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Updated: Jul 7, 2026

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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通过U7 snRNA介导的外原跳转来拯救衰变肌肉

Aurélie Goyenvalle1, Adeline Vulin, Françoise Fougerousse

  • 1Généthon & CNRS UMR 8115, 1, rue de l'Internationale, Evry, France.

Science (New York, N.Y.)
|November 6, 2004
PubMed
概括

这项研究表明,在mdx小鼠中,使用AAV介导的外显子跳转,持续产生功能性消毒素. 这种方法有效地通过恢复肌肉中的肌蛋白水平来纠正杜恩肌肉发育不良.

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