RNase-H 介导的中枢神经系统沉默证明是具有挑战性的。
RNase-H-mediated silencing in the CNS proves predictably nontrivial.
机构信息
Department of Paediatrics, University of Oxford, UK.
Department of Paediatrics, University of Oxford, UK.
出版信息
Med. 2022 Nov 11;3(11):733-734. doi: 10.1016/j.medj.2022.10.004.
PMID:36370693
Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease primarily affecting motor neurons. Recently, Miller et al. presented phase 3 data for the RNase-H-recruiting antisense oligonucleotide (ASO) targeting superoxide dismutase 1 (SOD1) in ALS, which represents a step forward in the deployment of ASO therapeutics against CNS targets.
摘要
肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，主要影响运动神经元。最近，Miller 等人公布了针对 ALS 中超氧化物歧化酶 1（SOD1）的 RNA 内切酶-H 招募反义寡核苷酸（ASO）的 3 期数据，这是在针对中枢神经系统靶点的 ASO 治疗药物的应用方面向前迈出的一步。
Zotero 插件