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Updated: Jul 14, 2026

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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使用MYOD1转化的人体尿液衍生细胞作为杜申尼肌肉衰竭中的表跳转疗法的预测平台的协议

Katsuhiko Kunitake1, Takami Ishizuka2, Eri Takeshita3

  • 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), Kodaira, Tokyo 187-8502, Japan.

STAR protocols
|June 8, 2025
PubMed
概括

反感性寡核酸疗法在杜申肌肉发育不良 (DMD) 方面表现有前途. 这项研究详细介绍了一种新方法,用于测试患者衍生细胞中的外子跳转疗效,以帮助发现DMD的药物.

关键词:
细胞生物学 细胞生物学细胞分化的过程细胞培养培养的细胞培养.细胞隔离细胞的隔离.遗传学 是一个遗传学.卫生科学 卫生科学 卫生科学分子生物学分子生物学干细胞是干细胞.

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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...