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Updated: Jan 6, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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消极性肌肉变质症 (Dystrophinopathies) 是一种肌肉变质的疾病.

Divya Jayaraman, Partha S Ghosh

    Continuum (Minneapolis, Minn.)
    |October 2, 2025
    PubMed
    概括

    肌肉发育不良症,就像杜恩和贝克尔肌肉发育不良症一样,是X关联的肌肉疾病. 新的基因疗法提供了希望,但它们的长期影响和安全性需要进一步研究.

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