Presented at TIDES Europe 2018 by Antti Aalto - ProQR Therapeutics
Axiomer Technology - Therapeutic oligonucleotides for directing site-specific A-to-I editing by endogenous ADAR enzymes
Presentation
Gepubliceerd op op TIDES Europe
Beschrijving
Axiomer® technology can introduce precise A-to-I modifications in endogenous RNA transcripts. This RNA editing enables e.g. the correction of G-to-A mutations, since inosines are interpreted as guanosines. RNA editing is achieved by Axiomer® Editing Oligonucleotides (EONs) that can direct site-specific deamination by endogenous ADARs (Adenosine Deaminases Acting on RNA). Rational EON design relies both on computational and empirical approaches to achieve high potency and drug-like properties.
