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1250 articles, sorted by publication time.
Researchers used in vivo CRISPR base editing to skip HTT exon 13, preventing the mutant protein from being cleaved into toxic fragments prone to aggregation; brain atrophy was subsequently reduced in mice, but delivery, off-target effects, long-term safety, and other hurdles remain before the approach can become a human treatment.
A multinational team identified nine additional patients, outlining neurological and cardiac manifestations as well as disease beginning in the fetal period; all four children who received high-dose nicotinic acid survived subsequent infections, but the extremely small number of cases remains insufficient to demonstrate efficacy or long-term safety.
A University of Hong Kong team combined normal samples to create a range of tumor scenarios, enabling a deep learning model to be trained when real cancer data are scarce. The model performed strongly in cell-line tests, but the results cannot yet be translated directly into clinical diagnosis.
The research team reduced either autophagy or LTβR signaling in specialized blood vessels, and both approaches decreased immune cell influx and skin inflammation. This pathway, which avoids directly suppressing immune cells, must still overcome hurdles involving selectivity and validation in humans.
The five-day antiviral regimen moves COVID-19 defenses into the brief window after exposure but before illness develops; a Phase 3 trial showed a lower risk of symptomatic infection, but drug interactions, pregnancy risks, and limits on eligibility continue to restrict its use.
The study extracted signals from the routine questionnaire response patterns of 45,604 older adults. Joint multinational training improved external validation performance, but at this stage the model is suited to helping prioritize assessments and cannot replace cognitive testing or clinical diagnosis.
The largest-ever genetic study of fibromyalgia identified 26 risk regions, adding molecular evidence for abnormal pain processing. Although HTT and GPR52 offer new directions for drug research, diagnosis and treatment remain a long way off.
The first participant has received an intrathecal dose. BEACON will assess changes in cognition and expressive communication to determine whether restoring UBE3A expression can translate into tangible clinical benefits.
A cross-modal AI model infers molecular signals such as proteins from more readily obtainable single-omics data while preserving the neighborhood relationships of cells within tissue. It has demonstrated research value in cancer and brain datasets, but computational reconstruction still cannot replace actual measurement.
Live-cell imaging captured intracellular tuberculosis bacteria with doubling times of less than 10 hours; they persisted after treatment with first-line antibiotics, suggesting that drug tolerance is not limited to slow-growing bacteria.
By integrating single-cell molecular maps, chromatin structure, and genetic data, the research team charted how heart failure rewrites gene regulation across different cell types, while providing more precise coordinates for interpreting noncoding risk variants.
The research team used active learning to guide automated experiments in search of stable metabolic cooperation between 15 gut bacteria and six dietary fibers; the results have been validated in human fecal microbial communities, but animal and clinical hurdles remain before disease treatment.