这项发表在 Nature Biotechnology 上的工作，通过单次 AAV 注射在活体中实现了显著的酶活性恢复。这为未来的临床试验提供了新的思路：或许我们不再需要针对每种病单独开发药物，而是可以采用“篮子试验”（Basket Trial） 设计——不管患有哪种遗传病，只要基因检测显示“UGA 无义突变”，就有望进入同一个治疗篮子。

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引言 在炎症性肠病（Inflammatory Bowel Disease, ...

新泽西州普林斯顿 - 临床阶段生物技术公司 PDS Biotechnology Corporation (NASDAQ:PDSB) ，市值约5100万美元，周四宣布美国专利局已为其领先的癌症免疫疗法资产PDS0101发出授权通知。

南京林业大学 林草学院、水土保持学院珍贵树种研究团队在《Plant Biotechnology Journal》上在线发表了题为“Two Indel Variations in the Regulated Carotenoid Pigment ...

引言 如果说基因编辑技术是一把能够改写生命密码的“上帝手术刀”，那么递送系统就是握住这把刀的手。早在1908年，诺贝尔奖得主保罗·埃尔利希就提出了“魔弹”构想。 1月12日，Jennifer A. Doudna团队在《Nature ...

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