gene editing

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...

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.

An hiPSC-derived multi-lineage lung model exhibiting proximal-distal epithelial features for modeling pulmonary fibrosis

A humanized multi-lineage lung model that captures epithelial-mesenchymal co-development and responsiveness to TGF-β1 stimulation and sensitivity to pirfenidone highlight its potential for antifibrotic drug screening.

Strigolactones: From Fundamental Biology to Applications in Tree Breeding.

This study proposes a research and breeding roadmap progressing from molecular validation to medium- and short-term characterization in model trees and finally to multisite long-term ecological assessment, and presents a multiscale theoretical framework and testable pathways for translating SLs research from model plan...

Advancing Drought and Salinity Stress Tolerance in Plants: Mechanisms, Molecular Tools and Breeding Strategies—A Review

This review comprehensively synthesizes recent advances in abiotic stress perception, signal transduction and hormone‐mediated regulation, highlighting their roles in shaping plant stress tolerance and proposes an integrated multi‐scale framework to provide a holistic understanding of drought and salinity stress tolera...