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生物制药KNOWLEDGE BASE

The full Yeasen knowledge base, organised by two-level categories and six content types, with source links on every article.

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Total 334 articles · 生物制药 Clear

Applications干货 | 6个月定制一剂药!从KJ的奇迹看基因编辑如何改写“罕见病无解”定律

2025年5月,《新英格兰医学杂志》披露全球首个个体化CRISPR基因编辑疗法重大突破:研究团队以“6个月极速响应”,为罹患罕见病——氨基甲酰磷酸合成酶1(CPS1)缺陷症的9.5个月大婴儿完成全流程定制化治疗开发。通过碱基编辑技术精准修复致病突变,患儿血氨水平、体重等关键指标显著改善,标志着人类在“单患者基因精准治疗”领域实现“从0到1”的历史性跨越。罕见

📂 基因编辑 👁 9,327 🗓 2026-10-09
Total 334 items / 17 pagesPrev1…7891011…17Next

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