Compositions and methods for inhibiting expression of the ALAS1 gene

摘要

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

主权项

1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, wherein said dsRNA comprises a sense strand and an antisense strand, wherein:
(i) the antisense strand is complementary to at least nucleotides 871-889 of SEQ ID NO:1 and comprises SEQ ID NO:1296, (ii) the sense strand comprises at least 15 contiguous nucleotides from SEQ ID NO:1295, (iii) a ligand and linker with a structure as shown below is attached to the 3′ end of the sense strand of the dsRNAand
(iv) the dsRNA comprises one or more nucleotides with a modification chosen from a 2′-O-methyl modified nucleotide, a 2′-fluoro modified nucleotide, or both.