abstract |
Disclosed are a double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and a method of using the dsRNA to inhibit the expression of TTR. A double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of transthyretin (TTR), wherein the dsRNA includes a sense strand and an antisense strand, and the antisense strand comprises TTR. Two regions comprising a region that is complementary to a portion of the encoding mRNA, the region of complementarity being less than 30 nucleotides in length, wherein the antisense strand and the sense strand comprise nucleotides having a particular sequence Strand ribonucleic acid (dsRNA). A pharmaceutical composition comprising dsRNA, wherein the disorder is transthyretin amyloidosis. [Selection figure] None |