Results 211 to 220 of about 556,664 (302)
First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria
Advanced Science, EarlyView.We report the first PROTACs designed to degrade progerin, introducing a novel therapeutic approach for progeria. The best compound, UCM‐18142, significantly reduces progerin levels and improves key disease phenotypes in patient‐derived cells and in the LmnaG609G/G609G mouse model, paving the way for new treatment strategies targeting the root cause of ...
Jon Macicior‐Michelena +5 more
wiley +1 more source
Splicing the narrative: alternative TARDBP splicing and its relation to neurodegeneration in ALS and FTD. [PDF]
J Clin InvestMiller MR, Dykstra M, Barmada S.
europepmc +1 more source
Bacterial Outer Membrane Vesicles in Potentiating Cancer Vaccines: Progress and Prospects
Advanced Science, EarlyView.Bacterial outer membrane vesicles (OMVs) have emerged as versatile platforms for cancer vaccine development owing to their intrinsic immunostimulatory properties and high engineering flexibility. This review summarizes OMV biology, immune mechanisms, and engineering strategies that enhance vaccine efficacy, discusses key translational challenges, and ...
Jiabeini Zhang +9 more
wiley +1 more source
The human branchpoint-interacting stem-loop sequence and structure regulates U2 snRNA expression, branchpoint recognition, and the transcriptome. [PDF]
Nucleic Acids ResStevers MB, Katzman S, Jurica MS.
europepmc +1 more source
Leveraging Artificial Intelligence and Large Language Models for Cancer Immunotherapy
Advanced Science, EarlyView.Cancer immunotherapy faces challenges in predicting treatment responses and understanding resistance mechanisms. Artificial intelligence (AI) and machine learning (ML) offer powerful solutions for cancer immunotherapy in patient stratification, biomarker discovery, treatment strategy optimization, and foundation model development.
Xinchao Wu +4 more
wiley +1 more source
DNA Replication Errors Drive Genome‐Wide Small Inverted Triplication Dynamics
Advanced Science, EarlyView.This study provides insight into the dynamic equilibrium mechanism of a novel structural variant, small inverted triplication (SIT), which is generated by misalignment of the 3’ flap generated under DNA replication stress with palindromic sequence. Alternatively, the end sequence may fold back on itself to form an inverted fragment.
Yi Lei +12 more
wiley +1 more source
Resolving non‑coding splice‑altering variants using an integrative genomic and transcriptomic workflow: application to FOXP1. [PDF]
Hum GenomicsPlanté-Bordeneuve P +9 more
europepmc +1 more source
Advanced Science, EarlyView.In this study, we identified an NSUN6‐dependent m5C‐modified tsRNA, m5C‐tRF3b CysGCA‐23 (mtRC), that is downregulated in BC and inversely correlated with disease progression. Mechanistically, mtRC suppresses BC malignancy by stabilizing RBM4, attenuating glycolysis, and thereby limiting H3K18 lactylation–mediated activation of IL1RAP and VASH2 ...
Xiaoling Ying +16 more
wiley +1 more source
Advanced Science, EarlyView.Glaucoma, a major cause of blindness, involves retinal ganglion cell (RGC) degeneration. This study shows growth hormone‐releasing hormone receptor (GHRHR) deficiency preserves RGC survival and restores vision, unlike activation which only aids survival.
Yan Tong +24 more
wiley +1 more source