Modulation of transthyretin expression for the treatment of cns related disorders
AssigneeIsis Pharmaceuticals, Inc.
InventorsRichard Alan Smith, Brett P. Monia
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Abstract
Compounds, compositions and methods are provided for modulating the expression of transthyretin in the brain, specifically the choroid plexus. The compositions comprise oligonucleotides, targeted to nucleic acid encoding transthyretin. Methods of using these compounds for modulation of transthyretin expression and for diagnosis and treatment of diseases and conditions associated with expression of transthyretin are provided.
Claims33
A method of inhibiting transthyretin in the brain of an animal comprising;
- administering a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the modified oligonucleotide is complementary to human transthyretin;
- and thereby inhibiting transthyretin in the brain of an animal.
A method comprising identifying an animal having a central nervous system disorder;
- and
- administering to the brain of the animal having a central nervous system disorder a therapeutically effective amount of a transthyretin inhibitor.
A method of reducing amyloid fibril formation in an animal comprising;
- administering to the brain of the animal a therapeutically effective amount of a compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the modified oligonucleotide is complementary to human transthyretin;
- and
- thereby reducing amyloid fibril formation in an animal.
A method of preventing, ameliorating, or treating of a central nervous system disorder in an animal comprising;
- identifying an animal having a central nervous system disorder;
- administering to the brain of the animal a therapeutically effective amount of a compound comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the modified oligonucleotide is complementary to human transthyretin;
- and
- thereby preventing, ameliorating, or treating of a central nervous system disorder in an animal.
A method of inhibiting transthyretin in the choroid plexus of an animal comprising;
- administering a modified oligonucleotide consisting of 12 to 30 linked nucleosides, wherein the modified oligonucleotide is complementary to human transthyretin;
- and thereby inhibiting transthyretin in the brain of an animal.
Dependencies are inferred from the claim text.