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
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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.
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… was diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Before founding … background in RNA modulation and orphan drug discovery and development and is currently in charge of our R&D. Gerard has … has led in the development of structure-guided methods for optimizing chemically modified oligonucleotides for …
