Presented at Oligonucleotide Therapeutics Society (OTS) 2018 by Julien Boudet - ProQR Therapeutics
Gepubliceerd op op Oligonucleotide Therapeutics Society (OTS)
Julien Boudet, Lenka van Sint Fiet, Antti Aalto, Roxana Redis, Tess Hoogeboom, Stephan Freriks, Petra de Bruijn, Lisanne van Wissen, Monika Hiller, Cherie Kemmel, Gerard Platenburg, Janne Turunen and Bart Klein
ProQR Therapeutics, 2333 CK Leiden, The Netherlands.
A robust computational approach is developed to screen for compatible interactions between backbone chemical modifications of EONs and the side-chains of the ADAR2 deaminase domain. Our data have been successfully correlated with docking studies performed with the Autodock Vina program. Experimental results show that the modelled patterns of specific chemical modifications are compatible with enzymatic activity. We demonstrate the applicability of a structure-based modelling approach as a tool for in silico screening of oligonucleotides for targeted RNA editing. We will extend our structure-based computational tools to other domains of ADARs and combine in silico screening with data from
high-throughput biochemical assays.
Presented at Oligonucleotide Therapeutics Society (OTS) 2018 by Julien Boudet - ProQR Therapeutics
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Author(s): Doherty EE, et al.
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