Presented at the RNA Editing Summit on July 12, 2023 by Lenka Van Sint Fiet - ProQR Therapeutics
Harnessing chemical modifications to improve ADAR potency and unlock Axiomer broad applicability
Presentation
Published on at RNA Editing Summit
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The 2023 edition of the RNA Editing Summit focused on optimizing RNA editing technology to safely deliver novel payloads, enhancing potency and durability to achieve clinical validation in patients faster. This presentation details the ongoing effort led by ProQR to put in place effective strategies to increase RNA editing yields of ADARs using chemical modifications and oligonucleotide design. The potential of the Axiomer platform in multiple organs, as well as its applicability for the treatment of liver-originated disorders, including data from preclinical models are also included.
