Method of Treating Neurodegenerative Disease
AssigneeAlnylam Pharmaceuticals, Inc.
InventorPamela Tan
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Abstract
Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.
Claims23
A double-stranded ribonucleic acid (dsRNA), wherein said dsRNA comprises at least two sequences that are substantially complementary to each other and wherein a sense strand of the dsRNA comprises a first sequence and an antisense strand of the dsRNA comprises a second sequence comprising a region that is substantially complementary to the corresponding region of an mRNA encoding SNCA, wherein said region is less than 30 nucleotides in length, and wherein said first sequence is selected from the group consisting of said sense strand sequences in Tables 2, 3, and 4, and wherein said second sequence is selected from the group consisting of said antisense strand sequences in Tables 2, 3, and 4.
Dependencies are inferred from the claim text.