Article

Ataluren Suppresses a Premature Termination Codon in an MPS I-H Mouse

2022-03-14

Abstract excerpt

<title>Abstract</title> <p>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. To further evaluate the effectiveness of ataluren as...

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Literature Corpus work: b26f845a-0954-5a4b-a365-58efd0a3234b
DOI: 10.21203/rs.3.rs-1416568/v1

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