gene editing

Phytomelatonin-mediated epigenetic and RNA regulatory networks in plant abiotic stress resilience.

This work proposes that phytomelatonin functions as an epigenetic and epitranscriptomic trigger capable of converting transient stress perception into durable transcriptional competence and outlines how single-cell multi-omics, targeted epigenome editing, epitranscriptomic profiling and field-scale validation of priming strategies can transform this conceptual framework into testable mechanisms and crop-improvement strategies.

The emerging roles of non-coding RNAs in cancer.

This review examines how different types of ncRNAs contribute to cancer initiation, progression, and treatment resistance, and assesses their potential as diagnostic markers, prognostic factors, and therapeutic targets.

Functional assessment of inherited myeloid neoplasm-associated SAMD9L germline variants via Monoallelic CRISPR modelling.

Functional evidence is provided to fine-tune the classification of these SAMD9L variants and significantly advance the understanding of the molecular mechanisms by which SAMD9L variants drive inherited myeloid neoplasms.

Targeting Cas9 to Perform Rescue Experiments in Stably Transduced Knockout HEK293 Cells.

Cas9 ablation enabled restoration of CUL4B expression and function following introduction of a CUL4B expression plasmid, providing proof of concept for a broadly applicable approach to studying protein function through rescue experiments.

Advancing variant-agnostic liver-directed gene editing using homology-independent targeted integration.

A PepFect14 analog improves non‐viral CRISPR delivery in primary human cells to facilitate genome editing and repair

A PepFect14 analog is employed to deliver high-fidelity Cas9-ribonucleoproteins and non-viral repair templates into primary human skin cells to mediate gene editing and repair targeting genes underlying the group of genetic skin blistering disorders epidermolysis bullosa (EB).

Gene knockout of P450s CYP392A16 and CYP392D2 reveals unexpected in vivo effects and transcriptional rewiring in the extreme generalist Tetranychus urticae.

It is demonstrated that even resistance-associated detoxification genes that are considered functionally validated in vitro, can have strong context-dependent phenotypic effects in vivo, underscoring the complexity of resistance gene networks and highlighting the need to interpret CRISPR/Cas9 knockout phenotypes within the broader genetic and regulatory context.

Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310.

In a phase 1a trial, one-time CRISPR-Cas9 gene editing with CTX310 was associated with no treatment-related dose-limiting toxic effects and with sustained reductions in ANGPTL3 and atherogenic lipids at 1 year.

Pan-Genomic Dissection of GH1 β-Glucosidases in Brassica rapa Identifies BrBGLU10 as an Important Regulator of Pollen Development

Findings establish BrBGLU10 as an important regulator of pollen development and a potential target for fertility-related applications via gene editing in B. rapa and related Brassica crops.

Non-invasive Ultra-early in Utero Detection and Precision CRISPR-mediated Correction of Monogenic Embryonic Mutations: A Critical Appraisal of a Hypothetical Therapeutic Framework

Whether that proposition that a pathogenic single-gene variant might be identified non-invasively at the earliest stage of pregnancy and corrected in situ before irreversible pathology develops is presently coherent as a therapeutic framework is examined.

Gene and nucleic acid therapies for MMUT-deficient methylmalonic acidemia: from AAV-mediated gene addition to LNP-mediated mRNA delivery and precision genome editing

Current therapeutic evidence is concentrated primarily in MMUT- deficient isolated MMA, and further progress will require safer and more durable delivery platforms, improved tissue targeting, robust long-term safety assessment, clinically meaningful endpoints, and careful evaluation of accessibility and cost.

Chinese geneticists across the Pacific: a century of scientific legacies in genetics and genomics

This historical review presents the breakthrough discoveries by prominent Chinese geneticists during the early pioneering generation, the molecular genetics era, the transition to human and medical genetics, and current progress in genetic and genomic medicine.