Results 71 to 80 of about 42,118 (292)
Fostering collaborative research for rare genetic disease: The example of Niemann-Pick type C disease [PDF]
, 2016 Rare disease represents one of the most significant issues facing the medical community and health care providers worldwide, yet the majority of these disorders never emerge from their obscurity, drawing little attention from the medical community or the
Austin, Christopher P +8 more
core +2 more sources
Rethinking Extracellular Vesicle Signaling
Advanced Materials, EarlyView.Extracellular vesicles enable cell communication beyond intracellular cargo delivery. This perspective highlights two plausible surface‐based signaling modes: “bind‐and‐stay” and “bind‐and‐leave.” Transient binding to multiple cells challenges the one‐vesicle‐one‐cell model.
Wojciech Chrzanowski, Joy Wolfram
wiley +1 more source
Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population. [PDF]
, 2019 Mucopolysaccharidosis (MPS) VII is an ultra-rare, progressively debilitating, life-threatening lysosomal disease caused by deficiency of the enzyme, β-glucuronidase. Vestronidase alfa is an approved enzyme replacement therapy for MPS VII. UX003-CL301 was
Bauer, Mislen +7 more
core +1 more source
Advanced Materials Interfaces, EarlyView.Lipid drug carriers to which ultra‐small gold nanoparticles were added enabled more efficient radiotherapy of cultured pancreatic cancer tumors. These nanoparticles boosted radiation‐induced damage to tumors by generating more reactive molecules, though higher gold levels are needed for strong benefits.
Nazareth Milagros Carigga Gutierrez +17 more
wiley +1 more source
JCI Insight, 2020 Although congenital heart defects (CHDs) represent the most common birth defect, a comprehensive understanding of disease etiology remains unknown.
Po-Nien Lu +5 more
doaj +1 more source
Advanced Robotics Research, EarlyView.An introduction for multidrive and environment‐adaptive micro/nanorobotics: design and fabrication strategies, intelligent actuation, and their applications. Various intelligent actuation approaches—magnetic, acoustic, optical, chemical, and biological—can be synergistically designed to enhance flexibility and adaptive behavior for precision medicine ...
Aiqing Ma +10 more
wiley +1 more source
Macrophage polarization impacts tunneling nanotube formation and intercellular organelle trafficking. [PDF]
, 2019 Tunneling nanotubes (TNTs) are cellular extensions enabling cytosol-to-cytosol intercellular interaction between numerous cell types including macrophages.
Cherqui, Stephanie +4 more
core +1 more source
Linear cyclodextrin polymer prodrugs as novel yherapeutics for Niemann-Pick type C1 disorder [PDF]
, 2018 Niemann-Pick Type C1 disorder (NPC) is a rare lysosomal storage disease characterized by the accumulation of cholesterol in lysosomes. NPC has no FDA approved treatments yet, however 2-hydroxypropyl-β-cyclodextrin (HPβCD) has shown efficacy for treating ...
Aditya Kulkarni +8 more
core +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
Stem Cell ResearchFree sialic acid storage disorder (FSASD) is a rare, autosomal recessive, neurodegenerative disorder caused by biallelic mutations in SLC17A5, encoding the lysosomal transmembrane sialic acid exporter, SLC17A5.
Marya S. Sabir +10 more
doaj +1 more source