The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis.
- Resource Type
- Article
- Source
- PLoS ONE; 12/4/2019, Vol. 14 Issue 12, p1-23, 23p
- Subject
RECESSIVE genes FANCONI syndrome NONSENSE mutation INNER ear AMINOGLYCOSIDES LICENSES AMIKACIN - Language
- ISSN
- 19326203