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Updated: Jul 15, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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消极性肌痛病诊断和治疗方面的进展

Fawzy A Saad1, Gabriele Siciliano2, Corrado Angelini3

  • 1Department of Gene Therapy, Saad Pharmaceuticals, Juhkentali 8, 10132 Tallinn, Estonia.

Biomolecules
|September 28, 2023
PubMed
概括

肌肉发育不良症,因肌肉发育不良基因突变而产生的X关联肌肉疾病,包括杜申肌肉发育不良症和扩展性心肌病变. 诊断和基因疗法的进步,如外跳转,为这些疾病提供了更好的护理和早期检测.

关键词:
贝克尔肌肉缩症 贝克尔肌肉缩症基因编辑CRISPR基因编辑杜申尼肌肉发育不良症 杜申尼肌肉发育不良症扩张性心肌病症 扩张性心肌病症消极性表皮病 消极性表皮病 消极性表皮病基因药物:基因药物是一种基因药物.基因治疗的基因疗法药理疗法是一种药理疗法.主要基因编辑 基因编辑血清中的肌酸激酶酶是什么?

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