Results 71 to 80 of about 21,986 (247)

A Plug‐and‐Play Platform for Customizing Multivalent Degraders and Degrader‐Drug Conjugates

Advanced Science, EarlyView.
Membrane proteins remain challenging targets for conventional TPD approaches. Here, the authors develop UPTAB, a modular platform leveraging ultrahigh‐affinity orthogonal Im/CL protein pairs for lysosomal degradation of membrane proteins. Mono‐targeted (Type‐I), dual‐targeted (Type‐II), and tri‐targeted (Type‐III) UPTABs enable simultaneous degradation
Mengqing Zhao, Yan Deng, Jianjian Han, Yong Xie, Kai Bao, Lixin Ma, Lilong Liu, Wuxiang Mao   +7 more
wiley   +1 more source

Generation and characterization of two iPSC lines derived from subjects with Free Sialic Acid Storage Disorder (FSASD)

Stem Cell Research
Free sialic acid storage disorder (FSASD) is a rare, autosomal recessive, neurodegenerative disorder caused by biallelic mutations in SLC17A5, encoding the lysosomal transmembrane sialic acid exporter, SLC17A5.
Marya S. Sabir, Petcharat Leoyklang, Mary E. Hackbarth, Evgenia Pak, Amalia Dutra, Richard Tait, Laura Pollard, David R. Adams, William A. Gahl, Marjan Huizing, May Christine V. Malicdan   +10 more
doaj   +1 more source

SIRT6‐Mediated Deacetylation of ATF3 Promotes Silica‐Induced Lung Fibrosis by Enhancing its Nuclear Import via Binding to Importin α

Advanced Science, EarlyView.
SIRT6‐mediated ATF3 acetylation drives MGARP transcription and mitochondrial dysfunction in macrophages, promoting macrophage senescence and pulmonary fibrosis. Mechanistically, HSP70/Importin α competitively binds to ATF3, modulating its nuclear translocation.
Demin Cheng, Wenxia Bu, Fengxu Wang, Yueyuan Jin, Rongzhu Liu, Rui Zhao, Xuehai Wang, Mengna Jiang, Jinping Shen, Xinhang Cheng, Zuming Chen, Li Zhu, Jinlong Li, Zhenzhong Ge, Shichen Miao, Haotian Xu, Xiaoyu Zhou, Dongming Wang, Xinyuan Zhao   +18 more
wiley   +1 more source

Xenogeneic Mitochondrial Transplantation Improves Selected Age‐Associated Phenotypes in Mice

Advanced Science, EarlyView.
Yak‐derived xenogeneic mitochondrial transplantation improves selected age‐associated phenotypes in mice, enhances mitochondrial functional readouts, and engages host mitochondrial quality‐control pathways. Broad tissue biodistribution, increased ATP production and mtDNA copy number, reduced ROS levels and dysfunctional mitochondria, improved motility ...
Wenpeng Li, Xijia Wang, Yaning Hu, Zhen Yang, Tian Niu, Xingbo Zhao   +5 more
wiley   +1 more source

A Dual‐Phase Exosomal Nanotherapy Enhances Continual Efferocytosis by Coordinating Checkpoint Inhibition and Metabolic Reprogramming in Atherosclerosis

Advanced Science, EarlyView.
Here we developed Am@SExo, a dual‐functional engineered exosome that coordinates CD47–SIRPα checkpoint blockade with Arg1 mRNA–mediated metabolic reprogramming. Surface SIRPα promotes recognition of apoptotic cells and plaque targeting, while Arg1 expression enhances arginine–ornithine metabolism, Rac1 activation, and actin remodeling in macrophages ...
Danwen Zheng, Shiteng Cai, Jinfeng Gao, Sheng Zhao, Bohan Wei, Xueyi Weng, Zhengmin Wang, Qiaozi Wang, Qiyu Li, Chengzhi Han, Weiyan Li, Yiwen Tan, Yuyuan Fu, Meng Ji, Zheyong Huang, Yanan Song, Juying Qian, Junbo Ge   +17 more
wiley   +1 more source

Cholesterol Ester Storage Disease in Two Field Spaniels With Lysosomal Acid Lipase Deficiency

Journal of Veterinary Internal Medicine
Cholesterol ester storage disease (CESD) is a rare genetic lysosomal storage disorder resulting from lower lysosomal acid lipase (LAL) activity. LAL is an essential enzyme required in intracellular lipid metabolism, and deficiency results in disability ...
Pernilla Syrjä, Mathilda M. H. Ylenius, Matilda Kråkström, Alex M. Dickens, Elina Rautala, Anna Huupponen, Anders Eriksson, Sanna J. Viitanen   +7 more
doaj   +1 more source

A Wireless 3D Magneto‐Mechanical Stimulation Platform Drives In Situ Chondrogenic Commitment of Endogenous MSCs

Advanced Science, EarlyView.
An intracellular magneto‐mechanical platform utilizing MSC‐targeted nanomotors is developed. After intra‐articular delivery, these nanomotors target endogenous BMSCs and are actuated within lysosomes to execute trans‐planar rotational‐bouncing motions under a rotating‐fluctuating 3D magnetic field, generating amplified mechanical stimulation.
Zhenguang Li, Li Peng, Laiya Lu, Yingying Wang, Xiaolei Chen, Feng Yin, Cuijun Deng, Yu Cheng   +7 more
wiley   +1 more source

Gene therapy in Anderson-Fabry disease. State of the art and future perspectives

Cardiogenetics, 2020
Anderson-Fabry disease (AFD) is an X-linked lysosomal storage disorder caused by a deficiency of the lysosomal enzyme, agalactosidase A. The inadequate enzymatic activity leads to systemic storage of glycosphingolipids, mostly globotriaosylceramide, in ...
Giorgio Spiniello, Federica Verrillo, Riccardo Ricciolino, Dario Prozzo, Andrea Tuccillo, Martina Caiazza, Marta Rubino   +6 more
doaj   +1 more source

Designer Dynamic DNA Nanoaggregate in Living Cell for Mitochondrial Energy Restriction

Advanced Science, EarlyView.
This study presents the Tech‐tetrahedron, a designer dynamic DNA nanoaggregate engineered for precise mitochondrial energy restriction. Its trinity‐functionalized design integrates navigable unit, telomerase‐activated latch, and self‐assembly module.
Ruijia Deng, Jing Sheng, Ben Niu, Wenjuan Fu, Yingjie Yang, Zuowei Xie, Shuang Xie, Yunxuan He, Meilin Gong, Jue Wang, Yan Pi, Ming Chen, Kai Chang   +12 more
wiley   +1 more source

Decreased bone formation and increased osteoclastogenesis cause bone loss in mucolipidosis II

EMBO Molecular Medicine, 2013
Mucolipidosis type II (MLII) is a severe multi‐systemic genetic disorder caused by missorting of lysosomal proteins and the subsequent lysosomal storage of undegraded macromolecules.
Katrin Kollmann, Jan Malte Pestka, Sonja Christin Kühn, Elisabeth Schöne, Michaela Schweizer, Kathrin Karkmann, Takanobu Otomo, Philip Catala‐Lehnen, Antonio Virgilio Failla, Robert Percy Marshall, Matthias Krause, Rene Santer, Michael Amling, Thomas Braulke, Thorsten Schinke   +14 more
doaj   +1 more source