Method of using modified oligonucleotides for hepatic delivery
OwnerPhillip Dan Cook, Muthiah Manoharan +1inventors
InventorsPhillip Dan Cook, Muthiah Manoharan, Clarence Frank Bennett
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Abstract
A method of modulating the expression of a nucleic acid in the hepatic system of a mammal is disclosed. In addition, a method of preferentially targeting an oligonucleotide to liver cells in a mammal is disclosed. Also, a method of treating an animal having a hepatic disease or disorder associated with a protein encoded by a gene is disclosed. A composition containing an oligonucleotide is disclosed.
Claims22
A method of modulating the expression of a nucleic acid in the hepatic system of a mammal, comprising the step of:
- administering to said mammal an oligonucleotide which hybridizes to said nucleic acid to modulate the expression of said nucleic acid,
- wherein said oligonucleotide has at least two sterol moieties covalently bonded thereto.
A method of preferentially targeting an antisense oligonucleotide to liver cells in a mammal, comprising the steps of:
- covalently bonding said oligonucleotide to at least two sterol moieties to form a sterol-oligonucleotide conjugate;
- and
- administering said sterol-oligonucleotide conjugate to said mammal to preferentially target said oligonucleotide to said liver cells in said mammal to modulate the expression of a gene in said liver cells.
A method of treating an animal having a hepatic disease or disorder associated with a protein encoded by a gene, comprising the step of:
- administering to said mammal an oligonucleotide which hybridizes to said gene,
- wherein said oligonucleotide has at least two sterol moieties covalently bonded thereto.
A composition, comprising an oligonucleotide,
- wherein said oligonucleotide has at least two sterol moieties covalently bonded thereto.
A composition, wherein said oligonucleotide is an antisense nucleotide.
Dependencies are inferred from the claim text.