gene editing

Neoantigen in cancer immunotherapy: mechanism, therapeutic strategies and future perspectives.

Overall, despite existing challenges, neoantigen-targeted immunotherapies hold promise for advancing precision oncology and improving patient outcomes.

Porcine Expanded Potential Stem Cells as a Versatile Platform for Multiplex Genome Editing and Immunophenotyping in Xenotransplantation

It is demonstrated that porcine expanded potential stem cells, derived from preimplantation embryos, provide a robust and versatile platform for generating donor cells for xenotransplantation and for functionally evaluating genetic modifications, thereby advancing the prospects of xenotransplantation.

Induced pluripotent stem cell reprogramming: methodological evolution and challenges in clinical translation

This review summarizes the trajectory of iPSC reprogramming technologies and identifies the core “translational triltrilas”, namely, the inherent tradeoffs between security, homogeneity, and scalability, and proposes a comprehensive strategy to overcome these bottlenecks.

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...

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.

Automated PLC Programming Using a Multi-Task LLM System

Programmable Logic Controllers (PLCs) are central to industrial automation, yet their programming remains largely manual and time-consuming. While Large Language Models (LLMs) excel at general-purpose code generation, their use in industrial control is limited by tight coupling with physical hardware, constrained I/O s...

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.

CRISPR-mediated Correction of Oncogenic AS-NMD in Splicing Factor Mutant Cancer

The CRISPR strategy shows compelling evidence as a therapeutic approach targeting PE in cancer and other human diseases as well as the preferential advantages of CRISPR over the antisense technology recently developed targeting the PE of EZH2.

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 n...

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...