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.
… a rare genetic disorder, with the objective to develop RNA therapies for rare diseases. Prior to founding ProQR, … In 1992 Dr. Valerio was appointed professor of gene therapy at the University of Leiden, where he also received …
