用反义寡核苷酸治疗肌萎缩侧索硬化症。
InFUSing antisense oligonucleotides for treating ALS.
机构信息
Centre de ressources et de compétences sur la SLA, Centre Hospitalier Universitaire d'Angers, Angers, France; Laboratoire de Neurobiologie et Neuropathologie, Centre Hospitalier Universitaire d'Angers, Angers, France; Univ Angers, Inserm, CNRS, MITOVASC, SFR ICAT, Angers, France.
Centre de ressources et de compétences sur la SLA, Centre Hospitalier Universitaire d'Angers, Angers, France; Univ Angers, Inserm, CNRS, MITOVASC, SFR ICAT, Angers, France.
出版信息
Trends Mol Med. 2022 Apr;28(4):253-254. doi: 10.1016/j.molmed.2022.02.006. Epub 2022 Mar 1.
The question of a loss or toxic gain of function in FUS-related amyotrophic lateral sclerosis is still debated. Recently, Korobeynikov et al. argued that FUS mutations lead to a gain of function and showed that lowering wild-type and mutant FUS levels could be a promising therapeutic strategy.
在 FUS 相关性肌萎缩侧索硬化症中，功能丧失或毒性获得的问题仍存在争议。最近，Korobeynikov 等人认为 FUS 突变导致功能获得，并表明降低野生型和突变型 FUS 水平可能是一种有前途的治疗策略。
Zotero 插件