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美国男婴患有因基因变异导致的罕见病，自今年2月以来接受了据称为世界首例的定制基因编辑疗法，目前效果良好。美国《新英格兰医学杂志》近日刊发研究报告介绍他的治疗情况，称这一病例为其他罕见病寻求基因疗法带来希望。
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