ReviewAnnals of the New York Academy of Sciences2020
Small molecule targeting of RNA structures in neurological disorders.
Review in Annals of the New York Academy of Sciences, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 23 citations in OpenAlex.
- Review
- RNA dysregulation in neurodegenerative diseases.The EMBO journal · 2025Review
- Review
- Using the structural diversity of RNA: protein interfaces to selectively target RNA with small molecules in cells: methods and perspectives.Frontiers in molecular biosciences · 2023Review
- Partners in crime: Proteins implicated in RNA repeat expansion diseases.Wiley interdisciplinary reviews. RNA · 2022Review
- Systematically Studying the Effect of Small Molecules Interacting with RNA in Cellular and Preclinical Models.ACS chemical biology · 2021Review
- Novel Heteroaryl Compounds as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Novel Substituted Triazines as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Novel Substituted Pyridazines as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Novel Substituted Thiadiazoles as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Novel Substituted Pyridazines as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Novel Substituted Pyrazines as Splicing Modulators for Modulating Splicing of mRNA and Uses Thereof for Treating Diseases.ACS medicinal chemistry letters · 2021Article
- Affecting RNA biology genome-wide by binding small molecules and chemically induced proximity.Current opinion in chemical biology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Aberrant RNA structure and function operate in neurological disease progression and severity. As RNA contributes to disease pathology in a complex fashion, that is, via various mechanisms, it has become an attractive therapeutic target for small molecules and oligonucleotides. In this review, we discuss the identification of RNA structures that cause or contribute to neurological diseases as well as recent progress toward the development of small molecules that target them, including small molecule modulators of pre-mRNA splicing and RNA repeat expansions that cause microsatellite disorders such as Huntington's disease and amyotrophic lateral sclerosis. The use of oligonucleotide-based modalities is also discussed. There are key differences between small molecule and oligonucleotide targeting of RNA. The former targets RNA structure, while the latter prefers unstructured regions. Thus, some targets will be preferentially targeted by oligonucleotides and others by small molecules.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.