Results 151 to 160 of about 3,524,002 (263)

Novel Fused Pyrazole Amide Analogs as Glucosylceramide Synthase Inhibitors for Treating Lysosomal Storage Diseases, Neurodegenerative Diseases, Cystic Diseases, and Cancer. [PDF]

ACS Med Chem Lett, 2023
Sabnis RW.
europepmc   +1 more source

ROS‐Responsive Nanoparticle Delivery of Dexmedetomidine Protects the Gut Vascular Barrier After Intestinal Ischemia/Reperfusion via the HDAC‐H3K27ac‐TCF4 Axis

Advanced Science, EarlyView.
A low dose of dexmedetomidine, delivered by ROS‐responsive nanoparticles, preferentially accumulates in the ischemic intestine and restores gut vascular barrier integrity after ischemia/reperfusion injury. This nanoparticle‐based strategy prevents bacterial translocation and liver damage without cardiorespiratory side effects, working through HDAC ...
Hu‐Fei Zhang, Jian‐Tong Shen, Hua‐Hua Zhang, Si‐Qi Gao, Ji‐Ao Wang, Yue‐Ling Wang, Mei‐Ling Li, Zi‐Meng Liu, Yi‐Nan Zhang, Yan Li, Xu‐Yu Zhang   +10 more
wiley   +1 more source

Human, economic, and social impact of lysosomal storage diseases. [PDF]

Orphanet J Rare Dis
Brignani E, de Castro-López MJ, Gonzalez-Meneses A, Morales M, Pintos-Morell G, Poveda JL, Vilalta I, Cruz J, Ballester-Lozano GF, David de Paz H.   +9 more
europepmc   +1 more source

Beyond Biomimetics: Pathology‐Informed Engineering Rescues Reconstituted HDL From Inflammatory Dysfunction for Sepsis Immunotherapy

Advanced Science, EarlyView.
A pathology‐informed engineering strategy rescues reconstituted HDL from inflammatory dysfunction in sepsis. By integrating a ceria nanozyme‐celastrol coordination complex, the platform preserves HDL structure and endotoxin handling, sustains hierarchical intracellular delivery, and enhances immunoregulation across pathological models, translating ...
Han Zhou, Lei Zhang, Xingyu Cai, Jiajia Su, Guangyu Dai, Xue Chen, Hanming Wang, Jia Hua, Jianping Liu, Chunmeng Sun, Xiancheng Chen, Erhao Wang, Wenli Zhang   +12 more
wiley   +1 more source

Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]

EBioMedicine
eBioMedicine.
europepmc   +1 more source

Editorial: Neuronopathic lysosomal storage diseases - specific neuronal characteristics and therapeutic approaches. [PDF]

Front Mol Neurosci, 2022
Flunkert S, Hutter-Paier B, Sun Y, Kehr J.   +3 more
europepmc   +1 more source

Spatial Transcriptomics Reveals a T Cell‐Mediated Microglial Activation Axis of Immune Checkpoint Inhibition‐Related Adverse Events in the Brain

Advanced Science, EarlyView.
Immune checkpoint inhibition (ICI) reshapes the hippocampal neuroimmune niche. Spatial transcriptomic profiling and human brain histological analyses identify neuroinflammation and altered neuronal and glial programs. Notably, T cell depletion prevents ICI‐driven microglial activation, establishing the T cell–microglia crosstalk axis as a driving ...
Devyani Swami, Sanad M. El‐Khatib, Suhas Sureshchandra, Elise Nguyen, Janaki Manoja Vinnakota, Robert Zeiser, Shivashankar Othy, Munjal M. Acharya   +7 more
wiley   +1 more source

Japanese experience of newborn screening for lysosomal storage diseases and adrenoleukodystrophy. [PDF]

Orphanet J Rare Dis
Onuki T, Tajika M, Sugiyama Y, Shimura M, Ichimoto K, Tanaka T, Nyuzuki H, Kosuga M, Migita O, Ito T, Sasai H, Bo R, Hamada J, Hamazaki T, Sakai N, Inoue T, Nakamura K, Okuyama T, Murayama K.   +18 more
europepmc   +1 more source

Correction to: A systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases. [PDF]

Cost Eff Resour Alloc, 2022
Katsigianni EI, Petrou P.
europepmc   +1 more source

Restores Aβ Clearance by Overcoming PCSK9–LRP1 Dysregulation and TRIB3‐Mediated Autophagy Blockade in Alzheimer's Disease

Advanced Science, EarlyView.
PCSK9 upregulation in cerebrovascular endothelial cells and microglia destabilizes the Aβ transporter LRP1, while TRIB3‐associated autophagy blockade further limits intracellular Aβ degradation. A RAP‐modified tea‐polyphenol nanoparticle co‐delivers siPCSK9 and siTRIB3 to restore LRP1‐mediated Aβ efflux and autophagy‐dependent degradation, reducing Aβ ...
Jie Miao, Jing Wang, Yankun Li, Haipei Zhang, Yanli Zhang, Qingnian Li, Bo Xiao, Wenhu Zhou, Junhong Guo   +8 more
wiley   +1 more source