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透视病毒介导的基因补充可以拯救致病性ABCA3变体

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    bioRxiv : the preprint server for biology
    |September 5, 2025
    PubMed
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    基因添加疗法对ABCA3缺陷,严重的肺部疾病的原因显示出希望. 在特定模型中,ABCA3变异的补充部分恢复了细胞功能并减少了炎症.

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