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Updated: Jul 16, 2025

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基础编辑纠正了常见的萨拉病SLC17A5 c.115C>T变体

Jerry F Harb1, Chloe L Christensen1, Shih-Hsin Kan1

  • 1CHOC Children's Research Institute, Orange, CA 92868, USA.

Molecular therapy. Nucleic acids
|September 20, 2023
PubMed
概括

腺基编辑 (ABE) 显示了通过纠正致病SLC17A5变体来治疗自由酸储存障碍 (FSASDs) 的前景. 这种基因疗法方法有效地减少了细胞模型中的疾病病理,为一种新的治疗提供了希望.

关键词:
克里斯普尔-Cas9是什么意思MT:编辑RNA/DNA的方法在 SLC17A5A5 中.萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨拉萨自由的酸储存疾病自由的酸储存疾病

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