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Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin
Methods of inducing read-through of a nonsense mutation associated with ataxia telangiectasia, Rett syndrome or spinal muscular atrophy by erythromycin or azithromycin
Disclosed is treatment of genetic neurodegenerative or neurodevelopmental diseases that are caused by or associated with nonsense mutations or premature termination codons using macrolides. Further disclosed are methods for identifying agents that induce read-through of nonsense mutations and premature termination codons and uses thereof.
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