Gubatan et al. demonstrate vitamin D’s immunoregulatory role on host immune-microbe interactions in patients with inflammatory bowel disease. Vitamin D alters the profiles of IgA- and IgG-bound gut…

Here, Fang et al. highlight emerging and established benefits of GLP-1 medicines in neurological and psychiatric disorders. These medicines exert direct and indirect effects on the brain, improving ne...

Djalalvandi et al. show that blockers of Opa1-targeting microRNAs found upregulated in diseased skeletal muscle can restore mitochondrial architecture and muscle performance in mitochondrial myopathy ...

Histopathological evaluation is a cornerstone of cancer identification but often involves time-consuming labeling processes (∼days per sample) and exp…

Silva and colleagues refine an EV isolation method to optimize recovery of cancer-derived large oncosomes (LOs). This approach enables high-yield profiling by mass spectrometry and bulk/single-LO RNA-...

De Nardo et al. profile the plasma and liver-secreted proteome from obese individuals spanning the spectrum of MASLD. They identify biomarkers for MASH in persons with obesity and develop and validate...

Bai et al. establish a rhesus monkey model for DMD with exon 50 mutations, closely mimicking human pathology. Using this model, they develop and evaluate a single-cut gene therapy targeting exon 51 of...

Kowala et al. demonstrate a clinically relevant transplantation strategy synergizing engineered human myogenic cells and hydrogel-mediated delivery in a dystrophic mouse model. Upon long-term engraftm...

Komaki et al. show that brogidirsen, a dual-targeting antisense oligonucleotide, shows promising safety and dose-dependent dystrophin restoration in a phase 1/2 trial for Duchenne muscular dystrophy. Potential biomarkers and motor function stabilization are observed. High efficacy is validated through patient-derived cell-based assays, warranting a subsequent trial to confirm its therapeutic potential.

Malignant rhabdoid tumor (MRT) is an aggressive childhood cancer with limited treatment options. Using patient-derived tumor organoids (tumoroids), Kes et al. identify nucleotide biosynthesis as a vulnerability in MRT. Treatment with methotrexate (MTX) and BAY-2402234 reduced nucleotide levels and induced apoptosis in vitro, with MTX demonstrating reduced MRT growth in vivo.

Excellent news — assuming it pans out… Newly discovered antibody protects against all COVID-19 variants @utaustin @CellRepMed https://t.co/3ni1mvR5tB — Medical Xpress (@medical_xpress) September 3, 20...

Excellent news — assuming it pans out… Newly discovered antibody protects against all COVID-19 variants @utaustin @CellRepMed https://t.co/3ni1mvR5tB — Medical Xpress (@medical_xpress) September 3, 20...