Results 51 to 60 of about 31,524 (206)

Unraveling A4GALT Mechanism and Its Modulation With Adamantyl‐Galactosylceramide Analogues: Advancing Fabry Disease Therapeutic Strategies

Angewandte Chemie, EarlyView.
A 310‐helix‐mediated conformational switch promotes a front‐face SNi‐like catalysis by human A4GALT. Mechanism‐guided design identifies AdaGalCer as a selective modulator of globotriaosylceramide (Gb3) biosynthesis, opening a clear route toward new Fabry disease therapeutics.
Nicky de Koster, Òscar Vidal‐Gironès, Rowan de Graaf, Ken Kok, Lindsey T. Lelieveld, Jackelien Hoogervorst, Maria J. Ferraz, José Pablo Rivas‐Fernández, Rob F. Lammers, Herman S. Overkleeft, Johannes M. F. G. Aerts, Rolf G. Boot, Carme Rovira, Marta Artola   +13 more
wiley   +2 more sources

A Rapid and Simple UHPLC-MS/MS Method for Quantification of Plasma Globotriaosylsphingosine (lyso-Gb3)

Molecules, 2021
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by α-galactosidase A gene (GLA) mutations, resulting in loss of activity of the lysosomal hydrolase, α-galactosidase A (α-Gal A).
Alessandro Perrone, Susan Mohamed, V. Donadio, R. Liguori, M. Contin   +4 more
semanticscholar   +1 more source

Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease.

PLoS ONE, 2015
Fabry disease is caused by deficient activity of α-galactosidase A (GLA) and characterized by systemic accumulation of glycosphingolipids, substrates of the enzyme.
Hideaki Sueoka, Mikio Aoki, Takahiro Tsukimura, Tadayasu Togawa, Hitoshi Sakuraba   +4 more
doaj   +1 more source

Entropy Drives the Formation of Salt Bridges in the Protein GB3 [PDF]

Angewandte Chemie International Edition, 2017
AbstractSalt bridges are very common in proteins. But what drives the formation of protein salt bridges is not clear. In this work, we determined the strength of four salt bridges in the protein GB3 by measuring the ΔpKa values of the basic residues that constitute the salt bridges with a highly accurate NMR titration method at different temperatures ...
Ning Zhang, Yefei Wang, Liaoyuan An, Xiangfei Song, Qingshan Huang, Zhijun Liu, Lishan Yao   +6 more
openaire   +2 more sources

A novel soluble analog of the HIV-1 fusion cofactor, globotriaosylceramide (Gb3), eliminates the cholesterol requirement for high affinity gp120/Gb3 interaction

Journal of Lipid Research, 2002
We have analyzed the interaction of adamantyl Gb3 (adaGb3), a semi-synthetic soluble analog of Gb3, with HIV-1 surface envelope glycoprotein gp120. In this analog, which was orginally designed to inhibit verotoxin binding to its glycolipid receptor, Gb3,
Radhia Mahfoud, Murugesapillai Mylvaganam, Clifford A. Lingwood, Jacques Fantini   +3 more
doaj   +1 more source

Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

Genetics in Medicine, 2020
Purpose To assess the utility of globotriaosylsphingosine (lyso-Gb 3 ) for clinical monitoring of treatment response in patients with Fabry disease receiving migalastat.
D. Bichet, J. Aerts, C. Auray-Blais, H. Maruyama, A. Mehta, N. Skuban, E. Krusinska, R. Schiffmann   +7 more
semanticscholar   +1 more source

Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. [PDF]

PLoS ONE, 2013
Fabry disease (FD) results from mutations in the gene (GLA) that encodes the lysosomal enzyme α-galactosidase A (α-Gal A), and involves pathological accumulation of globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3).
Brandy Young-Gqamana, Nastry Brignol, Hui-Hwa Chang, Richie Khanna, Rebecca Soska, Maria Fuller, Sheela A Sitaraman, Dominique P Germain, Roberto Giugliani, Derralynn A Hughes, Atul Mehta, Kathy Nicholls, Pol Boudes, David J Lockhart, Kenneth J Valenzano, Elfrida R Benjamin   +15 more
doaj   +1 more source

Tumor Delivery of Ultrasound Contrast Agents Using Shiga Toxin B Subunit

Molecular Imaging, 2011
The present study demonstrates the targeting of ultrasound contrast agents to human xenograft tumors by exploiting the overexpression of the glycolipid Gb3 in neovasculature.
Olivier Couture, Estelle Dransart, Sabrina Dehay, Fariba Nemati, Didier Decaudin, Ludger Johannes, Mickael Tanter   +6 more
doaj   +1 more source

Sex differences of urinary and kidney globotriaosylceramide and lyso-globotriaosylceramide in Fabry mice

Journal of Lipid Research, 2011
The aim of our study was to measure globotriaosylceramide (Gb3) and lyso-Gb3 levels by tandem mass spectrometry in the urine and kidney in Fabry (gla knockout) mice and wild-type controls.
Brandon Durant, Sabrina Forni, Lawrence Sweetman, Nastry Brignol, Xing-Li Meng, Elfrida R. Benjamin, Raphael Schiffmann, Jin-Song Shen   +7 more
doaj   +1 more source

Susceptibility of Human B-Lymphoblastoid Cells to Shiga Toxin Intoxication Homologues

Microorganisms
Shiga toxins (Stx), produced by Stx-producing Escherichia coli (STEC), are known to target Gb3-expressing cells, contributing to organ pathology such as in the kidney and brain.
Alfredo G. Torres, Alexander J. Badten, Susana Oaxaca-Torres, Itziar Chapartegui-González, Ennzo Ortega, Rama R. Atitkar, G. Jilani Chaudry, Carlton C. Brinkley, Angela Melton-Celsa   +8 more
doaj   +1 more source