CRISPR有了新型“快递车”

Core Insights - CRISPR technology represents a revolutionary gene-editing tool that offers unprecedented hope for treating genetic diseases, cancer, and rare diseases by precisely modifying disease-causing genes [1] - A significant breakthrough has been achieved by a team from Northwestern University, which has developed a new "gene delivery vehicle" that enhances the efficiency of delivering CRISPR tools into cells while reducing damage and improving gene repair accuracy [3] Delivery Mechanisms - Current methods for delivering CRISPR into cells primarily rely on two vehicles: modified viruses and lipid nanoparticles (LNPs). Viruses are efficient but pose safety risks due to immune responses, while LNPs are safer but have lower delivery efficiency [4][5] - The new system, termed "Lipid Nanoparticle Spherical Nucleic Acids" (LNP-SNA), features a special DNA shell that enhances visibility and acceptance by cells, significantly improving delivery efficiency [6] Performance Metrics - The new delivery vehicle demonstrates over three times the efficiency of traditional lipid nanoparticles, with significantly lower toxicity to cells. The success rate of gene editing has also increased by over 60% [6] - This technology is modular, allowing for targeted delivery to specific cell types, such as liver, brain, or cancer cells, by altering the DNA shell's "code" [7] Clinical Applications - Seven drugs based on similar spherical nucleic acid technology are currently in human clinical trials, with some testing cancer treatment efficacy. The new technology is being promoted by several biotech companies for rapid clinical trial application [7] - The breakthrough emphasizes that while CRISPR itself is powerful, the method of delivery is equally critical, marking a significant advancement in gene therapy capabilities [7]