Serpina1 sirnas: compositions of matter and methods of treatment
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsAlfica Sehgal, David Bumcrot, Brian Bettencourt
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AssigneeAlnylam Pharmaceuticals, Inc.
InventorsAlfica Sehgal, David Bumcrot, Brian Bettencourt
Estimating the term…
The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpinal gene, and methods of using such dsRNA compositions to inhibit expression of Serpinal. In one embodiment, an iRNA for inhibiting expression of a Serpinal gene includes at least two sequences that are complementary to each other. The iRNA includes a sense strand having a first sequence and an antisense strand having a second sequence. The antisense strand includes a nucleotide sequence that is substantially complementary to at least part of an mRNA encoding Serpinal, and the region of complementarity is 30 nucleotides or less, and at least 15 nucleotides in length. Generally, the iRNA is 19 to 24, e.g., 19 to 21 nucleotides in length.
1-38. (canceled)
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of Serpinal, wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region of complementarity which comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from one of the antisense sequences listed in Tables 3 and 4.
A method of inhibiting Serpinal expression in a cell, the method comprising:
A method of treating a disorder mediated by Serpinal expression comprising administering to a patient in need of such treatment a therapeutically effective amount of a double-stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand, the antisense strand comprising a region of complementarity which comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from one of the antisense sequences listed in Tables 3 and 4.
Dependencies are inferred from the claim text.