Unlocking the Potential of Innovative Editing Oligonucleotides (EONs) to Address Liver Originated Disorders
Gepubliceerd op op TIDES USA
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The Axiomer® platform uses editing oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR). The therapeutic possibilities of this editing platform are not only 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 with the potential to prevent diseases. Through now further developed design principles there has been an established improvement of specificity and efficacity of EONs. The presentation reviews EONs structure activity, the potential of Axiomer platform as well as its applicability for the treatment of liver originated disorders.
