Results 121 to 130 of about 84,505 (291)

Galactosialidosis in two siblings

The Turkish Journal of Pediatrics, 1996
Galactosialidosis is a rare lysosomal storage disease associated with deficiencies of alpha-galactosidase and beta-neurominidase. In this report, two siblings with galactosialidosis, resembling Niemann-Pick disease with the presence of foamy cells
A Yuce, N Kocak, G T Besley
doaj  

Nanoreporter Identifies Lysosomal Storage Disease Lipid Accumulation Intracranially. [PDF]

Nano Lett, 2023
Antman-Passig M, Yaari Z, Goerzen D, Parikh R, Chatman S, Komer LE, Chen C, Grabarnik E, Mathieu M, Haimovitz-Friedman A, Heller DA.   +10 more
europepmc   +1 more source

Dynamic Shifts in ER–Plasma Membrane Junctions Signaling Define Pro‐Metastatic N‐Glycosylation and Predict Prostate Cancer Progression

Advanced Science, EarlyView.
Prostate cancer remains a leading cause of male cancer death, yet screening cannot reliably identify aggressive disease, underscoring the need for tissue biomarkers. It is shown that primary tumors increase ER–plasma membrane junction signaling via STIM1/ORP5, whereas metastasis features their loss, Golgi dispersal, and rapid conversion of high‐mannose
Amanda J. Macke, Tania Kamal, Taylor E. Divita, Artem N. Pachikov, Chad A. LaGrange, Rajesh Ravichandran, Martha Morton, Robert Powers, Haowen Qiu, Jean‐Jack M. Riethoven, Colm Morrissey, Melinda Wojtkiewicz, Rebekah L. Gundry, Carol A. Casey, Armen Petrosyan   +14 more
wiley   +1 more source

A Nanobody‐LNP Platform for Targeting and Relicensing Dendritic Cells for Potent Cancer Immunotherapy

Advanced Science, EarlyView.
Plastin‐2 (PLS2) is identified as a dual‐function receptor on DCs that mediates both nanoparticle uptake and immunomodulation. A nanobody‐LNP platform is engineered to integrate antigen delivery with relicensing DCs. The therapeutic strategy elicits potent anti‐tumor T cell responses and leads to significant inhibition of established tumors in vivo ...
Shugang Qin, Zhiying Huang, Hai Huang, Yupei Zhang, Yuting Chen, Xi He, Zhenyi Niu, Zhongzheng Xiang, Chaoyu Zou, Xiangrong Song   +9 more
wiley   +1 more source

The Trichinella Super‐Pangenome Reveals the Evolution of Encapsulation and Predicted Host–Parasite Protein Interactions

Advanced Science, EarlyView.
ABSTRACT The muscle capsule of Trichinella is a critical structure that impedes immune attacks and drug penetration, yet the molecular mechanisms underlying its formation remain poorly understood. Using a high‐quality super‐pangenome comprising 12 Trichinella species, we compared extensive genomic variations between encapsulating and non‐encapsulating ...
Qingbo Lv, Yi Liu, Ning Xu, Xiao Zhang, Yaming Yang, Chengyao Li, Hanhai Mao, Mingyuan Liu, Xiaolei Liu   +8 more
wiley   +1 more source

Brain and Liver Dual‐Targeting Oridonin Nanoparticles to Enhance Aβ Clearance for Alzheimer's Disease Therapy

Advanced Science, EarlyView.
We developed a nanoparticle named OAF, which simultaneously targeted to both the brain and liver via the transferrin receptor 1 (TfR1) receptor, promoting lipoprotein receptor‐related protein 1 (LRP1) expression to enhance amyloid‐beta (Aβ) clearance. In AD mice model, OAF significantly reduced Aβ deposition and cognitive impairment, while a mitigating
Wenshuai Gong, Wenting Hui, Sai Qiao, Qifeng Ji, Miao Liu, Bangle Zhang, Daozhou Liu, Yumei Wu, Siyuan Zhou   +8 more
wiley   +1 more source

Microglia‐Targeted Biomimetic Tetrahedral Framework Nucleic Acid Nanovesicles for Synergistic Treatment of Sepsis‐Associated Encephalopathy

Advanced Science, EarlyView.
Sepsis‐associated encephalopathy (SAE) lacks effective therapies. We developed ME@FDsi, a biomimetic nanodrug using a tetrahedral framework nucleic acid to deliver disulfiram and siTNFα. It crosses the blood‐brain barrier, targets M1 microglia, inhibits pyroptosis and inflammation, and scavenges ROS.
Huimin Shi, Qiuxia Gao, Wenying Wang, Bin Li, Yukun Chen, Zhijun Yao, Yujie Li, Junrui Li, Na Li, Gong Gu, Zhimin Hou, Mengyuan Yang, Ruilin Zhang, Hongju Yang, Yuhui Liao, Hongyi Lei   +15 more
wiley   +1 more source

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease. [PDF]

Mol Ther, 2022
Monaco A, Maffia V, Sorrentino NC, Sambri I, Ezhova Y, Giuliano T, Cacace V, Nusco E, De Risi M, De Leonibus E, Schrader T, Klärner FG, Bitan G, Fraldi A.   +13 more
europepmc   +1 more source

Tumor‐Induced Splenic Remodeling: Mechanisms of Systemic Immunosuppression and Emerging Therapeutic Opportunities

Advanced Science, EarlyView.
ABSTRACT Despite the transformative impact of cancer immunotherapies such as immune checkpoint blockade, durable clinical responses remain limited. Increasing evidence indicates that antitumor immunity is governed not only by the tumor microenvironment, but also by systemic immune regulation mediated by peripheral immune organs. Among these, the spleen
Yuehua Liu, Xiaoqian Nie, Xiaofei Gao
wiley   +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