Modification of a guide RNA can mimic the effect of hyperactive mutants and advance the approach of recruiting endogenous ADARs for site-directed RNA editing reported in Journal of the American Chemical Society (JACS) by Doherty EE et al.
This presentation highlights the therapeutic possibilities of Axiomer platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
This is Gerard Platenburg's presentation at the 5th International Conference on Base Editing, Prime Editing & Related Enzymes (Deaminet 2024) in San Diego, California.
What is RNA and how can we use the body's own RNA editing machinery to treat disease? Learn more about the Axiomer® RNA editing technology and how we are creating a new class of medicines.
… Axiomer technology - RNA base editing ProQR invented an entirely new way to treat genetic … way to use RNA oligonucleotides to alter RNA. The Axiomer™ RNA editing technology enables the editing of specific single …
… ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs In … to focus exclusively on the development of the Axiomer ® RNA editing technology platform across multiple therapeutic … sepofarsen and will now focus exclusively on its Axiomer ® RNA-editing technology platform. Following the results from …
LEIDEN, Netherlands & CAMBRIDGE, Mass., June 18, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will participate in the RNA Editing Summit, June 18-20, 2024, in Boston, Mas