Biomembrane-inspired lipid nanoparticles enhance CRISPR-Cas9 editing for hemophilia A.

Lipid nanoparticles (LNPs) have substantially advanced RNA-based therapies; however, their use for CRISPR-Cas9 remains limited by sub-optimal endosomal escape, innate immune activation, transient nuclease expression, and restricted tissue specificity. Here, we engineered biomembrane-inspired LNPs containing sphingomyelin and C18-galactosyl ceramide (C18-GalCer) to improve liver-targeted CRISPR delivery. The optimized formulation increased in vivo editing efficiency 2.

Direct Synthesis of Highly Siliceous Faujasite-type Zeolite Enabled by Low Charge Density Organic Structure-directing Agents.

Ultrastable Y (USY) zeolites (Faujasite-type, ) with high SiO/AlO ratios (SARs) have been widely applied in fluidized catalytic cracking and hydrocracking processes. However, their preparation typically involves labor-intensive post-treatments that inevitably introduce defects, extra-framework species, and compositional gradients. Herein, we report the direct synthesis of -type zeolite with a record-high SAR up to 21.

Generating Beta Zeolite Nanosheets of Intergrown Polymorph B and C Using Polycationic Structure-Directing Agent.

Zeolitic nanosheets possess great potential in catalysis due to their enhanced transport property and accessibility toward bulky molecules compared to conventional micron- meter scale crystals. However, the generation of Beta zeolite nanosheets, which are crucial for industrial catalysis, is still challenging for its intergrowth nature. In this work, aluminosilicate Beta nanosheets of ca.

OliTag-seq enhances in cellulo detection of CRISPR-Cas9 off-targets.

The potential for off-target mutations is a critical concern for the therapeutic application of CRISPR-Cas9 gene editing. Current detection methodologies, such as GUIDE-seq, exhibit limitations in oligonucleotide integration efficiency and sensitivity, which could hinder their utility in clinical settings. To address these issues, we introduce OliTag-seq, an in-cellulo assay specifically engineered to enhance the detection of off-target events.