From experimental NMR data towards a software tool to predict optimal sequences for therapeutic morpholino oligonucleotides KU Leuven
Phosphorodiamidate morpholino oligonucleotides (PMOs) are synthetic single-stranded RNA-like molecules that have recently entered the market for the treatment of Duchenne Muscular Dystrophy. PMOs are antisense oligonucleotides that, by binding to complementary RNA sequences, modulate the expression of specific genes, including those of proteins that are considered as ‘undruggable’ for compounds that directly target proteins. Therefor, they ...