Category
84 papers
It is argued that formation of a tumour-intrinsic niche is a prerequisite for BRAF-mutant CRC seeding to distant organs and that interference with niche formation may help avoid metastatic relapse.
It is concluded that bridging the gap between foundational CRISPR research and its real-world applications is imperative and future efforts should focus on democratizing tools via open-source platforms, advancing delivery systems, and fostering sustainable innovation through synthetic biology integration to fully realize the transformative potential of genome editing in organisms beyond model organisms.
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 therapies hold tremendous promise for treating genetic eye diseases. However, their development is limited by the lack of non-viral delivery platforms that can target specific ocular cell types. Here, we describe 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. We characterize the safety of CART-RNA nanoparticles in mice and show that they facilitate delivery of diverse RNA cargoes to the corneal endothelium, including circular RNA and CRISPR/Cas9. We verify that these nanoparticles can be redosed and apply them to achieve corneal gene editing. We further demonstrate CART transfection of corneal endothelial cells from a human donor in vitro and in a non-human primate in vivo, supporting the feasibility of clinical translation. Our findings establish CARTs as a platform for non-viral gene delivery to the eye, with the potential to treat corneal dystrophies and other vision disorders.
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.
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.
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.
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.
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.
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.
Generative AI
A new method for surgically removing training examples from a model reveals that as datasets grow, the link between what a model learns and what it produces dissolves.
In his latest book, Professor Eugene Fitzgerald examines the forces that turn breakthroughs into value — and why innovation resists simple formulas.
General Science
Generative AI
General Science
