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AI赋能ProAPOBECs RNA碱基编辑系统,为自闭症等多种遗传疾病提供全新 ...
基因编辑技术正在重塑精准医疗的未来。与永久性的DNA编辑不同,RNA 编辑仅在转录本层面产生可逆修饰,因此被视为更安全的遗传疾病潜在治疗策略。然而,现有的C-to-U RNA编辑系统一直面临着效率低下、序列偏好性强(主要限于 UC 序列)、脱靶效应高等技术瓶颈,限制了其临床应用。如何突破这些技术壁垒?如何实现高效、精准、安全的体内RNA编辑?这正是科学家们急需解决的关键问题。
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