Abstract
In this issue of Cell Chemical Biology, Shibata et al. (2020) rescue expression of CFTR from a defective gene by inhibiting splicing factors required for the inclusion of a pathogenic pseudo exon. Their work highlights the untapped potential of RNA splicing as a therapeutic target.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1453-1455 |
| Number of pages | 3 |
| Journal | Cell Chemical Biology |
| Volume | 27 |
| Issue number | 12 |
| DOIs |
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| State | Published - Dec 17 2020 |
| Externally published | Yes |
ASJC Scopus subject areas
- Biochemistry
- Molecular Medicine
- Molecular Biology
- Pharmacology
- Drug Discovery
- Clinical Biochemistry