Core Viewpoint - The article highlights a groundbreaking achievement in neuroscience, specifically in gene editing for treating rare genetic disorders like Alternating Hemiplegia of Childhood (AHC), marking a significant milestone in personalized gene therapy [1][2]. Group 1: Research Breakthrough - Researchers successfully corrected gene mutations in the brain associated with AHC using a single injection technique, significantly improving symptoms and extending the lifespan of affected mice [1][2]. - The study, published in the journal Cell, involved a collaboration among institutions including the Jackson Laboratory and the Broad Institute, focusing on two common mutation sites in the ATP1A3 gene [2][3]. Group 2: Gene Editing Technology - The research tested next-generation gene editing technologies, with Prime Editing showing remarkable results by repairing up to 85% of mutated brain cells and restoring normal protein function [2][3]. - The use of a harmless virus, AAV9, as a delivery vehicle to cross the blood-brain barrier was a key innovation, allowing for effective gene editing in the brain [4][6]. Group 3: Implications for Treatment - This advancement opens new possibilities for treating not only AHC but also other hereditary neurological disorders, potentially transforming the approach to previously deemed "incurable" diseases [5][6]. - The combination of the viral delivery system and Prime Editing technology is seen as a revolutionary step in making precise brain gene editing a reality, with ongoing tests exploring the feasibility of intervention after symptoms appear [6].