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US 2022/0033815 A1Published application
Systemic in vivo delivery of oligonucleotides
AssigneeOncoimmunin, Inc.
InventorsBeverly PACKARD, Akira KOMORIYA
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AssigneeOncoimmunin, Inc.
InventorsBeverly PACKARD, Akira KOMORIYA
Estimating the term…
This invention provides a method for the systemic in vivo delivery of oligonucleotides. The invention utilizes the presence of one or plurality of HES linked to an oligonucleotide to deliver a nucleic acid sequence of interest into the cytoplasm of cells and tissues of live organisms. The delivery vehicle is nontoxic to cells and organisms. Since delivery is sequence-independent and crosses membranes in a receptor-independent manner, the delivered oligonucleotide can target complementary sequences in the cytoplasm as well as in the nucleus of live cells. Sequences of bacterial or viral origin can also be targeted. The method can be used for delivery of genes coding for expression of specific proteins, antisense oligonucleotides, siRNAs, shRNAs, Dicer substrates, miRNAs, anti-miRNAs or any nucleic acid sequence in a living organism. The latter include mammals, plants, and microorganisms such as bacteria, protozoa, and viruses.
A composition comprising an H-type excitonic structure (HES)-oligonucleotide containing an oligonucleotide gapmer, wherein the oligonucleotide gapmer specifically hybridizes to a ribonucleic acid sequence of interest and is a substrate for RNAse H when hybridized to the ribonucleic acid.
-12. (canceled)
-24. (canceled)
A method for modulating a ribonucleic acid in a subject, said method comprising administering to the subject an effective amount of an H-type excitonic structure (HES)-oligonucleotide containing a oligonucleotide gapmer, wherein the oligonucleotide gapmer specifically hybridizes to a ribonucleic acid of interest and is a substrate for RNAse H when hybridized to the ribonucleic acid.
A method for treating a disease or disorder in a subject, said method comprising administering to a subject in need thereof, a therapeutically effective amount of a composition comprising an H-type excitonic structure (HES)-oligonucleotide containing an oligonucleotide gapmer that specifically hybridizes to a ribonucleic acid of interest and is a substrate for RNAse H when hybridized to the ribonucleic acid.
-32. (canceled)
A method for decreasing the amount of a ribonucleic acid of interest in a cell, said method comprising contacting a cell expressing the ribonucleic acid of interest with an effective amount of a composition comprising an HES-oligonucleotide containing an oligonucleotide gapmer, wherein the oligonucleotide gapmer specifically hybridizes to the ribonucleic acid of interest and is a substrate for RNAse H when hybridized to the ribonucleic acid.
Dependencies are inferred from the claim text.