Axiomer® platform applicability and potential in therapeutically relevant models for targeted RNA editing presented at TIDES EU 2018 by Antti Aalto - ProQR Therapeutics
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 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.
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.