基因编辑革命正从实验室走向市场。美国批准了世界上首款采用Crispr技术的疗法，这一获得诺贝尔奖的发现有望成为修改基因以治疗疾病的强大新工具。
基因编辑革命正从实验室走向市场。
美国批准了世界上首款采用Crispr技术的疗法，这一获得诺贝尔奖的发现有望成为修改基因以治疗疾病的强大新工具。
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这款由Vertex Pharmaceuticals和Crispr Therapeutics共同开发的新疗法名为Casgevy，获准用于治疗严重的镰状细胞病患者。
美国食品药品管理局(FDA)做出的这一具有里程碑意义的决定预示着一种强大新型疗法的诞生，这种疗法可以关闭或替换基因，以解决长期令医生和研究人员束手无策的疾病。
几家公司正在开发基于Crispr的疗法，用于治疗心脏病、癌症和罕见遗传疾病。下一代基因编辑技术有望使这种疗法更容易实施，同时减少副作用。
Casgevy是为患者提供基于Crispr的治疗的第一步。与开发中的Crispr药物不同，Casgevy将在实验室而不是患者体内对细胞进行基因编辑。
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在体外对细胞进行基因编辑有助于确保治疗不会意外改变其他与目标疾病无关的基因。但这对患者来说是比较困难的方式，因为他们需要接受数天的高剂量化疗，以便为基因编辑后的细胞腾出空间。
FDA周五还批准了第二款镰状细胞病疗法，即由Bluebird Bio开发的Lyfgenia，该疗法将通过对患者的细胞进行基因编辑来产生血红蛋白替代品。Bluebird称其Lyfgenia的收费将为310万美元。