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柳叶刀 | 针对眼耳疾病的基因治疗有哪些挑战?
《柳叶刀》(The Lancet) 发表了一项使用基因补充疗法治疗AIPL1相关视网膜营养不良儿童的首次人体(first-in-human)干预性研究。该疾病会导致患儿从出生起就出现严重的视力损害。在接受治疗后, ...
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