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用一个双胺抑制剂向导致1型肌性衰竭 (DM1) 的有毒RNA

Chun-Ho Wong1, Lien Nguyen, Jessie Peh

  • 1Department of Chemistry, University of Illinois at Urbana-Champaign , 600 South Mathews Avenue, Urbana, Illinois 61801, United States.

Journal of the American Chemical Society
|April 8, 2014
PubMed
概括

一种新型化合物 - - 联体3 - - 能够有效地破坏1型肌性衰竭 (DM1) 细胞中的毒性相互作用. 这一发现为通过恢复正常细胞功能来治疗DM1提供了有希望的新疗法.

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