Results 121 to 130 of about 38,108 (242)

COVID-19 and Vaccination Status in Lysosomal Storage Diseases: A Single-Center Experience. [PDF]

Turk Arch Pediatr, 2023
Yoldaş Çelik M, Canda E, Yazıcı H, Erdem F, Yazıcı Özkaya P, Bal Sahbudak Z, Kalkan Uçar S, Çoker M.   +7 more
europepmc   +1 more source

Nucleoside‐Modified mRNA Encoding Alpha‐Galactosidase A Ameliorates Fabry Disease Phenotypes in Human IPSC‐Derived Cardiomyocytes

Advanced Science, EarlyView.
Human iPSC‐derived Fabry cardiomyocytes exhibited broad transcriptional dysregulation, apoptosis, mitochondrial dysfunction, impaired reactive oxygen species handling, altered contractility, and abnormal calcium transient decay, potentially mediated by phospholamban hyperphosphorylation.
Malte Juchem, Lea Oehlsen, Sedef Ersoy, Jia Li Ye, Natalie Weber, Elisa Mohr, Wilson Agyapong, Junqing Liu, Maximilian Fuchs, Ke Xiao, Christopher Jahn, Reto Eggenschwiler, Tobias Cantz, Julia Beimdiek, Falk F. R. Buettner, Theresia Kraft, Jan Hegermann, Ivonne J. Knorr, Lisa Ernst, Linda Feldbrügge, Resa Puffert, Kevin Schmidt, Christian Bär, Jeannine Hoepfner, Thomas Thum   +24 more
wiley   +1 more source

A Decoy‐Receptor‐Armed Biomimetic Nanotherapeutic With Inherent Tropism for Conserved Pathogenic Macrophages for Treating Osteoarthritis and Intervertebral Disc Degeneration

Advanced Science, EarlyView.
A biomimetic doppelgänger nanosystem neutralizes extracellular inflammatory cytokines and silences intracellular pyroptosis, reprogramming pathogenic macrophages to attenuate both joint and spine degeneration. ABSTRACT Osteoarthritis (OA) and intervertebral disc degeneration (IVDD) are debilitating musculoskeletal disorders driven by shared ...
Fudong Li, Bin Zhang, Zhiqiu Zhang, Bing Zheng, Linhui Han, Chen Yan, Jialin Li, Kaiqiang Sun, Tianyi Zhao, Jiangang Shi   +9 more
wiley   +1 more source

Engineering CAR‐Macrophages With Advanced Delivery Systems for Tissue Repair

Advanced Science, EarlyView.
This review highlights how engineered macrophages equipped with chimeric antigen receptors (CAR) guide tissue repair by recognizing disease‐related targets, clearing harmful cells, and reshaping local immune environments. It summarizes macrophage biology, CAR design, delivery platforms, and functionalization strategies, and discusses emerging ...
Yixin Zhang, Jintian Yu, Liang Yuan, Ziliang Fu, Yiming Yao, Lingna Chen, Shixiong Chen, Aikemu Ainiwaer, Xiaolin Cui   +8 more
wiley   +1 more source

Gene therapy for lysosomal storage diseases: Current clinical trial prospects. [PDF]

Front Genet, 2023
Kido J, Sugawara K, Nakamura K.
europepmc   +1 more source

Dual‐Gene Edited Extracellular Vesicles Remodel the Redox Homeostasis to Inhibit Ferroptosis in Intervertebral Disc Degeneration

Advanced Science, EarlyView.
ABSTRACT Intervertebral disc degeneration (IDD) is driven by ferroptosis of nucleus pulposus cells (NPCs) as a core pathological mechanism. Nucleus pulposus progenitor cells (NPPCs), exhibiting stem cell‐like properties, yield extracellular vesicles (PEVs) with high affinity for NPCs and enable targeted phenotypic regulation.
Jing Yan, Yazhou Lin, Shuo Miao, Minghao Chao, Chengbai Dai, Ziwen Hao, Rui Chen, Ming Guan, Bin Pan, Fenglei Gao, Quan Zhou   +10 more
wiley   +1 more source

Purifying and profiling lysosomes to expand understanding of lysosomal dysfunction–associated diseases

The Journal of Clinical Investigation
Lysosome storage dysfunction plays a central role in numerous human diseases, but a lack of appropriate tools has hindered lysosomal content profiling in clinical settings. In this issue of the JCI, Saarela et al. introduce a method called tagless LysoIP
Ali Shilatifard, Issam Ben-Sahra
doaj   +1 more source

A Microenvironment‐Adaptive Scaffold Rejuvenates Peripheral Nerve Regeneration During Aging Via Dual Modulation of Ferroptotic Stimuli in Schwann Cells

Advanced Science, EarlyView.
(i) Aging impairs peripheral nerve regeneration via its dual ferroptosis‐mediated pathologies in senescent schwann cells. (ii) The composite nerve conduit is a symbiosis niche integrating microenvironment‐responsive nanoparticles (Ga‐PTAs) and maxillofacial‐derived mesenchymal stem cells (Mmscs).
Ning Zhan, Lixin Tian, Lianjie Peng, Jingyu Zhang, Lining Lin, Xiaoting Zhang, Yaxian Luo, Zhichao Liu, Xinhua Yao, Mouyuan Sun, Yong He, Mengfei Yu, Huiming Wang   +12 more
wiley   +1 more source

Phase Separation, Material State, and Condensate Fate in Mammalian Autophagy

Advanced Science, EarlyView.
ABSTRACT Biomolecular phase separation has emerged as a key organizing principle in macroautophagy (hereafter autophagy). In mammalian cells, phase‐separated condensates not only serve as substrates for selective degradation, but also act as dynamic platforms for cargo recognition, signaling integration, and autophagosome assembly.
Yuanqiang Lin, Mingran Dai, Enyong Dai, Rui Kang, Daniel J. Klionsky, Daolin Tang, Yu Sun, Ningning Lv, Junnan Jiang   +8 more
wiley   +1 more source

Targeting the NR1D1–IGF2BP2–V‐ATPase Axis With Hybrid Nanovesicles Restores Macrophage Rhythms to Reverse Sepsis‐Induced Immunosuppression

Advanced Science, EarlyView.
Sepsis disrupts immune‐cell rhythms and weakens bacterial clearance. Biomimetic nanovesicles combining erythrocyte and inflammation‐activated macrophage membranes deliver siNR1D1 to dysfunctional macrophages, restoring the NR1D1–IGF2BP2–V‐ATPase pathway, circadian regulation, phagolysosomal acidification, and antimicrobial defense.
Lang Chen, Wenyi Lin, Lang Jiang, Xuehui Gao, Chenyan Yu, Jili Han, Zhenping Liu, Xuefeng Li, Mei He, Chang Li, Qilan Li, Chaoying Song, Jiqian Xu, You Shang   +13 more
wiley   +1 more source