2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
Abstract
Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2′-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.
Claims
exact text as granted — not AI-modified1 . A method for assessing the activity of an oligomeric compound comprising contacting the oligomeric compound with a cell in vitro, wherein the oligomeric compound comprises a first oligomer and a second oligomer, wherein:
at least a portion of said first oligomer is capable of hybridizing with at least a portion of said second oligomer, at least a portion of said first oligomer is complementary to and capable of hybridizing with a selected target nucleic acid, each of said first and second oligomers is a chimeric oligomer,
wherein the second oligomer has an internal segment comprising at least four consecutive nucleosides each having a 2′-OCH 3 substituent group, and
wherein the chimeric oligomers are not a gapmers;
measuring or detecting a phenotypic change in the cell in vitro; and
thereby assessing the activity of the oligomeric compound.
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