Results 121 to 130 of about 3,452,999 (260)

Molecular Glue Degraders Enhance CAPRIN1‐Dependent Lysosomal Degradation of APP and Reduce Amyloid β in Alzheimer's Disease

Advanced Science, EarlyView.
A new class of lysosome‐directed molecular glue degraders selectively enhance CAPRIN1–APP interactions, driving APP degradation and reducing amyloid‐β production in human neurons and Alzheimer's disease mouse models. This CAPRIN1‐dependent targeted protein degradation strategy reveals a previously unrecognized therapeutic approach for disrupting the ...
Sunghan Jung, Xu Wang, Bin Liu, Raktim Roy, Nancy Jaiswal, Ratan K. Rai, Yuichiro Takagi, Cen Gao, Lifan Zeng, Ho‐Yin Lo, Reagan K. Wohlford, Ryan K. Higgins, Bruce T. Lamb, Anantha Shekhar, Anita C. Bellail, Chunhai Hao   +15 more
wiley   +1 more source

Barriers to a successful healthcare transition for individuals with urea cycle disorders. [PDF]

Mol Genet Metab, 2023
Ladha FA, Le Mons C, Craigen WJ, Magoulas PL, Marom R, Lewis AM.   +5 more
europepmc   +1 more source

Mitochondria‐Targeted Multimodal Nanotherapeutics Suppress Oxidized mtDNA‐Driven Inflammation at the Source

Advanced Science, EarlyView.
Ox‐mtDNA fragments escaping from mitochondria drive robust inflammatory responses. A targeted nanoplatform simultaneously inhibits mitochondrial FEN1‐mediated mtDNA cleavage and scavenges ROS, preventing the generation of immunogenic Ox‐mtDNA fragments. The released SeNPs further promote autophagic clearance of cytosolic mtDNA. Consequently, cGAS‐STING,
Wen‐Ling Li, Juan Cao, Yu‐Lin Wang, Yu‐Han Fan, Jia‐Qi Xu, Xian Wu Cheng, Lei Xing, Hu‐Lin Jiang   +7 more
wiley   +1 more source

Probing the Energy Landscape of α‐Synuclein Amyloid Fibril Formation by Systematic K‐to‐Q Mutagenesis

Advanced Science, EarlyView.
We use lysine‐to‐glutamine mutations to study the effect of electrostatics on the kinetics and thermodynamics of alpha‐synuclein amyloid fibril formation. We find that mutational effects map on their structural context within fibrils and identify residues that modulate the energy landscape of alpha‐synuclein self‐assembly. Our work outlines a scalable,
Antonin Kunka, Azad Farzadfard, Jacob Aunstrup Larsen, Rasmus Krogh Norrild, Federica Saraceno, Celia Fricke, Hossein Mohammad‐Beigi, Ahmed Sadek, Jonas Folke, Susana Aznar, Alexander K. Buell   +10 more
wiley   +1 more source

Monitoring the treatment of urea cycle disorders using phenylbutyrate metabolite analyses: Still many lessons to learn. [PDF]

Mol Genet Metab, 2023
Glinton KE, Minard CG, Liu N, Sun Q, Elsea SH, Burrage LC, Nagamani SCS.   +6 more
europepmc   +1 more source

Supramolecular Zinc–Polyphenol Coordination Assemblies Mitigate Colonic Inflammation by Regulating Mucosal Barrier and Microbiome

Advanced Science, EarlyView.
A carrier‐free zinc‐coordinated polyphenol nanoplatform (ZnCA NPs) is designed as an “all‐in‐one” therapeutic strategy for intestinal inflammation. The ZnCA NPs exhibit enhanced colonic accumulation and broad‐spectrum reactive oxygen and nitrogen species scavenging.
Qiwen Xie, Mai Ye, Xuexia Liu, Jing Wang, Ying Chen, Lu Tan, Jinheng Fu, Xiaochang Huang   +7 more
wiley   +1 more source

Averting the foul taste of pediatric medicines improves adherence and can be lifesaving – Pheburane® (sodium phenylbutyrate)

Patient Preference and Adherence, 2016
Gideon Koren,1 Michael J Rieder,1 Yona Amitai2 1Department of Physiology and Pharmacology, The University of Western Ontario, London, ON, Canada; 2Bar-Ilan University, Ramat Gan, Israel Background: Children’s aversions to poor and mostly bitter ...
Koren G, Rieder MJ, Amitai Y
doaj  

Clinical, biochemical and genetic characteristics of patients with argininosuccinate lyase deficiency from a single center cohort in China

Orphanet Journal of Rare Diseases
Background Argininosuccinate lyase deficiency (ASLD) is a rare autosomal recessive urea cycle disorder (UCD) resulting from mutations in the ASL gene.
Kaichuang Zhang, Deyun Lu, Lili Liang, Yi Yang, Ruifang Wang, Yuning Sun, Zhuwen Gong, Haijuan Zhi, Wenjuan Qiu, Lianshu Han   +9 more
doaj   +1 more source

The GPX3‐VCAM1 Axis Gates Pro‐Fibrotic Tubule Cell Fate in Hyperuricemic Nephropathy

Advanced Science, EarlyView.
Hyperuricemia induces NLRP3 inflammasome signaling activation and downregulation of GPX3 in proximal tubular epithelial cells, leading to oxidative stress–driven VCAM1 upregulation, emergence of pro‐fibrotic proximal tubule cells, macrophage recruitment, and renal fibrosis.
Yunfei Qi, Qiang Zhao, Yue Lve, Yaming Yuan, Tingting Zhou, Yue Yuan, Dexi Wu, Yifan Tang, Yan Liu, Zhigang Li, Chunping Jiang, Qiurong Ding   +11 more
wiley   +1 more source

Self‐Propelled HPB@Lip@AB Nanomotors Ameliorate Dry Eye Disease

Advanced Science, EarlyView.
A self‐propelled HPB@Lip@AB nanomotor system is developed for dry eye disease (DED) therapy, facilitating rapid penetration across ocular surface barriers while integrating hydrogen therapy with multi‐enzyme‐like antioxidant activity. The nanoplatform restores mitochondrial function, suppresses oxidative stress, inflammation, and apoptosis, and ...
Jing Li, Xi Wan, Xinru Su, Zechuan Li, Yue Wang, Ying Wang, Ruofei Li, Huangzhao Wei, Jianbin Zhang, Shuai Wang   +9 more
wiley   +1 more source