ATAXIN3 (ATXN3) RNAi agent compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsAdam Castoreno, Elane Fishilevich, Jason A. Gilbert, Charalambos Kaittanis, James D. McIninch, Stuart Milstein, Mark K. Schlegel
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Abstract
The disclosure relates to double-stranded ribonucleic acid (dsRNA) agents and compositions targeting the ATXN3 gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an ATXN3-associated disease or disorder, such as SCA3, using such dsRNA agents and compositions.
Claims20
A double-stranded ribonucleic acid (dsRNA) agent for inhibiting expression of ATXN3, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein the antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from a nucleotide sequence of an antisense sequence set forth as SEQ ID NO:
- 1898, 1862, or 1912, and the sense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from a nucleotide sequence of a sense sequence set forth as SEQ ID NO:
- 1839, 1803, or 1853.
Dependencies are inferred from the claim text.