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Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4(st72) allele in zebrafish
Nonsense suppression therapy (NST) utilizes compounds such as PTC124 (Ataluren) to induce translational read-through of stop variants by promoting the insertion of near cognate, aminoacyl tRNAs that yield functional proteins. We used NST with PTC124 to determine if we could successfully rescue nonse...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Public Library of Science
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6481805/ https://www.ncbi.nlm.nih.gov/pubmed/31017898 http://dx.doi.org/10.1371/journal.pone.0212121 |