Results 121 to 130 of about 238,474 (313)
BiomedicinesBackground: The multisystem manifestations of Fabry disease can create major challenges in patient care. Although enzyme replacement therapy with recombinant agalsidase beta has demonstrated clinical benefits, the standard fortnightly, multi-hour ...
Dominique P. Germain +6 more
doaj +1 more source
Lysosomal and Mitochondrial Liaisons in Niemann-Pick Disease
Frontiers in Physiology, 2017 Lysosomal storage disorders (LSD) are characterized by the accumulation of diverse lipid species in lysosomes. Niemann-Pick type A/B (NPA/B) and type C diseases Niemann-Pick type C (NPC) are progressive LSD caused by loss of function of distinct ...
Sandra Torres +10 more
doaj +1 more source
Advanced Science, EarlyView.Chronic septic arthritis involves intracellular bacterial persistence and lipid‐immune crosstalk via the PGRN‐BMP lysosomal axis. A dual‐targeting nanoparticle system (NPs@PGRN) restores lysosomal bactericidal function, reduces bacterial burden, and reprograms macrophage immunity, offering a novel therapeutic strategy. ABSTRACT Chronic septic arthritis,
Congsun Li +12 more
wiley +1 more source
DCAF13 Safeguards Hematopoietic Stem Cells via RRS1‐Regulated Ribosome Biogenesis
Advanced Science, EarlyView.This study establishes DCAF13 as an essential regulator for hematopoietic stem cell (HSC) function. Its deletion in mice causes lethal pancytopenia and HSC depletion. Mechanistically, DCAF13 interacts with RRS1 and mediates its non‐degradative K27‐linked ubiquitination, thereby stabilizing RRS1 to maintain ribosome biogenesis and protein translation ...
Mengke Li +25 more
wiley +1 more source
CellsNeurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease, are characterized by progressive neuronal loss, leading to cognitive and motor impairments.
Jessica Tittelmeier +1 more
doaj +1 more source
The Aging Heart: Mitophagy at the Center of Rejuvenation. [PDF]
, 2020 Aging is associated with structural and functional changes in the heart and is a major risk factor in developing cardiovascular disease. Many recent studies have focused on increasing our understanding of the basis of aging at the cellular and molecular ...
Gustafsson, Åsa B, Liang, Wenjing J
core +1 more source
Advanced Science, EarlyView.Antibody–drug conjugates (ADCs) transform breast cancer therapy, yet resistance limits their durability. Emerging evidence reveals that ADC failure is not solely tumor‐intrinsic but shaped by dynamic tumor–microenvironment interactions that alter drug delivery, processing, and response.
Minji Seo, Jangsoon Lee, Naoto T. Ueno
wiley +1 more source
Halofuginone is a Molecular Glue Degrader of Integrin β4
Advanced Science, EarlyView.Integrin β4 overexpression is linked to aggressive tumors and poor prognosis, but targeted therapies are lacking. We identified halofuginone (HF) as a molecular glue degrader that promotes integrin β4 degradation via the CRL4BWDR18 E3 ligase. HF suppresses tumor progression in vitro and in vivo, offering a new strategy for targeting oncogenic ...
Wei Gong +8 more
wiley +1 more source
Mechanism of Secondary Ganglioside and Lipid Accumulation in Lysosomal Disease. [PDF]
Int J Mol Sci, 2020 Breiden B, Sandhoff K.
europepmc +1 more source
Polyglutamine-expanded androgen receptor interferes with TFEB to elicit autophagy defects in SBMA. [PDF]
, 2014 Macroautophagy (hereafter autophagy) is a key pathway in neurodegeneration. Despite protective actions, autophagy may contribute to neuron demise when dysregulated.
Batlevi, Yakup +11 more
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