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马萨诸塞州剑桥 - 临床阶段生物技术公司Wave Life Sciences Ltd. (NASDAQ:WVE)宣布在其用于治疗α-1抗胰蛋白酶缺乏症(AATD)的WVE-006疗法的RNA编辑方面取得重大突破。该公司公布了其Phase 1b/2a RestorAATion-2研究的积极机制证明数据,标志着首次在人体中成功实现RNA编辑的临床示范。 这项研究涉及两名携带AATD纯合子ZZ基因型的患 ...