Genome editing corrected rare brain mutations in mice. Could it help fight neurological diseases?
Peer-Reviewed Publication
Updates every hour. Last Updated: 14-Nov-2025 19:11 ET (15-Nov-2025 00:11 GMT/UTC)
Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with neurological diseases. In tests that also included patient derived cells, the cutting-edge technique not only fixed mutations causing alternating hemiplegia in childhood (AHC) — it also reduced symptoms and extended survival in mice that had AHC and were otherwise at risk of sudden death. The findings were published in Cell.
Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, to correct the root cause of alternating hemiplegia of childhood (AHC), a debilitating genetic condition, in mice. The team used a scalable approach to develop prime editing treatments that directly repaired five different AHC-causing genetic mutations. Mice that received the treatment had far fewer and less severe symptoms of AHC, and survived more than twice as long as untreated mice.
Hydrogen peroxide (H2O2) is a versatile oxidant widely used in pharmaceuticals, environmental protection, and chemical manufacturing. However, conventional H2O2 production relies on energy-intensive processes and costly metal-based catalysts, raising economic and environmental concerns. As a sustainable alternative, photocatalytic H2O2 synthesis harnesses solar energy, water, and oxygen under mild conditions. This research group summarizes recent advancements in the development of metal-free organic semiconductors for photocatalytic H2O2 generation. Notably, it delves into novel surface reaction mechanisms, including anthraquinone intermediate, peroxy acid intermediate, bipyridine intermediate, and dual channel synergistic mechanisms for optimizing photocatalyst performance. They also highlight the critical role of advanced characterization techniques, including in-situ characterizations and computational simulations, in understanding structure-property relationships and real-time catalytic processes. By proposing new strategies for material modification and potential device-based applications, this review aims to stimulate further research and promote the industrialization of photocatalytic H2O2 production, contributing to sustainable chemical processes.
The application of CAR-T cell therapy against solid tumors is often hindered by the dense and rigid tumor extracellular matrix (ECM). While combining CAR-T with hyaluronidase (HAase) to reduce ECM is apparent, the efficacy is limited because of low accumulation and penetration efficiency of HAase inside the tumor tissue. Herein, the stimuli-responsive HAase-loaded nanogels (H-NGs) which are conjugated on the surface of CAR-T cells were designed for synergistically improving HAase accumulation, ECM degradation and CAR-T cell efficacy. The conjugation of H-NGs on the T cell surface was achieved through metabolic oligosaccharide engineering (MOE) in a semi-quantitatively controlled manner. Intravenous injection of H-NGs armed CAR-T cells resulted in more ECM degradation than co-injection of CAR-T cells and free H-NGs, leading to an 83.2% tumor inhibition rate and relieves tumor suppressive microenvironment in the Raji solid tumor model. Proteomic analysis of the harvested tumor tissues indicated that the combining of H-NGs and CAR-T cell collaboratively reduces cell adhesion and enhanced leukocyte transendothelial migration. Overall, this work simultaneously boosts the efficacy of hyaluronidase and CAR-T cells in combating solid tumor, which has broad application potential in cancer combination therapy.
A research team has mapped and characterized the DELLA gene family in kiwifruit and explored its role in fruit softening and ripening delay.
A groundbreaking study led by researchers at Huazhong University of Science and Technology (HUST) has developed a high-performance near-infrared (NIR) computational spectrometer using finely-tuned lead sulfide (PbS) quantum dots (QDs). This innovation, published in Nano Research, achieves a spectral resolution of 1.5 nm, making it a powerful tool for applications ranging from qualitative material identification to quantitative alcohol content measurement in liquor. The study highlights the critical role of QD monodispersity and precise synthesis in enhancing spectrometer performance, paving the way for portable, low-cost NIR spectrometers in industrial and consumer applications.
Single-atom cobalt catalysts have been recognized as promising alternatives to natural enzymes. However, their relatively low catalytic activity greatly limits their further application. Herein, Single cobalt sites immobilized on defective carbon nanosheets (2D Co-CN(H)) can act as efficient oxidase mimics with high atom utilization efficiency. In particular, the 2D Co-CN(H) catalysts are found to be twice as effective as defect-free Co-CN catalysts. Combined experimental and theoretical analyses reveal that the defects around atomic cobalt sites can rationally regulate the electronic distribution, significantly promoting the cleavage of O-O bonds and thus improving their oxidase-like performance. Taking advantage of the excellent oxidase-like activity of 2D Co-CN(H) catalysts and the good photothermal properties of oxTMB, an innovative dual-mode colorimetric-photothermal sensing platform toward effective discrimination and detection of dihydroxybenzene isomers has been successfully constructed. This study not only highlights the important role of defects on the oxidase-like activity of single-atom nanozymes, but also broadens their potential applications in environmental conservation.