Mutations leading to premature termination codons (PTCs) are the cause of 10-15% of all inherited genetic disorders. The presence of a PTC within the open reading frame of a gene leads to the degradation of mRNA and the synthesis of truncated, non-functional polypeptides. Both events eventually limit the amount of protein that can be formed and are responsible for severe phenotypes as observed for many patients. Currently, there are no means to treat this class of diseases. We develop tRNA-based strategies to readthrough PTC and eventually generate a full-length, functional protein. Thus, we aim to take the first steps in creating a therapeutic that can help millions people worldwide suffering from PTC-caused diseases.
