新型基因编辑技术同时修复多个致病突变

Core Insights - A new gene editing technology based on reverse transcriptase has been developed, allowing for more precise and efficient repair of multiple pathogenic mutations in mammalian cells, laying a significant foundation for broad-spectrum gene therapies [1][2] Group 1: Technology Development - The new technology can simultaneously correct various pathogenic mutations in mammalian cells and has successfully cured mutations causing scoliosis in zebrafish embryos, marking the first use of reverse transcriptase technology for precise mutation repair in vertebrates [1] - Existing gene editing technologies typically target one or two specific mutations, limiting effective treatment options for many patients; the new technology can replace entire defective regions with healthy DNA segments, akin to "patching" genes, thus offering hope for more patients [1] Group 2: Efficiency and Application - The new technology has increased editing efficiency from 1.5% to approximately 30% in mammalian cells, significantly improving the success rate of gene editing [2] - The technology utilizes an RNA delivery system encapsulated in lipid nanoparticles, effectively overcoming challenges associated with the delivery phase of traditional gene editors [2] - The research team is currently developing a gene therapy for cystic fibrosis, a disease known to have over a thousand pathogenic mutations, with the new technology potentially benefiting a broader patient population by directly replacing entire defective regions [2]