Compositions and methods for inhibiting snca expression
AssigneeDicerna Pharmaceuticals, Inc.
InventorsBob Dale BROWN, Henryk T. DUDEK, Seongmoon CHEONG, Shiyu WANG, Travis GRIM, Matthew Guese COSTALES, Maire JUNG
Life of this patent
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Abstract
Oligonucleotides (e.g., RNAi oligonucleotides) are provided herein that inhibit SNCA gene expression, including oligonucleotides conjugated to a targeting ligand (e.g., GalNAC moiety or lipid moiety). Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders, and/or conditions associated with SNCA gene expression.
Claims48
A RNAi oligonucleotide for reducing SNCA gene expression, the oligonucleotide comprising a sense strand and an antisense strand, wherein the sense strand is 15 to 50 nucleotides in length, wherein the sense strand and the antisense strand form a duplex region, wherein the antisense strand comprises a region of complementarity to a SNCA mRNA target sequence of any one of SEQ ID NOs:
- 1683-2066, and wherein the region of complementarity is at least 15 contiguous nucleotides in length.
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