Results 151 to 160 of about 3,523,765 (264)

Novel Fused Pyrazole Amide Analogs as Glucosylceramide Synthase Inhibitors for Treating Lysosomal Storage Diseases, Neurodegenerative Diseases, Cystic Diseases, and Cancer. [PDF]

ACS Med Chem Lett, 2023
Sabnis RW.
europepmc   +1 more source

Mutant KRAS‐m6A Epitranscriptome Axis Promotes Colorectal Cancer and is a Therapeutic Target

Advanced Science, EarlyView.
Mutant KRAS‐m6A axis promotes colorectal cancer (CRC) progression. Mutant KRAS stabilizes METTL3, leading to increased m6A‐modified BCL9L mRNA and translation of BCL9L protein. BCL9L in turn mediates TGF‐β secretion to induce differentiation of Treg and an immunosuppressive microenvironment.
Danyu Chen, Pingmei Huang, Ruizhi Tao, Fenfen Ji, Qinyao Wei, Lok Hin Ko, Lianxin Zhou, Huarong Chen, Alvin Cheung, Wei Kang, Ko Fai To, Jun Yu, Chi Chun Wong   +12 more
wiley   +1 more source

Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]

EBioMedicine
eBioMedicine.
europepmc   +1 more source

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. [PDF]

Front Mol Neurosci, 2022
Flunkert S, Hutter-Paier B, Sun Y, Kehr J.   +3 more
europepmc   +1 more source

SIRT7‐Mediated H2BK120 Succinylation Drives Aberrant Mitophagy in Sepsis‐Associated Cognitive Dysfunction

Advanced Science, EarlyView.
Sepsis triggers hippocampal SIRT7 loss, promoting histone H2B succinylation and activation of the PD‐1/PD‐L1–PINK1 axis. This metabolic‐epigenetic cascade drives aberrant mitophagy and neuronal injury, ultimately causing cognitive deficits. Targeting SIRT7‐dependent succinylation offers a potential strategy to protect brain function during sepsis ...
Na Meng, Jiateng Zhou, Xiaoyu Guo, Ting Hong, Xuelian Li, Xingyu Wei, Chanhua Zhang, Xixue Zhang, Songbin Liu, Yulin Zhang, Liangfang Yao, Lina Huang, Weidong Gu   +12 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

Japanese experience of newborn screening for lysosomal storage diseases and adrenoleukodystrophy. [PDF]

Orphanet J Rare Dis
Onuki T, Tajika M, Sugiyama Y, Shimura M, Ichimoto K, Tanaka T, Nyuzuki H, Kosuga M, Migita O, Ito T, Sasai H, Bo R, Hamada J, Hamazaki T, Sakai N, Inoue T, Nakamura K, Okuyama T, Murayama K.   +18 more
europepmc   +1 more source

Correction to: 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

Multifunctional Nanovaccine‐Radiotherapy Synergy Drives Anti‐Tumor Immunity via Multi‐Cascade Cell Death for Pancreatic Cancer Therapy

Advanced Science, EarlyView.
Machine‐learning‐driven transcriptomic analysis identifies FSTL3 as a key tumor‐microenvironment regulator in desmoplastic pancreatic adenocarcinoma. A cRGD‐modified liposome co‐loading gemcitabine and siFSTL3 serves as an irradiation‐triggered in situ nanovaccine. It synergizes with radiotherapy to induce ferroptosis and immunogenic cell death through
Chengyi Huang, Yushu Han, Xiang Liu, Chuanhao Wang, Xiaofei Zhu, Di Chen, Yangyang Gong, Liang Chen, Wenjuan Chen, Zhenyuan Miao, Bufu Tang, Huojun Zhang   +11 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