PlayBall10 schreef op 21 september 2017 14:09:
ProQR Presents in vivo Proof of Concept Data for the Axiomer® RNA Editing Platform Technology
Key updates
ProQR presents in vivo data in a relevant disease model, establishing proof of concept for its novel and proprietary Axiomer® RNA editing platform technology.
Axiomer® technology Editing Oligonucleotides (EONs) recruit endogenous ADAR to make single nucleotide changes to RNA in a highly specific and targeted manner.
This platform technology potentially has wide applicability for genetic diseases caused by single nucleotide G-to-A mutations.
In vivo proof of concept data will be presented at upcoming Oligonucleotide Therapeutics Society (OTS) Meeting on September 25, 2017.
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