为基因治疗装上“安全导航” 西电团队探索生物医药新赛道

Core Viewpoint - The research team at Xi'an University of Electronic Science and Technology has developed a novel non-ionic delivery system that addresses the "toxicity-efficiency" dilemma in mRNA therapy, enhancing safety and efficacy in gene therapy applications [1][2]. Group 1: Technology Overview - The new delivery system, termed TNP, utilizes thiourea groups to form strong hydrogen bond networks with mRNA, allowing for efficient loading without charge dependence, unlike traditional lipid nanoparticles (LNP) [2]. - TNP significantly extends the in vivo expression duration of mRNA to seven times that of LNP, improves targeting efficiency to the spleen, and achieves a near 100% cell survival rate, indicating high biocompatibility [2][3]. Group 2: Mechanism of Action - TNP employs a unique intracellular transport mechanism that avoids the Rab11-mediated recycling pathway, achieving a high intracellular retention rate of 89.7%, compared to only 27.5% for LNP [2]. - The interaction between thiourea groups and endosomal membrane lipids induces membrane permeabilization, allowing for the direct release of intact mRNA into the cytoplasm, thus circumventing lysosomal degradation [2]. Group 3: Implications for Gene Therapy - The innovative non-ionic delivery technology is expected to lower the costs of gene therapy, making treatments more accessible for patients with rare and chronic diseases [3]. - The research team has already developed multiple targeted delivery systems based on this technology, which are currently in animal testing phases for applications in tumor immunotherapy and gene editing for rare diseases [3].