ReviewDrug discovery today2013
Rational design of chemical genetic probes of RNA function and lead therapeutics targeting repeating transcripts.
Review in Drug discovery today, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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.
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Who cites it
16 citing papers in PubMed, 26 citations in OpenAlex.
- Repurposing Oseltamivir Against CACS omega · 2025Article
- Novel Quinazoline Derivatives Inhibit Splicing of Fungal Group II Introns.ACS chemical biology · 2025Article
- HT-SELEX-based identification of binding pre-miRNA hairpin-motif for small molecules.Molecular therapy. Nucleic acids · 2022Article
- Discovery of a potent small molecule inhibiting Huntington's disease (HD) pathogenesis via targeting CAG repeats RNA and Poly Q protein.Scientific reports · 2019Article
- Insights into the development of chemical probes for RNA.Nucleic acids research · 2018Article
- Targeting RNA in mammalian systems with small molecules.Wiley interdisciplinary reviews. RNA · 2018Review
- Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes.Nature communications · 2018Article
- RNA Dysregulation in Amyotrophic Lateral Sclerosis.Frontiers in genetics · 2018Review
- A flow cytometry-based screen identifies MBNL1 modulators that rescue splicing defects in myotonic dystrophy type I.Human molecular genetics · 2017Article
- Review
- Structural features of a 3' splice site in influenza a.Biochemistry · 2015Article
- Inhibition of Non-ATG Translational Events in Cells via Covalent Small Molecules Targeting RNA.Journal of the American Chemical Society · 2015Article
- Structure based approaches for targeting non-coding RNAs with small molecules.Current opinion in structural biology · 2015Review
- Article
- Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs.Nucleic acid therapeutics · 2014Article
- Oligonucleotide-based strategies to combat polyglutamine diseases.Nucleic acids research · 2014Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
RNA is an important yet vastly underexploited target for small molecule chemical probes or lead therapeutics. Small molecules have been used successfully to modulate the function of the bacterial ribosome, viral RNAs and riboswitches. These RNAs are either highly expressed or can be targeted using substrate mimicry, a mainstay in the design of enzyme inhibitors. However, most cellular RNAs are neither highly expressed nor have a lead small molecule inhibitor, a significant challenge for drug discovery efforts. Herein, I describe the design of small molecules targeting expanded repeating transcripts that cause myotonic muscular dystrophy (DM). These test cases illustrate the challenges of designing small molecules that target RNA and the advantages of targeting repeating transcripts. Lastly, I discuss how small molecules might be more advantageous than oligonucleotides for targeting RNA.
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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.