自带导航 主动敲门 CRISPR有了新型“快递车”

Core Viewpoint - CRISPR technology has made significant advancements in gene editing, but the challenge remains in effectively delivering these tools to the target cells safely and efficiently. A breakthrough from Northwestern University has introduced a new delivery system that enhances the efficiency and accuracy of CRISPR applications in gene therapy [1][3]. Group 1: Current Delivery Methods - Current methods for delivering CRISPR include modified viruses and lipid nanoparticles (LNPs), each with their own limitations. Viruses are efficient but can trigger immune responses, while LNPs are safer but have low delivery efficiency [2]. - Another method involves ex vivo editing, which is complex and costly, making it impractical for most diseases. Thus, there is a need for a safer and more efficient in vivo delivery system [2]. Group 2: New Delivery System - The new system, termed "Lipid Nanoparticle Spherical Nucleic Acids" (LNP-SNA), features a DNA shell that enhances visibility and uptake by cells, significantly improving delivery efficiency [3]. - This innovative delivery vehicle has shown to be over three times more efficient in entering cells compared to traditional lipid nanoparticles, with a significantly lower toxicity profile. The success rate of precise gene editing has increased by over 60% [3]. Group 3: Versatility and Future Applications - The LNP-SNA system is modular, allowing for customization to target specific cell types, such as liver, brain, or cancer cells, thereby enabling precise delivery [4]. - Seven drugs based on similar spherical nucleic acid technology are currently in human clinical trials, with some focusing on cancer treatment. The technology is being promoted by various biotech companies for rapid clinical application [4].