Results 101 to 110 of about 42,118 (292)
Orthopedic management of the extremities in patients with Morquio A syndrome. [PDF]
, 2014 BackgroundMusculoskeletal involvement in Morquio A syndrome (mucopolysaccharidosis IVA; MPS IVA) contributes significantly to morbidity and mortality. While the spinal manifestations of the disorder have received considerable attention in the literature,
Bache, C Edward +6 more
core +1 more source
Advanced Science, EarlyView.This study investigates the impact of various post‐processing methods (filtration, sonication, dialysis, and heating) on lipid nanoparticles produced using microfluidics. The nanoparticles are designed for efficient delivery of miRNA into cells. The work highlights how processing conditions influence nanoparticle stability and quality, providing ...
Alicja Kosik‐Kozioł +8 more
wiley +1 more source
Tau is central in the genetic Alzheimer-frontotemporal dementia spectrum [PDF]
, 2005 In contrast to the common and genetically complex senile form of Alzheimer's disease (AD), the molecular genetic dissection of inherited presenile dementias has given important mechanistic insights into the pathogenesis of degenerative brain disease ...
Cruts, Marc +5 more
core +1 more source
Integrative Approaches to Treating Cellular Senescence in Kidney Disease
Advanced Science, EarlyView.ABSTRACT Cellular senescence in the kidney plays a crucial role in the progression of acute kidney injury and chronic kidney disease. Therapeutic approaches targeting senescent cells, such as small molecule senolytic and senomorphic drugs, display efficacy in preclinical models.
Tomoka Misawa +3 more
wiley +1 more source
The Journal of Clinical InvestigationLysosome storage dysfunction plays a central role in numerous human diseases, but a lack of appropriate tools has hindered lysosomal content profiling in clinical settings. In this issue of the JCI, Saarela et al. introduce a method called tagless LysoIP
Ali Shilatifard, Issam Ben-Sahra
doaj +1 more source
Impact of ER stress and the unfolded protein response on Fabry disease
EBioMedicineSummary: Fabry disease (FD) is a lysosomal storage disorder caused by pathogenic missense and nonsense variants in the α-galactosidase A (GLA) gene, leading to absent or reduced enzyme activity.
Malte Lenders, Elisa Rudolph, Eva Brand
doaj +1 more source
Glycogenosis type II : a study on clinical heterogeneity and enzyme replacement therapy [PDF]
, 1989 Glycogenosis type II is a lysosomal storage disorder caused by deficiency of acid a-glucosidase and characterized by heart failure and skeletal muscle weakness.
Ploeg, A.T. (Ans) van der
core
Advanced Science, EarlyView.This study reveals that chronic alcohol exposure selectively upregulates ACSL1 expression in prefrontal cortical microglia, driving lipid metabolism reprogramming and lipid droplet accumulation, which activates the NLRP3 inflammasome and sustains neuroinflammation. To reverse this process, a dual‐targeted lipid nanoparticle, siACSL1@LNP‐MR, is designed
Liang Hao +8 more
wiley +1 more source
T Cell Exhaustion in Cancer Immunotherapy: Heterogeneity, Mechanisms, and Therapeutic Opportunities
Advanced Science, EarlyView.T cell exhaustion limits immunotherapy efficacy. This article delineates its progression from stem‐like to terminally exhausted states, governed by persistent antigen, transcription factors, epigenetics, and metabolism. It maps the exhaustion landscape in the TME and proposes integrated reversal strategies, providing a translational roadmap to overcome
Yang Yu +7 more
wiley +1 more source
Lipids in Health and Disease, 2006 Summary Background Arylsulfatase A (ASA)-deficient mice are a model for the lysosomal storage disorder metachromatic leukodystrophy. This lipidosis is characterised by the lysosomal accumulation of the sphingolipid sulfatide.
De Deyn PP +7 more
doaj +1 more source