Nature：全员中文属名，仇子龙/程田林/李斐团队利用体内碱基编辑，在出生后治疗神经发育障碍，改善自闭症样行为

Core Viewpoint - The research highlights the potential of in vivo base editing as a viable strategy to alleviate symptoms associated with Snijders Blok-Campeau Syndrome (SNIBCPS), a genetic neurodevelopmental disorder caused by mutations in the CHD3 gene [2][10]. Group 1: Disease Overview - Snijders Blok-Campeau Syndrome (SNIBCPS) is an autosomal dominant genetic disorder caused by pathogenic mutations in the CHD3 gene, first described in 2018, with over 100 reported cases [5]. - Key clinical features of SNIBCPS include global developmental delay, language delay, intellectual disability, hypotonia, facial dysmorphism, and brain structural abnormalities, often accompanied by autism spectrum disorder (ASD) [5]. Group 2: Research Findings - The research team developed a humanized mouse model (Chd3 hR1025W/+) that exhibited communication, cognitive, and autism-related behavioral deficits [3][7]. - A base editor, TeABE, was designed to correct the A•T base pair mutation, and intravenous delivery via a dual AAV system restored CHD3 protein levels and improved cognitive and autism-like behaviors in the mouse model [3][8]. - In non-human primates (NHP), intrathecal delivery of AAV9 carrying TeABE achieved widespread brain transduction and base editing, supporting the clinical translational feasibility of this approach [3][8]. Group 3: Implications for Treatment - The findings establish in vivo base editing as a feasible treatment method for CHD3-related neurodevelopmental disorders, demonstrating that precise single-base editing in the brain postnatally can restore protein dosage and function [10].