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 |
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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
- Drug Discovery
- Molecular Medicine
- Molecular Biology
- Biochemistry
- Clinical Biochemistry
- Pharmacology