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Ataluren suppresses a premature termination codon in an MPS I-H mouse

Ataluren suppresses a premature termination codon in an MPS I-H mouse

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9329424

Ataluren suppresses a premature termination codon in an MPS I-H mouse

About this item

Full title

Ataluren suppresses a premature termination codon in an MPS I-H mouse

Publisher

Berlin/Heidelberg: Springer Berlin Heidelberg

Journal title

Journal of molecular medicine (Berlin, Germany), 2022-08, Vol.100 (8), p.1223-1235

Language

English

Formats

Publication information

Publisher

Berlin/Heidelberg: Springer Berlin Heidelberg

More information

Scope and Contents

Contents

Abstarct
Suppressing translation termination at premature termination codons (PTCs), termed readthrough, is a potential therapy for genetic diseases caused by nonsense mutations. Ataluren is a compound that has shown promise for clinical use as a readthrough agent. However, some reports suggest that ataluren is ineffective at suppressing PTCs. T...

Alternative Titles

Full title

Ataluren suppresses a premature termination codon in an MPS I-H mouse

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9329424

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9329424

Other Identifiers

ISSN

0946-2716

E-ISSN

1432-1440

DOI

10.1007/s00109-022-02232-0

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