基因快递卡车(多功能基因递送系统)

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“基因快递卡车”实现多功能精准递送
Ke Ji Ri Bao· 2025-05-27 23:49
Core Insights - A research team funded by the National Institutes of Health has developed a "gene delivery truck," a multifunctional system capable of precisely targeting various types of neural cells in the brain and spinal cord, laying the groundwork for future precision gene therapies for brain disorders [1][2] - This delivery system can accurately transport genetic material to specific cells in the brain and spinal cord for research or therapeutic purposes, revolutionizing the way scientists explore neural circuits and providing a new tool for neuroscience research without relying on transgenic animals [1][2] Group 1 - The new system is based on a modified adeno-associated virus as a vector, which can efficiently deliver DNA to target cells, demonstrating broad applicability validated across multiple species and experimental systems, including surgically removed brain tissue samples [1] - The toolkit includes dozens of delivery systems targeting key brain cell types, such as excitatory neurons, inhibitory interneurons, and neurons affected in diseases like amyotrophic lateral sclerosis and spinal muscular atrophy [2] - An AI-supported computational program is included in the platform to identify "light switches" that activate gene expression in specific brain cells, significantly reducing the time and effort needed to find such regulatory elements [2] Group 2 - This series of innovative tools is expected to greatly advance the understanding of human brain function and diseases, particularly targeting specific cells in the prefrontal cortex associated with decision-making and other higher cognitive functions [2] - The development represents a significant leap in neuroscience and gene therapy, opening up new avenues for revealing the mysteries of the human brain and developing novel therapeutic strategies for various neurological and psychiatric disorders [2]