Results 101 to 110 of about 123,112 (260)

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

Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction.

Molecular Therapy, 2002
Lysosomal storage diseases are an intriguing target for gene therapy approaches, as transduction of a "depot" organ with a transgene encoding a lysosomal enzyme can be followed by secretion, systemic distribution, downstream uptake, and lysosomal ...
E. Ding, Hui-jing Hu, B. Hodges, F. Migone, D. Serra, Fang Xu, Yuan-Tsong Chen, A. Amalfitano   +7 more
semanticscholar   +1 more source

ARL3 Enhances ERα Stability via USP10 Deubiquitination to Promote Endocrine Resistance and Drive Mitochondrial Metabolic Reprogramming in HR+ Breast Cancer

Advanced Science, EarlyView.
This study identifies ADP‐ribosylation factor‐like protein 3 (ARL3) as a novel chaperone that stabilizes estrogen receptor α (ERα) in breast cancer. ARL3 promotes tumor growth by recruiting USP10 to remove ubiquitin chains from ERα, preventing its degradation.
Han Li, Yang Liu, Zehao Cai, Kang Li, Shun Gao, Ailin Lan, Dan Shu, Kuan He, Xin Liu, Yang Peng, Shipeng Guo, Haochen Yu, Aishun Jin, Meiying Shen, Shengchun Liu   +14 more
wiley   +1 more source

Extracellular origin of the lipid lysosomal storage in cultured fibroblasts from Wolman's disease [PDF]

, 1987
Robert Salvayre, Anne Nègre, Arlette Maret, Jean Radom, Louis Douste‐Blazy   +4 more
openalex   +1 more source

K29‐Linked Ubiquitination of Transcription Regulators Controls Cell Proliferation in the Unfolded Protein Response

Advanced Science, EarlyView.
Under endoplasmic reticulum stress, cells activate the unfolded protein response (UPR). During the UPR, the cohesin complex at the promoter of cell proliferation‐related genes undergoes K29‐linked ubiquitination, recruiting the cohesin release factor WAPL to release cohesin, leading to transcriptional downregulation and inhibition of cell growth ...
Qiushuang Zhang, Xucong Teng, Yicong Dai, Yuncong Wu, Hongwei Hou, Jinghong Li   +5 more
wiley   +1 more source

Chitotriosidase activity as additional biomarker in the diagnosis of lysosomal storage diseases [PDF]

, 2016
N. V. Olkhovych
openalex   +1 more source

A Novel FGFR3‐Targeting Antibody‐Drug Conjugate Induces Tumor Cell Apoptosis through the cGAS–STING Pathway in Bladder Cancer

Advanced Science, EarlyView.
LZU‐WZLYCS01 is a novel FGFR3‐targeting ADC for bladder cancer with 7‐ethyl‐9‐fluorocamptothecin (A2) as its cytotoxic payload. LZU‐WZLYCS01 intracellularly releases A2, which targets MAD2L1 to activate the cGAS‐STING pathway and induce tumor cell apoptosis.
Shu Cui, Xiongfei Luo, Guangrui Fan, Jingqi Jiang, Yingru Wang, Enguang Yang, Jinpeng Ma, Ze Zhang, Yuhan Wang, Juan Wang, Dengtuo Wang, Hanzhang Wang, Liang Cheng, Junqiang Tian, Zhilong Dong, Yingqian Liu, Zhiping Wang   +16 more
wiley   +1 more source

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  

Lactate Metabolic Reprogramming Mediated by CircRNA–LDHA Complex Facilitates Innate Immune Evasion of Liver Cancer

Advanced Science, EarlyView.
CircSMPD4 physically combines with lactate dehydrogenase A (LDHA) and reduces its acetylation level via SIRT2‐dependent deacetylation, thereby inhibiting chaperone‐mediated autophagy‐lysosome degradation of LDHA. Upregulating LDHA leads to increasing lactate production and extracellular export, which in turn suppresses natural killer cell cytotoxicity ...
Hao Shen, Boqiang Liu, Jing He, Weijun Zhao, Weiqi Li, Lingfeng Ma, Lidan Hou, Yi Wang, Chenqi Jin, Yushun Chang, Jie Lin, Jia Zhao, Binghan Jin, Yuanshi Tian, Xiujun Cai, Liang Shi, Yifan Wang   +16 more
wiley   +1 more source

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