AGENTS FOR THE TREATMENT OF GENETIC DISEASES ARISING AS A RESULT OF NONSENS-MUTATIONS, AND METHODS OF IDENTIFICATION OF THESE AGENTS

摘要

Treatment of genetic neurodegenerative or neuroontogenetic diseases that are caused or associated with nonsense mutations or codons of premature termination using macrolides. Additionally, methods for identifying agents that induce end-to-end impregnation of nonsense mutations and termination codons and their use are described.