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单次剂量的CRISPR疗法NTLA-2002在III期试验中将遗传性血管性水肿发作减少87%。了解这将如何改变治疗并铺平治愈之路。
首个成功的体内基因编辑三期试验:lonvo-z疗法将HAE发作减少87%。了解这一突破及预防性治疗的终止。