Abstract: The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions. Also, kits are provided which comprise the identified compounds and/or proteins in addition to suitable means for performing the methods of the invention.

Abstract: The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions. Also, kits are provided which comprise the identified compounds and/or proteins in addition to suitable means for performing the methods of the invention.

Abstract: The present invention relates to a method for identifying a pharmaceutically active compound that modulates the rpL35 (rpL35/rpL29)-dependent translation of at least one mRNA in a mammalian cell. The mRNA may comprise a premature termination codon (PTC), undergoes premature translation termination, causes programmed ?1 ribosomal frame shifting (?1PRF), or is a polycistronic mRNA. Furthermore a respective screening system, methods of treating or preventing a disease or condition, and compounds that modulate the rpL35 (rpL35/rpL29)-dependent translation, in particular atazanavir or derivatives thereof and artemisinin or artesunate or derivatives thereof are provided.

Abstract: The present invention relates to systems and methods for screening compounds and/or mutant ribosomal proteins in a eukaryotic cell that increase or decrease the translation of a target gene and thereby ameliorate or revert a defective and/or undesired translation of a target gene. Disclosed are compounds and proteins as identified with the methods and systems of the invention, pharmaceutical and cosmetic compositions thereof, their uses for the preparation of a medicament, methods of treatment of a disease or condition or cosmetic condition related to the defective translation of a gene, for example genetic diseases such as Epidermolysis bullosa, as well as diagnostic measures practical for the clinical evaluation of such diseases or conditions.