gene editing

VASP cooperates with zyxin to organize mechanically competent adhesions

Focal adhesions (FAs) are dynamic macromolecular assemblies that anchor the actin cytoskeleton to the extracellular matrix via integrin receptors, thereby regulating cell morphology and migration. Here, we show that the vasodilator-stimulated phosphoprotein (VASP) cooperates with the mechanosensitive scaffold protein...

Development of an Efficient In Vitro Propagation Protocol for the Elite Bidah Pomegranate (Punica granatum L.) Landrace in Saudi Arabia

The Bidah pomegranate (Punica granatum L.) is an elite landrace native to Wadi Bidah in the Al-Baha region of Saudi Arabia. This cultivar is distinguished by its unique taste, high yield, genetic component, and phytochemical profile. In addition, its resilience to harsh environmental conditions aligns closely with nati...

CRISPR/Cas9 induced disruption of oca2 and tyr genes associated with pigmentation in Danio rerio

Targeted disruption of genes has been successfully carried out in several model and non-model fish species. In ornamental fish industries, Danio rerio (D. rerio) represents the most significant fish species and is available in several color varieties. Recently, CRISPR/Cas9 has been used to disrupt pigmented genes in se...

Functional dissection of ultraconserved non-coding regions present near the Hoxd complex in vertebrates

Ultraconserved non-coding elements (UCEs) exhibit near-perfect sequence conservation across vertebrate genomes, implying severe evolutionary constraint and essential biological functions. Near the vertebrate Hoxd complex, a master regulator of axial patterning and limb morphogenesis, three Conserved Regions (CR1, CR2,...

Artificial Intelligence as a Catalyst for Cell-Based Therapies in Retinal Disease

Retinal degenerative diseases, including age-related macular degeneration (AMD), retinitis pigmentosa (RP), and Stargardt disease, represent leading causes of irreversible vision loss worldwide, collectively affecting hundreds of millions of people. Cell-based therapies, particularly those leveraging induced pluripoten...

Rapid development of durable bacterial blight resistance in African rice through marker-assisted pyramiding of four resistance genes

Abstract Bacterial blight of rice causes substantial crop losses in Asia and Africa. The recent introduction of Asian strains into African countries has led to two independent outbreaks detected in 2019, causing severe damage in Madagascar and Tanzania. The strains are highly virulent on local rice varieties and rapidl...

Evolutionary Diversification and Functional Roles of MYB Transcription Factors in Algae: Insights from Genomic Distribution and Regulatory Networks

MYB transcription factors (TFs) represent a conserved family of DNA-binding regulators involved in diverse cellular and physiological processes. MYB TFs are well-characterized in terrestrial plants, but their evolutionary history and functional diversity in algae remain poorly understood. This review summarizes current...

Programmable Enzymes for Large-Cargo Genome Engineering: From Targeted DNA Integration to Therapeutic Genome Writing

Genome engineering is undergoing a conceptual transition from the correction of individual sequence variants toward the programmable installation of larger functional genetic elements. CRISPR-associated nucleases established RNA-guided genome targeting as a broadly accessible technology, while base and prime editing su...

Retrotransposons: A New Frontier in In Vivo Genome Engineering

Genome engineering has transformed cell therapy, but clinical implementation remains largely confined to ex vivo workflows followed by adoptive cell transfer. These approaches require costly manufacturing, often rely on viral vectors, and carry risks associated with unwanted genomic alterations. Recent advances in lipi...

High-Throughput Screening of a Lipid Nanoparticle (LNP) Library Identifies Lead Formulations for CRISPR-Cas9 Ribonucleoprotein Delivery and Gene Editing.

Delivery of CRISPR-Cas9 ribonucleoprotein (RNP) using synthetic materials can bypass the host translational machinery and limit the duration of genomic exposure, minimizing the risk of off-target mutations. Despite their advantages, lipid nanoparticle (LNP) platforms for CRISPR-Cas9 RNP delivery that achieve highly eff...

Ocular Gene Therapy in China: Technological Framework, Clinical Translation, and Future Perspectives.

This review summarizes the technological landscape and representative clinical advances of ocular gene therapy in China, highlighting the evolution from established gene-replacement approaches toward increasingly differentiated therapeutic strategies and key challenges in interpreting clinical benefit and translating e...

Emerging stem cell strategies for neurodegenerative diseases.