Intravitreal administration of small molecule read-through agents demonstrate functional activity in a nonsense mutation mouse model
Author:
Publisher
Elsevier BV
Subject
Cellular and Molecular Neuroscience,Sensory Systems,Ophthalmology
Reference39 articles.
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3. The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis;Brasell;PloS One,2019
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5. Suppression of a nonsense mutation in mammalian cells in vivo by the aminoglycoside antibiotics G-418 and paromomycin;Burke;Nucleic Acids Res.,1985
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