In these mutations, a codon that codes for an amino acid is changed into a
preventing translation of the complete, functional protein.
This type of mutation can be found in nearly all known monogenetic diseases including most rare diseases,
summing up to 5,000 – 10,000 diseases or tens of million patients worldwide. It is a viable yet challenging therapeutic strategy to treat these PTCs with
translational readthrough-inducing drugs (TRIDs).