Results 131 to 140 of about 2,496,873 (280)

Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal. [PDF]

Mol Ther Methods Clin Dev, 2021
Marcó S, Haurigot V, Jaén ML, Ribera A, Sánchez V, Molas M, Garcia M, León X, Roca C, Sánchez X, Bertolin J, Pérez J, Elias G, Navarro M, Carretero A, Pumarola M, Andaluz A, Espada Y, Añor S, Bosch F.   +19 more
europepmc   +1 more source

Beyond Biomimetics: Pathology‐Informed Engineering Rescues Reconstituted HDL From Inflammatory Dysfunction for Sepsis Immunotherapy

Advanced Science, EarlyView.
A pathology‐informed engineering strategy rescues reconstituted HDL from inflammatory dysfunction in sepsis. By integrating a ceria nanozyme‐celastrol coordination complex, the platform preserves HDL structure and endotoxin handling, sustains hierarchical intracellular delivery, and enhances immunoregulation across pathological models, translating ...
Han Zhou, Lei Zhang, Xingyu Cai, Jiajia Su, Guangyu Dai, Xue Chen, Hanming Wang, Jia Hua, Jianping Liu, Chunmeng Sun, Xiancheng Chen, Erhao Wang, Wenli Zhang   +12 more
wiley   +1 more source

Lysosomal storage disease 2 - Pompe's disease

, 2008
Pompe's disease, glycogen-storage disease type II, and acid maltase deficiency are alternative names for the same metabolic disorder. It is a pan-ethnic autosomal recessive trait characterised by acid alpha-glucosidase deficiency leading to lysosomal ...
van der Ploeg, Ans T., Reuser, Arnold J. J.   +1 more
core   +1 more source

Reactivation of mTOR signaling slows neurodegeneration in a lysosomal sphingolipid storage disease

Neurobiology of Disease
Sandhoff disease, a lysosomal storage disorder, is caused by pathogenic variants in the HEXB gene, resulting in the loss of β-hexosaminidase activity and accumulation of sphingolipids including GM2 ganglioside.
Hongling Zhu, Y. Terry Lee, Colleen Byrnes, Jabili Angina, Danielle A. Springer, Galina Tuymetova, Mari Kono, Cynthia J. Tifft, Richard L. Proia   +8 more
doaj   +1 more source

Spatial Transcriptomics Reveals a T Cell‐Mediated Microglial Activation Axis of Immune Checkpoint Inhibition‐Related Adverse Events in the Brain

Advanced Science, EarlyView.
Immune checkpoint inhibition (ICI) reshapes the hippocampal neuroimmune niche. Spatial transcriptomic profiling and human brain histological analyses identify neuroinflammation and altered neuronal and glial programs. Notably, T cell depletion prevents ICI‐driven microglial activation, establishing the T cell–microglia crosstalk axis as a driving ...
Devyani Swami, Sanad M. El‐Khatib, Suhas Sureshchandra, Elise Nguyen, Janaki Manoja Vinnakota, Robert Zeiser, Shivashankar Othy, Munjal M. Acharya   +7 more
wiley   +1 more source

Storage problems in lysosomal diseases

, 2010
Biochemical disorders in lysosomal storage diseases consist of the interruption of metabolic pathways involved in the recycling of the degradation products of one or several types of macromolecules.
Stéphanie Bigou, Jean Michel Heard, Sandrine Vitry, Jérôme Ausseil, Julie Bruyère, Elise Roy   +5 more
core   +1 more source

Magnetically Guided Macrophage Immunobots Coordinate Iron‐Metabolic Cascades and Immunogenic Ferroptosis for Tumor Immunotherapy

Advanced Science, EarlyView.
Magnetically guided macrophage immunobots integrate active tumor targeting with immunometabolic therapy. After systemic administration, magnetic actuation enhances immunobot retention and tumor penetration, while LPS‐functionalized FePt microrollers sustain M1‐like polarization through inflammatory and iron‐amplified oxidative signaling and trigger ...
Xirui Zeng, Yingfei Zhang, Chong Wu, Qijia Duan, Mengqi Fan, Kuan Wang, Shirong Cai, Immihan Ceren Yasa   +7 more
wiley   +1 more source

Developmental Environmental Chronotoxicity by Polystyrene Nanoplastics Exacerbates High‐Fat Diet‐Induced Pediatric MAFLD Through Circadian Disruption and Mitochondrial Dysfunction

Advanced Science, EarlyView.
Polystyrene nanoplastics exacerbate high‐fat diet‐induced metabolic dysfunction in an age‐dependent manner. Juvenile mice exhibit greater susceptibility, characterized by circadian disruption, impaired mitochondrial function, reduced energy metabolism, and enhanced hepatic lipid accumulation.
Peihao Xu, Shuting Huang, Leilei Zhu, Junyi Huang, Kewei Li, Guangming Li, Jiannong Wu, Mingyuan Zhou, Fangmei Zhou, Zhishan Ding, Yingzhi Shen   +10 more
wiley   +1 more source

Profiling glycosphingolipid changes in mouse and human cellular models of lysosomal free sialic acid storage disorder

Free sialic acid storage disorder (FSASD) is an autosomal recessive lysosomal storage disease caused by biallelic pathogenic variants in SLC17A5, which encodes the lysosomal sialic acid transporter, sialin.
Platt, Frances, Sabir, Marya, Rha, AK, Dobrenis, K   +3 more
core   +2 more sources

Restores Aβ Clearance by Overcoming PCSK9–LRP1 Dysregulation and TRIB3‐Mediated Autophagy Blockade in Alzheimer's Disease

Advanced Science, EarlyView.
PCSK9 upregulation in cerebrovascular endothelial cells and microglia destabilizes the Aβ transporter LRP1, while TRIB3‐associated autophagy blockade further limits intracellular Aβ degradation. A RAP‐modified tea‐polyphenol nanoparticle co‐delivers siPCSK9 and siTRIB3 to restore LRP1‐mediated Aβ efflux and autophagy‐dependent degradation, reducing Aβ ...
Jie Miao, Jing Wang, Yankun Li, Haipei Zhang, Yanli Zhang, Qingnian Li, Bo Xiao, Wenhu Zhou, Junhong Guo   +8 more
wiley   +1 more source