核糖体关联质量控制因子ASCC3的CRISPR激活改善了小鼠的脆弱X综合征表型

Ji Geng1, Xiying Wang1, Jie Pan1

  • 1Department of Pathology, Stanford University School of Medicine, Stanford, CA 94305, USA.

PubMed
概括

脆弱X综合征 (FXS) 与FMRP损失有关. 这项研究揭示了FMRP通过ASCC3调节碰撞的核糖体,影响翻译,并为FXS提供潜在的治疗点.

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