Hidtil ukendte aminoglycosider og anvendelser deraf ved behandling af genetiske lidelser

摘要

A new class of paromomycin-derived aminoglycosides (Formula I), which exhibit efficient stop-codon mutation suppression activity, low cytotoxicity and selectivity towards eukaryotic cells are provided. Also provided are processes of preparing these paromomycin-derived aminoglycosides and intermediates thereof, as well as pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders.