PTC124

A drug similar to gentamicin that has been developed to treat genetic diseases in which a mutation causes a premature stop in a gene so that the full normal protein product is not produced. PTC124 binds to a component in the transcription process allowing it to continue past the stop point. The phenomenon was first noted with gentamicin and the new drug has been developed tpo be safer than this antibiotic and more easily absorbed. Trials on mice with CYSTIC FIBROSIS and muscular dystrophy had produce encouraging results. About a third of genetic diseases are caused by a premature STOP MUTATION.

单词	ptc124
释义	PTC124 PTC124 A drug similar to gentamicin that has been developed to treat genetic diseases in which a mutation causes a premature stop in a gene so that the full normal protein product is not produced. PTC124 binds to a component in the transcription process allowing it to continue past the stop point. The phenomenon was first noted with gentamicin and the new drug has been developed tpo be safer than this antibiotic and more easily absorbed. Trials on mice with CYSTIC FIBROSIS and muscular dystrophy had produce encouraging results. About a third of genetic diseases are caused by a premature STOP MUTATION.
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