292-PERT：基因编辑安装抑制tRNA用于疾病治疗

该文献介绍了一种名为 PERT（prime editing-mediated readthrough of premature termination codons）的创新基因组编辑疗法，旨在解决由无义突变（提前终止密码子，PTC）引起的遗传疾病。PERT利用Prime Editing (PE) 技术，将基因组中一个可有可无的内源性转移 RNA (tRNA) 永久转化为一个经过优化的抑制性 tRNA (sup-tRNA)。通过对数千种tRNA变体进行迭代筛选，研究人员成功设计出高效的sup-tRNA，该 sup-tRNA 能够以单基因拷贝的内源性表达水平实现有效的蛋白质拯救。这种与疾病无关的策略在多个疾病的人类细胞模型（如巴滕病和泰-萨克斯病）中展示了酶活性恢复，并在赫尔勒综合征小鼠模型中实现了疾病病理学的近乎完全的拯救。重要的是，研究发现PERT具有高度的安全性，没有检测到脱靶编辑或对天然终止密码子 (NTCs) 的广泛通读，这表明它有望成为治疗大量遗传疾病患者的持久性单次疗法。References: Pierce S E, Erwood S, Oye K, et al. Prime editing-installed suppressor tRNAs for disease-agnostic genome editing[J]. Nature, 2025: 1-12.前往小宇宙评论区与主播互动

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292-PERT：基因编辑安装抑制tRNA用于疾病治疗

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