Therapeutic oligonucleotides comprising pyrazolotriazine nucleotide analogues

Disclosed herein are double-stranded RNA nucleic acid molecules, which include at least one pyrazolotriazine nucleotide analog and have been modified to exhibit one of the following, increased on-target activity, increased target specificity, enhanced nuclease stability, reduced off target activity...

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Bibliographische Detailangaben
Hauptverfasser: Truffert, Jean-Christophe, Hildesheim, Jean, Lefoix, Myriam, Kleinman, Tirtsa, Avkin-Nachum, Sharon
Format: Patent
Sprache:eng
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Zusammenfassung:Disclosed herein are double-stranded RNA nucleic acid molecules, which include at least one pyrazolotriazine nucleotide analog and have been modified to exhibit one of the following, increased on-target activity, increased target specificity, enhanced nuclease stability, reduced off target activity and/or reduced immunogenicity when compared to an unmodified or similarly modified dsRNA; pharmaceutical compositions comprising such molecules and methods of use thereof in therapy.