Results 161 to 170 of about 3,524,002 (263)

How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases? [PDF]

Orphanet J Rare Dis
Spataro F, Solimando AG, Desantis V, Vacca A, Di Girolamo A, Ria R.   +5 more
europepmc   +1 more source

Altered blood-brain barrier transport of nanotherapeutics in lysosomal storage diseases. [PDF]

J Control Release, 2022
Solomon M, Loeck M, Silva-Abreu M, Moscoso R, Bautista R, Vigo M, Muro S.   +6 more
europepmc   +1 more source

Magnetoreceptor Promotes Hepatocellular Carcinoma Development and Blunts Sorafenib‐Induced Ferroptosis by Regulating Iron Homeostasis

Advanced Science, EarlyView.
Magnetoreceptor (MagR) regulates iron homeostasis and mitochondrial function in hepatocellular carcinoma (HCC) cells. Specifically, MagR deficiency suppresses tumor cell proliferation, migration, and invasion and enhances sorafenib‐induced ferroptosis through iron accumulation.
Tao Wang, Chenghua Song, Xin He, Mengzhou Wang, Wuming Liu, Xiaolin Wang, Yuanyuan Zhang, Jia Zhang, Lin Zhang, Dan Li, Yu Zhang, Zheng Wu, Yi Lyu, Rongqian Wu   +13 more
wiley   +1 more source

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases. [PDF]

Mol Genet Metab
Kell P, Mishra S, Gray-Edwards HL, Martin DR, Gaudioso A, Ledesma MD, Bongarzone ER, Sands MS, Sidransky E, Ory DS, Jiang X.   +10 more
europepmc   +1 more source

The Antifungal Antibiotic Filipin as a Diagnostic Tool of Cholesterol Alterations in Lysosomal Storage Diseases and Neurodegenerative Disorders. [PDF]

Antibiotics (Basel), 2023
Bruno F, Camuso S, Capuozzo E, Canterini S.   +3 more
europepmc   +1 more source

Huntingtin Aggregate‐Responsive Autophagy Gene Circuit Mitigates Disease Pathology in R6/2 Mice

Advanced Science, EarlyView.
CD98‐mediated receptor‐mediated transcytosis enables LIP‐CD98 nanocarriers to cross the blood–brain barrier and deliver ARAA to neurons. mHTT aggregates activate the 11G–NarX sensor, initiating Auto‐P and Trans‐P signaling through the VP48–NarL relay.
Jie Zhu, Xi‐xiu Xie, Lei Li, Chen Tian, Hao‐tian Wang, Xiao‐jie Wang, Xiao‐lin Yu, Gui‐feng Zhang, Rui‐tian Liu   +8 more
wiley   +1 more source

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 systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases. [PDF]

Cost Eff Resour Alloc, 2022
Katsigianni EI, Petrou P.
europepmc   +1 more source

LAPTM5 Downregulation‐Driven VAMP8 Phosphorylation Impairs Autophagosome‐Lysosome Fusion and Aggravates Septic Acute Lung Injury

Advanced Science, EarlyView.
LAPTM5 functions as a critical molecular scaffold bridging PGAM5 and VAMP to facilitate PGAM5‐mediated dephosphorylation of VAMP8. This cascade promotes autophagosome‐lysosome fusion, restores impaired autophagic flux, and clears damaged mitochondria, thereby mitigating cellular oxidative stress and systemic inflammation.
Lang Jiang, Qilan Li, Hao Liu, Xuehui Gao, Hongyu Nie, Lang Chen, Mei He, Xuefeng Li, Xiangming Fang, Jiqian Xu, Hao Zhang, Jiahong Xia, You Shang   +12 more
wiley   +1 more source

Chlorination‐Driven BODIPY Fluorescent Probes for the Selective Monitoring of Myeloperoxidase Activity in Cells and Inflamed Mouse Models

Angewandte Chemie, EarlyView.
A meso‐carboxamide‐substituted BODIPY fluorescent probe reports myeloperoxidase (MPO) activity via electrophilic chlorination by MPO‐derived HOCl, generating a highly emissive chlorinated product with a ca. 40 nm bathochromic shift. Applications in quantitative MPO assays, cellular imaging of MPO‐derived HOCl, redox‐dependent discrimination of cancer ...
Siyoung Cho, Yongyang Luo, Jeehyeon Bae, Youngmi Kim   +3 more
wiley   +2 more sources