Results 161 to 170 of about 39,202 (243)

Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach. [PDF]

JIMD Rep
Corazolla EM, Langeveld M, Brands MMMG, Sjouke B, Hollak CEM.   +4 more
europepmc   +1 more source

[Lysosomes and lysosomal storage diseases].

Tidsskrift for den Norske laegeforening : tidsskrift for praktisk medicin, ny raekke, 1975
K K, Lie, S O, Lie
openaire   +1 more source

Low‐Density Lipoprotein Receptor‐Targeted Biomimetic Nanoplatform Suppresses Glioblastoma Stemness and Remodels Antitumor Immunity via Dual Metabolic Disruption

Advanced Science, EarlyView.
A low‐density lipoprotein receptor‐oriented biomimetic nanoplatform couples brain‐tumor delivery with a mechanistically matched regorafenib‐erythrodiol combination. Concurrent disruption of autophagic nutrient salvage and cholesterol homeostasis collapses bioenergetic and signaling support, suppresses glioblastoma stemness, and promotes immunogenic ...
Yining Jiang, Wenzhuo Yang, Wenting Cheng, Jing Zuo, Lin Pan, Sheng Zhong, Ren Geng, Yao Zhao, Zijian Chen, Canhua Huang, Yang Bai, Liyan Zhao, Yunqian Li   +12 more
wiley   +1 more source

Chlorination‐Driven BODIPY Fluorescent Probes for the Selective Monitoring of Myeloperoxidase Activity in Cells and Inflamed Mouse Models

Angewandte Chemie, EarlyView.
A meso‐carboxamide‐substituted BODIPY fluorescent probe reports myeloperoxidase (MPO) activity via electrophilic chlorination by MPO‐derived HOCl, generating a highly emissive chlorinated product with a ca. 40 nm bathochromic shift. Applications in quantitative MPO assays, cellular imaging of MPO‐derived HOCl, redox‐dependent discrimination of cancer ...
Siyoung Cho, Yongyang Luo, Jeehyeon Bae, Youngmi Kim   +3 more
wiley   +2 more sources

Lysosomal storage diseases in North America: a comprehensive review of enzyme therapies and unmet needs. [PDF]

Ther Adv Rare Dis
Bennett LL.
europepmc   +1 more source

Age‐Related Lamin B1 Deficiency Activates SLC7A11‐Dependent Disulfidptosis to Impair Osteogenesis via Intercepting Mitochondrial Fission

Advanced Science, EarlyView.
This study reveals that age‐related LMNB1 deficiency in mesenchymal stem cells epigenetically upregulates SLC7A11, triggering actin disulfidptosis and mitochondrial dysfunction. This cascade impairs bone formation in senile osteoporosis. Furthermore, researchers identified the FDA‐approved drug naldemedine as a targeted SLC7A11 inhibitor to reverse ...
Wenhui Yu, Qibo Li, Weihao Zhang, Guan Zheng, Junhua Chen, Yipeng Zeng, Zipeng Xiao, Zibin Chen, Ziqian Liu, Yangfeng Lin, Jiajie Lin, Zepeng Su, Zhongyu Xie   +12 more
wiley   +1 more source

c-Abl/TFEB Pathway Activation as a Common Pathogenic Mechanism in Lysosomal Storage Diseases: Therapeutic Potential of c-Abl Inhibitors. [PDF]

Antioxidants (Basel)
Guerra MV, Castro J, Moreno A, Balboa E, Marugan JJ, Alvarez AR, Zanlungo S.   +6 more
europepmc   +1 more source

The emerging role and clinicopathological significance of MFSD12 in cancer and lysosomal storage diseases. [PDF]

Front Pharmacol
Ding L.
europepmc   +1 more source

Differential Diagnosis of Mucopolysaccharidoses Types I, II, III, IVA, and VI Through Analysis of Leukocytic Inclusions

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Mucopolysaccharidoses (MPS) are lysosomal storage disorders characterized by the accumulation of glycosaminoglycans (GAGs), which can lead to cytoplasmic alterations in leukocytes. The objective of this study was to characterize leukocyte inclusions in patients with different types of MPS and assess their diagnostic relevance.
Márcio A. W. Melo, Marcelo S. Kertenetzky, Cristina M. Silveira, Maíra M. Ribeiro, Paula F. Medeiros, Gabriela S. Arcanjo   +5 more
wiley   +1 more source

Hepatomegaly and Splenomegaly: An Approach to the Diagnosis of Lysosomal Storage Diseases. [PDF]

J Clin Med
Jerves Serrano T, Gold J, Cooper JA, Church HJ, Tylee KL, Wu HY, Kim SY, Stepien KM.   +7 more
europepmc   +1 more source