新基因编辑工具纠正小鼠脑部遗传疾病
研究人员发现一种新基因编辑疗法可以通过修复CHD3基因突变来治疗一种罕见的神经发育障碍——斯尼德勒斯-布洛克-康佩奥综合症。通过一种无害的病毒载体，将TadA嵌入的腺苷碱基编辑器（TeABE）送达小鼠模型，用于修复CHD3基因突变。
来源: Medical Xpress ｜ 阅读全文
发布时间: 2026-02-23 23:50 GMT+8（北京时间）
#基因编辑 #基因疗法 #神经发育障碍
Post #4067
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