Results 151 to 160 of about 156,351 (339)

BK Channels Alleviate Lysosomal Storage Diseases by Providing Positive Feedback Regulation of Lysosomal Ca2+ Release [PDF]

, 2016
Xian‐Ping Dong
openalex   +1 more source

GENETIC STUDY OF PATIENT WITH THE DIAGNOSIS OF NIEMANNPICK LYSOSOMAL STORAGE DISEASE [PDF]

, 2022
G.B. Latifova, A.K. Mammаdbayli, E.M. Rasulov   +2 more
openalex   +1 more source

Inhibition of SLC11A1‐Mediated Lysosomal Iron Accumulation in Microglia Promotes Repair Following White Matter Stroke

Advanced Science, EarlyView.
Genetic and pharmacological inhibition of SLC11A1 functioning as an H+/Fe2+ antiporter–mediated lysosomal iron accumulation in microglia promotes lysosomal lumen acidification, increases CTSD expression, enhances lysosomal myelin debris uptake and degradation, and promotes repair following white matter stroke. ABSTRACT White matter stroke (WMS) results
Lingling Qiu, Yajie Zhang, Yushi Tang, Hongli Hu, Ying Zhang, Junwen Xue, Hao Wang, Yecheng Wang, Chunfeng Liu, Jia Jia, Jian Cheng, Yongjun Cao   +11 more
wiley   +1 more source

Abstract 1907: Delivery of Apolipoprotein E3 Reconstituted High-Density Lipoprotein Nanodiscs for the Treatment of Lysosomal Storage Diseases

, 2023
Christy Nguyen, Vidya Metkar, Vasanthy Narayanaswami   +2 more
openalex   +1 more source

Lipid Droplet‐Localized Spindle Apparatus Coiled‐Coil Protein 1 Regulates Lipid Droplet Distribution

Advanced Science, EarlyView.
SPDL1‐L, a long isoform of SPDL1, is identified as a bona fide lipid droplet‐associated protein that acts as a dynein adaptor to drive perinuclear clustering of lipid droplets. By linking lipid droplets to the microtubule network, SPDL1‐L extends the function of SPDL1 beyond mitosis and contributes to lipid droplet organization and nuclear remodeling ...
Honggang Su, Huimin Pan, Yaqiang Liu, Wei Wang, Yang Jiao, Yumo Hang, Xiahe Huang, Yong E. Zhang, Yuhang Chen, Mei Ding, Xun Huang   +10 more
wiley   +1 more source

Lysosomal Delivery of Bioactive Proteins to Living Human Cells via Engineered Exosomes [PDF]

, 2018
Exosomes are naturally secreted nanovesicles derived from mammalian cells that are used for intercellular communication in vivo. As a result, they can potentially be used for intracellular delivery of therapeutics for disease treatment. We have developed
Levy, Daniel
core   +1 more source

Beyond Catalytic Therapy: Copper‐Paeonol Nanozymes Disrupt Fascin‐Mediated Actin Bundling to Suppress Tumor Growth and Metastasis

Advanced Science, EarlyView.
Copper‐paeonol nanozymes target tumor‐specific reactive oxygen species generation and disrupt fascin‐mediated actin bundling, effectively suppressing tumor growth and metastatic colonization. Abstract Fascin, an actin‐bundling protein universally upregulated in metastatic tumors, drives tumor migration and invasion by promoting filopodia and ...
Peiying Zhang, Huajun Li, Yisen Wang, Jie Xiang, Lei Fan, Shengzhe Zhang, Lizeng Gao, Hua Dai, Juqun Xi   +8 more
wiley   +1 more source

Effect of disease progression on CSF-directed AAV gene therapy in a large brain animal model of lysosomal storage disease

Molecular Therapy: Methods & Clinical Development
The lysosomal storage disease alpha-mannosidosis (AMD) is caused by a genetic deficiency of lysosomal alpha-mannosidase, leading to the widespread presence of storage lesions in the brain and other tissues.
Jacqueline E. Hunter, Caitlyn M. Molony, Wojciech K. Panek, Charles H. Vite, Sanjeev Chawla, Harish Poptani, John H. Wolfe   +6 more
doaj   +1 more source

Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood-onset beta-mannosidosis. [PDF]

, 2019
BACKGROUND: Deficiency in the enzyme β-mannosidase was described over three decades ago. Although rare in occurrence, the presentation of childhood-onset β-mannosidase deficiency consists of hypotonia in the newborn period followed by global development ...
Eisengart, Julie B., Lund, Troy C., Miller, Weston P., Orchard, Paul J., Patterson, Marc C., Pollard, Laura, Renaud, Deborah L., Simmons, Katrina, Wenger, David A.   +8 more
core   +1 more source

TriCON: A Carbon‐Based Triple‐Modal Nanoplatform for Pancreatic Cancer Therapy

Advanced Science, EarlyView.
We developed TriCON, a triple‐modality nanotherapeutic platform, to treat pancreatic ductal adenocarcinoma (PDAC) by synergizing gene editing, chemotherapy, and immunotherapy. TriCON utilizes CRISPR/Cas9 to target the poliovirus receptor (PVR), combined with nano‐encapsulated doxorubicin and checkpoint blockade. This approach achieved significant tumor
Xinyu Peng, Jiaxing Huang, Shengnan Lv, Jian Zhang, Liangliang Zhang, Huan Liu, Yan Liu, Zhen‐An Qiao, Feng Wei, Bao‐Lian Su   +9 more
wiley   +1 more source