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Nature 447, 87-91 (22 April 2007) | doi:10.1038/nature05756;
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Assistant Professor of Medicine
- Massachusetts General Hospital
- Boston, MA
Senior Scientific Manager / Chief Scientific Manager for Neuroscience Group In Vivo Pharmacology / Biology
- Syngene International
- Bangalore, Karnataka 560099 India
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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