Nonsense mutations, single nucleotide changes which replaces a codon that encodes for an amino acid with a stop codon resulting in truncated protein products, give rise to approximately 11% of inherited diseases in humans. Nonsense suppression therapeutics may act as viable treatments for such diseases by allowing ribosomes to read through premature termination codons and […]

In the present day, the vast majority of pharmaceutical therapeutics target specific proteins or enzymatic reactions. However, due to conformational challenges, in addition to other issues, a wide number of such proteins remain “undruggable” with current technologies. One solution put forward to address these issues has been to target these proteins before they undergo translation, […]

Upstream open reading frames (uORFs) are sequences with start and stop codons found upstream of the main protein coding region in an area known as the 5’ untranslated region (UTR). uORFs can vary by number per transcript, by length and by their position in relation to the 5’ cap and main start codon. uORFs act […]