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PTC124 targets genetic disorders caused by nonsense mutations.
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PTC124 targets genetic disorders caused by nonsense mutations.
PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 03; 447(7140):87-91.
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PubMed
subject areas
Alleles
Animals
Biological Availability
Codon, Nonsense
Dystrophin
Genetic Diseases, Inborn
Humans
Mice
Mice, Inbred mdx
Oxadiazoles
Phenotype
Protein Biosynthesis
RNA, Messenger
Substrate Specificity