Variable readthrough responsiveness of nonsense mutations in hemophilia A
Readthrough therapy relies on the use of small molecules that enable premature termination codons in mRNA open reading frames to be misinterpreted by the translation machinery, thus allowing the generation of full-length, potentially functional proteins from mRNA carrying nonsense mutations. In pati...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Ferrata Storti Foundation
2020-02-01
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| Series: | Haematologica |
| Online Access: | https://haematologica.org/article/view/9535 |