Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner
Around 12% of hereditary disease-causing mutations are in-frame nonsense mutations. The expression of genes containing nonsense mutations potentially leads to the production of truncated proteins with residual or virtually no function. However, the translation of transcripts containing premature sto...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-08-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/14/8/785 |