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Updated: Sep 10, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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杜申肌肉衰竭的基因疗法

Yuko Shimizu-Motohashi1

  • 1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan..

Brain & development
|August 23, 2025
PubMed
概括

使用腺相关病毒 (AAV) 治疗杜申肌肉衰竭 (DMD) 的基因治疗具有前景,但面临挑战. 目前正在进行的研究旨在提高DMD患者的安全性,有效性和可访问性.

关键词:
AAV 载体在CRISPR/Cas9中杜申肌肉衰竭基因治疗免疫反应微型氨酸转基因表达

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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...
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