Compositions And Methods For Inhibiting Expression Of The HAMP Gene

摘要

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.

主权项

1. 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%.