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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 bmp...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2214458475

Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4st72 allele in zebrafish

About this item

Full title

Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4st72 allele in zebrafish

Publisher

United States: Public Library of Science

Journal title

PloS one, 2019, Vol.14 (4), p.e0212121-e0212121

Language

English

Formats

Publication information

Publisher

United States: Public Library of Science

More information

Scope and Contents

Contents

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...

Alternative Titles

Full title

Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4st72 allele in zebrafish

Authors, Artists and Contributors

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_plos_journals_2214458475

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2214458475

Other Identifiers

ISSN

1932-6203

E-ISSN

1932-6203

DOI

10.1371/journal.pone.0212121

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