Compositions and methods for silencing apolipoprotein C-III expression

摘要

The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA that target apolipoprotein C-III (APOC3) gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for the treatment of lipid diseases or disorders such as atherosclerosis or a dyslipidemia such as hypertriglyceridemia or hypercholesterolemia).

主权项

1. A composition comprising a small-interfering RNA (siRNA) that silences apolipoprotein C-III (APOC3) gene expression, wherein the siRNA comprises a sense strand and a complementary antisense strand, and wherein the siRNA comprises a double-stranded region of about 19 to about 25 nucleotides in length, and wherein the antisense strand comprises a sequence set forth in SEQ ID NO:770 and/or wherein the sense strand comprises a sequence set forth in SEQ ID NO:769.