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Nature 447, 87-91 (22 April 2007) | doi:10.1038/nature05756;
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Associate Professor or Full Professor
- South Dakota State University
- Brookings, SD
Gastroenterologist
- Gastrointestinal Diseases Inc.
- Georgia, USA
PTC124 targets genetic disorders caused by nonsense mutations
Nonsense mutations promote premature translational termination and cause anywhere from 5–70% of the individual cases of most inherited diseases. Studies on nonsense-mediated cystic fibrosis have indicated that boosting specific protein synthesis from .
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