gene editing

RNA delivery to the corneal endothelium using charge-altering releasable transporters

A charge-altering releasable transporter (CART) that delivers RNA selectively to the corneal endothelium, a non-regenerative cell layer whose dysfunction underlies several blinding conditions and establishes CARTs as a platform for non-viral gene delivery to the eye, with the potential to treat corneal dystrophies and other vision disorders.

Targeted lipid nanoparticles unlock in vivo human haematopoietic stem cell gene editing.

CodeAssay: A Multi-Metric Benchmark with Audited Ground Truth for LLM Code Generation

These findings show that reliable evaluation of LLM-generated code requires validated ground truth, protected tests, and multiple explicitly interpreted measures, and that CodeAssay provides a reproducible basis for evidence-based model evaluation in AI-augmented software development.

Unified heterogeneity-aware benchmark of drug synergy prediction: a cross-study analysis of traditional machine learning and graph deep learning models.

The first comprehensive benchmarking framework specifically designed to accommodate inter-dataset heterogeneity is presented, finding that well-designed small datasets can match or even surpass the performance of larger benchmarks, suggesting that different metrics are applicable to different datasets/testing scenarios.

Navigating committor landscape of biomolecules with a general pairwise interaction model

A novel committor learning framework grounded in the AlphaFold 3 paradigm is proposed that elucidates how ligand substituents regulate the ratio between distinct binding pathways, offering new perspectives for structure-based drug design.

From single-pathway cascade to network pathophysiology: how genetically engineered mouse models reshaped our understanding of pancreatitis

Evidence is synthesized from genetically engineered mouse models that enhanced trypsinogen autoactivation alone is sufficient for spontaneous disease, that NF-κB-driven inflammation can be initiated independently of trypsin, and that loss of autophagy alone disrupts acinar homeostasis, which reframe pancreatitis as a network pathophysiology with two coupled layers.

Genomics-assisted Improvement of Resistance to Insect Pests, Diseases and Nematodes in Tomato: A Critical Appraisal of Discovery, Durability and Deployment

Whether, and where, genomic technologies have altered breeding outcomes rather than merely accelerating gene discovery is examined, and the available evidence indicates that genomic resources have substantially improved the resolution of resistance discovery and the precision of marker-assisted introgression, but have not yet demonstrably improved durability.

Correction to “Therapeutic Gene Editing: DNA Repair Pathways, Emerging Editors, and Clinical Progress”

Genomic architecture of hereditary defects in beef cattle populations: from molecular diagnostics to innovative therapy approaches (review)

Recommendations are formulated for building a genetic health management system in beef cattle breeding, based on mandatory DNA testing, inbreeding control, accounting for pleiotropic effects in selection, and the integration of advanced biotechnologies.

CRISPR/Cas9-Induced Dwarfism in Barley: Impacts on Yield-Related Traits and Root Architecture

These results provide new insights into HvDEP1′s role in both shoot and root systems and demonstrate that precise CRISPR/Cas9-mediated editing can rapidly introduce dwarfism while revealing trade-offs in other agronomic traits.

Cryptomeria japonica var. sinensis Miquel (Chinese Cedar): A Comprehensive Review of Its Biology, Phytochemistry, Biotechnology, and Utilization

This review provides a comprehensive foundation for understanding the biology, phytochemistry, biotechnology, and utilization of this invaluable botanical resource, offering a strategic roadmap for its sustainable management and commercial valorization.

Navigating Quality, Safety, and Analytical Testing Frameworks for Viral Vector–Based Gene Editing Therapies

This article aims to provide a working framework for verifying the potency, genomic integrity, and clinical safety of vector-based gene therapies—one intended to be useful both to laboratories developing these products and to those responsible for regulating them.