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 presentation reviews Axiomer editing oligonucleotides (EONs) structure activity, the potential of Axiomer platform as well as its applicability for the treatment of liver originated disorders.
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.
ProQR invented an entirely new way to treat genetic diseases. Axiomer enables RNA base editing by recruiting ADAR so your own body can treat disease. Learn more here.
This is Gerard Platenburg's presentation at the 5th International Conference on Base Editing, Prime Editing & Related Enzymes (Deaminet 2024) in San Diego, California.
In line with corporate strategy update in April, ProQR to focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas; update on initial pipeline targets expected in late 2022/early 2023
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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