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1,680 papers
The results demonstrate that common genetic variants can influence disease risk through post-transcriptional mechanisms that alter lipid homeostasis in astrocytes, a cell type increasingly recognized as central to ALS progression.
Advances in mesenchymal stem cells, extracellular vesicles, CRISPR-based gene editing, and emerging precision medicine strategies including multi-omics profiling, machine learning-based patient stratification, and adaptive trial design are discussed.
Findings indicate that the Cas12a system does not yield locus excision at the efficiency required for sensitive negative selection assays, and showed no meaningful correlation between gRNA efficiency, orientation, GC content, or cut site geometry and the success of locus excision.
A generalizable framework to modulate gene dosage by alternative splicing via genome editing is presented, allowing high-fidelity animal models for human diseases caused by haploinsufficiency and leaving endogenous gene expression regulatory logic intact.
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This work extensively characterized a CRISPR-engineered human hematopoietic stem and progenitor cell model of ASXL1 mutations, and is the first to functionally describe the mutant ASXL1 interactome in the context of human HSCs, identifying MECOM and BRD4 as actionable dependencies with therapeutic potential for ASXL1-m...
It is demonstrated that technology CRISPR–Cas9 gene editing holds significant promise as a therapeutic strategy for TP53-mutant CLL by enabling precise gene correction of functional replacement.
Roles for certain genetic variants in CHEK2 in NB are supported, as well as potential mechanistic targets for high-risk or therapy-resistant NB, and preclinical information is provided to inform diagnostics and potential therapeutic strategies for high-risk NB.
This dataset was generated as part of a research project investigating how transfer RNA (tRNA) abundance and tRNA modification regulate codon-dependent translation in hepatocellular carcinoma (HCC). The data cover two complementary aspects of tRNA-mediated translational regulation.The first project examines the relatio...
Environmental particulate exposure contributes to the severity and persistence of numerous acute and chronic lung diseases, including asthma, viral pneumonia, acute respiratory distress syndrome, and pulmonary fibrosis. Chitin, a highly abundant environmental polysaccharide present in fungi, insects, and mites, is cont...
A defined biorthogonal conjugation system that tethers non-viral nanoparticles to biomaterial platforms for sustained and localized CRISPR gene editing is established.
ABSTRACT Life sciences represent one of the most dynamic and transformative scientific domains in the modern world. Rapid technological progress, interdisciplinary research integration, and molecular-level understanding of biological systems have revolutionized healthcare, agriculture, environmental protection, and bio...
The file dohai_thesis_core_v1.zip contains the analysis scripts and associated input files used for the doctoral thesis titled “Network Organization of Genetic Susceptibility to Disease”. The scripts are organized according to the analytical workflow described in the Methods chapter. The workflow includes harmonization...
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A Blog post by NVIDIA on Hugging Face
Zero-knowledge proofs can verify computing results without rerunning workloads or exposing sensitive data. Magnetar helps bring them into the mainstream with support for standard programming languages, major speed gains, and quantum-resistant security. The post Proof-based general-purpose computing with Magnetar appeared first on Microsoft Research.
General Science
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