期刊
DRUG DISCOVERY TODAY
卷 26, 期 4, 页码 1030-1039出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.drudis.2020.12.019
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资金
- ABIVAX
Inflammatory diseases are believed to result from dysregulated inflammatory responses to environmental factors on susceptible genetic backgrounds. MiRNAs, such as miR-124, are endogenous, small noncoding RNAs that play a role in regulating gene expression involved in inflammation. ABX464, a small quinoline compound, has shown promising efficacy in ulcerative colitis by upregulating miR-124 and restoring physiological pathways lost in inflammatory diseases.
Inflammatory diseases are believed to develop as a result of dysregulated inflammatory responses to environmental factors on susceptible genetic backgrounds. Operating at the level of post-transcriptional gene regulation, miRNAs are a class of endogenous, small noncoding RNAs that can promote downregulation of protein expression by translational repression and/or mRNA degradation of target mRNAs involved in inflammation. MiR-124 is a crucial modulator of inflammation and innate immunity that could provide therapeutic restitution of physiological pathways lost in inflammatory diseases. A recently discovered small quinoline, ABX464, was shown to upregulate miR-124 in human immune cells. In vivo, in a proof-of-concept clinical study, ABX464 showed robust and consistent efficacy in ulcerative colitis (UC). In this review, we examine the current therapeutic options proposed for UC and discuss the drug candidate ABX464 in this context.
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