Results 51 to 60 of about 58,222 (243)

Differentiation of norm and pathology during selective biochemical skreening of lysosomal storage diseases with increased excretion of oligosaccharides [PDF]

The Ukrainian Biochemical Journal, 2015
Oligosaccharides are a class of polymeric carbohydrates, which are constituents of a glycoside portion of glycoprotein and glycolipid molecules. The lysosomal hydrolase dysfunction due to lysosomal storage disorders results in partial or complete failure
N. Y. Mytsyk, N. V. Olkhovych, N. G. Gorovenko   +2 more
doaj   +1 more source

YAP plays a crucial role in the development of cardiomyopathy in lysosomal storage diseases.

Journal of Clinical Investigation, 2020
Lysosomal dysfunction caused by mutations in lysosomal genes results in lysosomal storage disorder (LSD), characterized by accumulation of damaged proteins and organelles in cells and functional abnormalities in major organs, including the heart ...
Shohei Ikeda, Jihoon Nah, A. Shirakabe, P. Zhai, S. Oka, S. Sciarretta, K. Guan, H. Shimokawa, J. Sadoshima   +8 more
semanticscholar   +1 more source

Olink Proteomics Analysis Reveals Heterogeneous Responses to FcRn Blockade in Anti‐AChR Antibody‐Positive Myasthenia Gravis: FGF‐19 as a Novel Biomarker

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective This study aimed to systematically observe the clinical manifestations, immune cell subsets, and dynamic changes in serological indicators in patients with myasthenia gravis (MG) before and after efgartigimod (EFG) treatment. Methods We analyzed the baseline data, laboratory parameters, and lymphocyte subset proportions in MG ...
Tiancheng Luo, Deyou Peng, Zhouao Zhang, Mingjin Yang, Xinyan Guo, Tianyu Ma, Xiaoyu Huang, Mingming Xu, Linlin Fu, Yong Zhang   +9 more
wiley   +1 more source

Genomic Expression Analyses Reveal Lysosomal, Innate Immunity Proteins, as Disease Correlates in Murine Models of a Lysosomal Storage Disorder

PLoS ONE, 2012
Niemann-Pick Type C (NPC) disease is a rare, genetic, lysosomal disorder with progressive neurodegeneration. Poor understanding of the pathophysiology and a lack of blood-based diagnostic markers are major hurdles in the treatment and management of NPC ...
Md. Suhail Alam, Michelle Getz, I. Safeukui, Sue Yi, P. Tamez, Jenny Shin, P. Velázquez, K. Haldar   +7 more
semanticscholar   +1 more source

Oral Dosed Organo‐Silica Nanoparticles Restore Glucose Homeostasis and β‐Cell Function in Diabetes Rats

Advanced Functional Materials, EarlyView.
An oral nanoplatform, MOP@T@D, which can maintain glucose homeostasis and restore islet β cells in diabetic rats is developed. It achieves efficient intestinal absorption and liver‐targeted delivery. The nanoparticle disintegrates only in response to hyperglycemia to release insulin on demand and provides antioxidant protection through selenoprotein ...
Chenxiao Chu, Mingli Wei, Che Bian, Xiaoshuang Bi, Yaxin Deng, Peifu Xiao, Jiansong Zhao, Yuying Wang, Haibing He, Jingxin Gou, Tian Yin, Xing Tang, Li Yang, Hongbo Zhang, Yu Zhang   +14 more
wiley   +1 more source

Autophagy in lysosomal storage disorders [PDF]

Autophagy, 2012
Lysosomes are ubiquitous intracellular organelles that have an acidic internal pH, and play crucial roles in cellular clearance. Numerous functions depend on normal lysosomes, including the turnover of cellular constituents, cholesterol homeostasis, downregulation of surface receptors, inactivation of pathogenic organisms, repair of the plasma membrane
Lieberman AP, Puertollano R, Raben N, Slaugenhaupt S, Walkley SU, BALLABIO, ANDREA   +5 more
openaire   +4 more sources

New Insights into Atonic Postpartum Hemorrhage: Animal Model Construction Based on Placental Nanodelivery Systems

Advanced Healthcare Materials, EarlyView.
This study develops a placenta‐targeted nanodelivery system co‐loading HMGB1 protein and the NLRP3 agonist nigericin to establish an animal model of atonic postpartum hemorrhage. The model accurately recapitulates clinical phenotypes, including prolonged labor and uterine contractility dysfunction, while revealing inflammatory activation in placental ...
Jiangxue Qu, Ruiqi Wu, Hai Jiang, Lian Chen, Yingying Tian, Shuai Zeng, Tongmin Liu, Cheng Zhao, Huifeng Shi, Xiaolong Liang, Yangyu Zhao   +10 more
wiley   +1 more source

Strategies to Improve the Lipophilicity of Hydrophilic Macromolecular Drugs

Advanced Healthcare Materials, EarlyView.
Hydrophilic macromolecular drugs can be successfully lipidized by covalent attachment of lipids, by hydrophobic ion pairing with negatively or positively charged surfactants, and by dry or wet reverse micelle formation. Lipophilicity enhancement of hydrophilic macromolecules has several benefits including stability and bioavailability improvement ...
Sera Lindner, Stefan Keim, Soheil Haddadzadegan, Odile Fernandez Romero, Katrin Zöller, Gabriel Stern, Ilaria Cesi, Krum Kafedjiiski, Andreas Bernkop‐Schnürch   +8 more
wiley   +1 more source

Cardiac device implantation and device usage in Fabry and hypertrophic cardiomyopathy

Orphanet Journal of Rare Diseases, 2022
Background Fabry disease (FD) is a treatable X-linked condition leading to progressive cardiac disease, arrhythmia and premature death. We aimed to increase awareness of the arrhythmogenicity of Fabry cardiomyopathy, by comparing device usage in patients
Ravi Vijapurapu, William Bradlow, Francisco Leyva, James C. Moon, Abbasin Zegard, Nigel Lewis, D. Kotecha, Ana Jovanovic, Derralynn A. Hughes, Peter Woolfson, Richard P. Steeds, Tarekegn Geberhiwot   +11 more
doaj   +1 more source

Engineered ETS1‐Nanoconjugate Restores Immune Homeostasis through Dual Immune‐Vascular Modulation in Relapsing and Progressive Multiple Sclerosis

Advanced Healthcare Materials, EarlyView.
The biomimetic nanoplatform IMNP (ETS1 pDNA/PBAE@ITP‐MM) undergoes targeted disassembly at inflammatory vascular sites to release the ETS1 plasmid (pETS1). This release initiates a cascade of effects that inhibit pathogenic pathways and support immune homeostasis. (Abbreviations: EndMT, endothelial‐to‐mesenchymal transition; EC, endothelial cell; TC, T
Feng Zhang, Si‐Bo Yang, Yan Luo, Xiao‐Di Sun, Bo‐Hao Chang, Da‐Qiang Zhou, Jun‐Jie Jiang, Ya‐Nan Li, Jie‐Hong Wu, Hui‐Juan Jin, Hang Yang, Hang Hu, Yi‐Fan Zhou, Bo Hu   +13 more
wiley   +1 more source