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Updated: Aug 12, 2026

Exogenous Administration of Microsomes-associated Alpha-synuclein Aggregates to Primary Neurons As a Powerful Cell Model of Fibrils Formation
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Gys1反意义疗法在拉福拉病小鼠模型中预防疾病驱动的聚合物和发性排放

Katherine J Donohue1, Bethany Fitzsimmons2, Ronald C Bruntz1

  • 1Department of Molecular and Cellular Biochemistry, University of Kentucky, Lexington, KY, 40506, USA.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
|September 12, 2023
PubMed
概括

一种针对糖原合成酶1 (Gys1) 的反感性寡核酸减少了拉弗拉病的小鼠模型中的拉弗拉体形成和疾病症状. 这种Gys1-ASO疗法为导致这种神经退行性疾病的EPM2B突变提供了潜在的治疗方法.

关键词:
这是一种反意义的寡核酸.葡萄糖原是什么 葡萄糖原是什么糖原储存疾病是糖原储存疾病.糖原合成酶的使用方法拉福拉病是拉福拉的疾病.

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相关概念视频

G-protein Coupled Receptors01:21

G-protein Coupled Receptors

G-protein coupled receptors are ligand binding receptors that indirectly affect changes in the cell. The actual receptor is a single polypeptide that transverses the cell membrane seven times creating intracellular and extracellular loops. The extracellular loops create a ligand specific pocket which binds to neurotransmitters or hormones. The intracellular loops holds onto the G-protein.