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Evolutionarily conserved viral integration sites can serve as genomic safe harbors for human gene therapy, with at least six specific loci demonstrating tolerance to transgene insertion and low transcriptome disturbance. Novelty: ClaimNovelty.NOVEL_FINDING. Consensus alignment: ConsensusAlignment.NEUTRAL.

November 1, 2025iScience1 citationsOpen Access

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发展进化保守的病毒整合位点作为人类基因治疗的安全港

MQMarco A. Quezada‐RamírezUniversity of Massachusetts Chan Medical School MCMatthew A. CampbellUniversity of Alaska Fairbanks KPKrishna Mohan ParsiUniversity of Massachusetts Chan Medical School

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Abstract

HSPCs。这导致至少六个位点对转基因插入耐受，低转录组干扰，以及三个显示髓系免疫分支特异性调控的位点。因此，我们的方法产生了一份适合人类细胞治疗性转基因的候选GSH目录。

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发展进化保守的病毒整合位点作为人类基因治疗的安全港

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