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体内CRISPR治ATTR、HAE的后期临床（Intellia 系）
体内 CRISPR 以 LNP 递送 Cas9/sgRNA，一次注射敲除致病基因；Intellia 在 ATT…
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Intellia 系体内 CRISPR 概览：技术与管线
一、技术原理 Intellia Therapeutics 是全球第一个将 CRISPR-Cas9 直接用于人体…
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