Applicability of a structure-based modelling approach as a tool for in silico screening of oligonucleotides for targeted RNA editing presented at Oligonucleotide Therapeutics Society (OTS) 2018 by Julien Boudet
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.