Abstract
RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.
| Original language | English |
|---|---|
| Pages (from-to) | 223-231.e4 |
| Journal | Cell Chemical Biology |
| Volume | 27 |
| Issue number | 2 |
| DOIs | |
| State | Published - 20 Feb 2020 |
| Externally published | Yes |
Keywords
- RNA
- chemical biology
- click chemistry
- drug design
- intron retention
- medicinal chemistry
- microsatellite disease
- myotonic dystrophy
- nucleic acids
- repeat expansion disorder
All Science Journal Classification (ASJC) codes
- Biochemistry
- Molecular Medicine
- Molecular Biology
- Pharmacology
- Drug Discovery
- Clinical Biochemistry