Compositions And Methods For Inhibiting Expression Of The HAMP Gene
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsTomoko Nakayama, Anke Geick, Pamela Tan, Herbert Y. Lin
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Abstract
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the HAMP gene (HAMP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the HAMP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HAMP gene expression and the expression of the HAMP gene using the pharmaceutical composition.
Claims20
A method of inhibiting the expression of hepcidin antimicrobial peptide (HAMP) in a cell or tissue, comprising contacting the cell or tissue with a compound comprising an antisense strand and a sense strand that are each equal to or less than 30 nucleotides in length, wherein the compound is targeted to a nucleic acid molecule encoding HAMP and comprising the first nineteen nucleotides of SEQ ID NO:29 (GAACAUAGGUCUUGGAAUA), and wherein the compound specifically hybridizes with a 5′ untranslated region or coding region of the nucleic acid molecule, so that expression of HAMP is inhibited by at least 20%.
A compound for inhibiting the expression of hepcidin antimicrobial peptide (HAMP) in a cell or tissue, comprising an antisense strand and a sense strand that are each equal to or less than 30 nucleotides in length, wherein the compound is targeted to a nucleic acid molecule encoding HAMP and comprising the first nineteen nucleotides of SEQ ID NO:29 (GAACAUAGGUCUUGGAAUA), and wherein the compound specifically hybridizes with a 5′ untranslated region or coding region of the nucleic acid molecule, so that expression of HAMP is inhibited by at least 20%.
Dependencies are inferred from the claim text.