Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp...
Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4st72 allele in zebrafish
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United States: Public Library of Science
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English
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United States: Public Library of Science
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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 nonsense variants in human Bone Morphogenetic Protein 4...
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Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4st72 allele in zebrafish
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TN_cdi_plos_journals_2214458475
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2214458475
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1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0212121