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Updated: Jul 19, 2025

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

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分支点作为基因疾病外型跳转疗法的潜在目标

Hiroaki Ohara1,2,3,4, Motoyasu Hosokawa1, Tomonari Awaya1,5

  • 1Department of Anatomy and Developmental Biology, Graduate School of Medicine, Kyoto University, Kyoto 606-8501, Japan.

Molecular therapy. Nucleic acids
|August 7, 2023
PubMed
概括

研究人员通过专注于分支点来针对福山先天性肌肉发育不良症 (FCMD) 的异常拼接. 分支点向的反感性寡核酸 (BP-AONs) 成功地恢复了患者细胞中正常的FKTN mRNA和蛋白质生产.

关键词:
MT:寡核酸:治疗方法和应用.这是一种反意义的寡核酸.分支点分支点分支点分支点异常跳过疗法治疗的异常跳过疗法肌肉发育不良 肌肉发育不良 肌肉发育不良拼接切换 拼接切换 拼接切换拼接监管要素 拼接监管元素

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