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Updated: Jan 17, 2026

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
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对阿舍尔综合征,2A型的AAV介导的外因子跳转疗法

Stephanie A Mauriac1, Jiyoon Lee1, Jingyuan Zhang1

  • 1Department of Otolaryngology, F.M Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|September 25, 2025
PubMed
概括

使用矢量化反感性寡核酸 (ASO) 的新基因疗法策略显示,它对治疗阿舍尔综合征2A型 (USH2A) 的治疗具有前途. 这种方法旨在通过单次局部注射来纠正最常见的USH2A基因突变来恢复视力和听力.

关键词:
在 AAV AAV AAV 中.在ASO的基础上,ASO是ASO.在USH2A中使用.阿舍尔综合征是什么意思阿舍尔综合征是什么意思腺相关病毒的病毒.这是一种反意义的寡核酸.这是一种聋盲症,聋盲症.内耳的有机体内耳.视网膜的有机体接待员在里面

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