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 method of treating a disorder related to ALAS1 expression, comprising administering to a subject in need of such treatment a therapeutically effective amount of a double-stranded ribonucleic acid (dsRNA), wherein said dsRNA comprises a sense strand and an antisense strand, each of which are 15-30 nucleotides in length, wherein the antisense strand comprises a region of complementarity to an ALAS1 RNA and comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from SEQ ID NO: 1296, and wherein the antisense strand is complementary to at least nucleotides 871-889 of SEQ ID NO: 1.