Lipid formulated compositions and methods for inhibiting expression of transthyretin (TTR)
The invention relates to lipid formulated double-stranded ribonucleic acid (dsRNA) targeting a transthyretin (TTR) gene, and methods of using the dsRNA to inhibit expression of TTR.
1. A method for reducing a TTR amyloid deposit by >90% in a subject having the TTR amyloid deposit, comprising administering to the subject an effective amount of a composition comprising a TTR dsRNA and determining TTR amyloid deposition in the subject, wherein: the TTR dsRNA comprises a sense strand and an antisense strand, the antisense strand consisting of SEQ ID NO:1010 and the sense strand consisting of SEQ ID NO:1009; the effective amount is 0.01-30 mg/kg TTR dsRNA; and the composition comprises a lipid formulation comprising a cationic lipid.
2. The method of claim 1 , wherein the TTR amyloid deposit comprises V30M mutant TTR.
3. The method of claim 1 , wherein the subject is a human subject.
4. The method of claim 1 , wherein the TTR amyloid deposit comprises V30M mutant TTR and the subject is a human subject.