Results 121 to 130 of about 3,523,765 (264)

TFEB overexpression alleviates autophagy-lysosomal deficits caused by progranulin insufficiency

Scientific Reports
Progranulin is a pro-protein that is necessary for maintaining lysosomal function. Loss-of-function progranulin (GRN) mutations are a dominant cause of frontotemporal dementia (FTD).
Wren O. Nader, Kaylan S. Brown, Nicholas R. Boyle, Azariah K. Kaplelach, Shaimaa M. Abdelaziz, Skylar E. Davis, Qays Aljabi, Ahmad R. Hakim, Amelia G. Davidson, Giacynta A. Vollmer, Leah C. Wright, J. Bailey Echols, Joelle Saad, Nicholas S. Pena, Andrew E. Arrant   +14 more
doaj   +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

Supramolecular Degraders: An Emerging Paradigm in Targeted Protein Degradation

Advanced Science, EarlyView.
Dynamic supramolecular assembly reshapes targeted protein degradation by coordinating modular degrader construction, delivery, functional integration, and intracellular assembly or activation across proteasomal, endosomal–lysosomal, and autophagy–lysosomal pathways.
Kongjun Liu, Xue Yuan, Yan Zhou, Rui Yuan, Shaocheng Xie, Yong Chen, Peiyi Wang, Jianmei Gao, Qihai Gong   +8 more
wiley   +1 more source

Curated incidence of lysosomal storage diseases from the Taiwan Biobank. [PDF]

NPJ Genom Med, 2023
Tsai MM, Hung MZ, Lin YL, Lee NC, Chien YH, Hwu WL.   +5 more
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

Lysosomes and Storage diseases

, 1970
info:eu-repo/semantics ...
Grégoire, Anne, Tondeur, Michel, Pohl, Sylvie, Dustin, Pierre   +3 more
openaire   +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

Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases. [PDF]

Neural Regen Res, 2023
Mandolfo O, Bigger BW.
europepmc   +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