我国新型基因治疗策略有望推动孤独症、癫痫等疾病治疗

Core Insights - A research team from the Shenzhen Institute of Advanced Technology has developed a new gene therapy strategy called "AAVLINK," which addresses the challenge of efficiently delivering long genes using AAV (adeno-associated virus) vectors, potentially advancing clinical applications for gene therapies targeting neurological disorders like autism and epilepsy [1][2]. Group 1: Gene Therapy Development - The AAVLINK method innovatively splits long genes into two segments, each carried by separate AAVs, with one AAV containing a special "molecular magic tape" (lox site) and the other carrying the second gene segment along with the Cre recombinase gene [2]. - This approach allows for precise recombination of the split gene segments within target cells, enabling the expression of a complete functional gene [2]. - The technology has been shown to efficiently reconstruct large gene segments in various cell types without producing truncated proteins, demonstrating significantly higher recombination efficiency compared to traditional methods [2]. Group 2: Safety and Future Research - The research team has developed a 2.0 version of the AAVLINK technology that addresses potential issues related to gene rearrangement and immune responses, enhancing the safety of clinical applications [2]. - Animal studies indicate that this technology can effectively improve behavioral and epilepsy phenotypes in relevant mouse models [2]. - Future research will focus on exploring the systemic delivery efficiency of the technology, understanding its mechanisms, establishing disease models, and conducting preclinical studies in primate models to facilitate its practical application [2].