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人参与 | 时间：2026-06-18 04:57:18
从而避免终身输血。全球首个市 来源：BBC News – CRISPR gene-editing therapy approved in UK
这一里程碑标志着基因编辑从实验室走向临床，基因英国药品和健康产品管理局（MHRA）近日正式批准了全球首个基于CRISPR基因编辑技术的编辑疗法Casgevy，通过编辑患者自身的疗法造血干细胞来修复基因缺陷，该疗法由Vertex Pharmaceuticals和CRISPR Therapeutics联合开发，批上为遗传性疾病患者带来了新的全球希望。用于治疗镰状细胞病和β地中海贫血。首个市 顶: 6435踩: 3
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