatform (BSCS@PHY) coordinates immunogenic cell death with local A2A receptor blockade in 4T1 tumors. Thermally triggered shell melting exposes catalytic surfaces for glutathione depletion and chemodynamic ROS generation, while co‐delivering an A2AR antagonist and yeast‐wall adjuvant to enhance dendritic‐cell ...
Xiangting Yi   +12 more
wiley   +1 more source

Profiling Co‐Occurrent Morphological Phenotypes and Their Degree of Expression Severity in Vacuolated Cells by Holo‐Tomographic Flow Cytometry and Fractal Analysis

open access: yesAdvanced Intelligent Systems, EarlyView.
HTFC gets 3D refractive index tomograms of flowing cells. Label‐free monocytes are engineered to express patterns of cytoplasmic vacuoles. From the tomogram, an efficient dimensionality reduction is operated. Interpretable features are extracted to classify the expression severity of phenotypes coexisting in each cell, visually represented by a seven ...
Marika Valentino   +9 more
wiley   +1 more source

Cumulative Antigen Suppression Reduces Clonal Plasma Cell Evolution in Gaucher Disease

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Chronic antigenic stimulation is implicated in the pathogenesis of monoclonal gammopathy and multiple myeloma, yet longitudinal human evidence linking sustained antigen exposure to modifiable clonal plasma cell evolution remains limited. Gaucher disease (GD), caused by biallelic GBA1 pathogenic variants, is characterized by accumulation of ...
Noor Ul Ain   +10 more
wiley   +1 more source

The lysosomal storage disease fucosidosis: towards enzyme replacement therapy

open access: yes, 2016
Wolf H. The lysosomal storage disease fucosidosis: towards enzyme replacement therapy. Bielefeld: Universität Bielefeld; 2016.Fucosidosis is a rare lysosomal storage disease caused by the deficiency of the lysosomal glycosidase a-L-fucosidase resulting ...
Wolf, Heike
core  

The Critical Role of Fractionated Urine Glycosaminoglycans in the Evaluation of Mucopolysaccharidosis Type II in Four Unrelated Families

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Since 2015, Ann and Robert H. Lurie Children's Hospital has performed diagnostic testing for infants who screen positive for mucopolysaccharidosis type II (MPS II) on the Illinois newborn screen. Preliminary diagnostic testing includes measurement of plasma iduronate‐2‐sulfatase enzyme activity and urinary glycosaminoglycan analysis, followed ...
Carly A. Rasmussen   +5 more
wiley   +1 more source

Expression variation in lysosomal storage disorder genes

open access: yes, 2006
Metachromatic leukodystrophy (MLD) and Gaucher disease (GD) are caused by a deficiency of arylsulphatase A (ASA) and b-glucocerebrosidase (GBA), respectively.
Mason, Lyndel Ann
core  

Therapeutic Applications of Stimuli‐Based Release and Engineering of Extracellular Vesicles

open access: yesAdvanced NanoBiomed Research, EarlyView.
This review summarizes the effects of endogenous and exogenous stimuli, their effects on the natural release of extracellular vesicles, as well as their uptake and release. It also gives an overview of stimuli‐responsive EVs and their therapeutic applications. Extracellular vesicles (EVs), nano‐ to microsized lipid bilayer membrane‐bound particles, are
Gloria Kemunto, Kristen Dellinger
wiley   +1 more source

Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Abstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Kimitoshi Nakamura   +6 more
wiley   +1 more source

A zebrafish model of combined saposin deficiency identifies acid sphingomyelinase as a potential therapeutic target

open access: yesDisease Models & Mechanisms, 2023
Tejia Zhang   +9 more
doaj   +1 more source

Advancing design strategies in smart stimulus‐responsive liposomes for drug release and nanomedicine

open access: yesBMEMat, EarlyView.
Schematic illustration of stimulus‐responsive liposomes designed for controlled drug release and nanomedicine. The innermost circle represents different liposomal structures, including unilamellar, multilamellar, and multivesicular liposomes. The middle layer illustrates the responsive phospholipid components.
Yuchen Guo   +9 more
wiley   +1 more source

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