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Updated: Jul 11, 2025

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane
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针对性基因组编辑恢复了成年小鼠的听觉功能,这些小鼠因人类微RNA突变引起的渐进性听力损失

Wenliang Zhu, Wan Du, Arun Prabhu Rameshbabu

    bioRxiv : the preprint server for biology
    |November 14, 2023
    PubMed
    概括

    基因组编辑成功地恢复了成年小鼠的听力,这些小鼠具有微RNA-96突变. 这种新疗法为治疗主导迟发性听力损失 (DFNA50.0) 提供了希望.

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    In-vitro Mutagenesis01:16

    In-vitro Mutagenesis

    To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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