Compositions and methods for inhibiting expression of the lect2 gene
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsGregory Hinkle, Huilei Xu
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Abstract
The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.
Claims68
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of LECT2, wherein said dsRNA comprises a sense strand that is 15-30 base pairs in length and an antisense strand that is 15-30 base pairs in length and the antisense strand is complementary to at least 15 contiguous nucleotides of SEQ ID NO:
- 1.
A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of LECT2, wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region of complementarity to a LECT2 RNA transcript, which antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from one of the antisense sequences listed in Tables 2 and 3.
The dsRNA of claim of any of the preceding claims, wherein the duplex region is 15-30 nucleotide pairs in length.
The dsRNA of claim of any of the preceding claims, wherein the duplex region is 17-23 nucleotide pairs in length.
The dsRNA of claim of any of the preceding claims, wherein the duplex region is 19-21 nucleotide pairs in length.
The dsRNA of claim of any of the preceding claims, wherein the duplex region is 21-23 nucleotide pairs in length.
The dsRNA of any of the preceding claims, wherein the region of complementarity is at least 17 nucleotides in length.
The dsRNA of any of the preceding claims, wherein the region of complementarity is 19 nucleotides in length.
The dsRNA of any of the preceding claims, wherein the region of complementarity is between 19 and 21 nucleotides in length.
The dsRNA of any one of the preceding claims, wherein at least one strand comprises a 3′ overhang of at least 1 nucleotide.
The dsRNA of any of the preceding claims wherein the sense strand is conjugated to at least one ligand.
The dsRNA of any one of the preceding claims, wherein the region of complementarity consists of an antisense sequence selected from the antisense sequences disclosed in Tables 2 and 3.
The dsRNA of any one of the preceding claims, wherein the dsRNA comprises a sense strand consisting of a sense sequence selected from the sense sequences disclosed in Tables 2 and 3, and an antisense strand consisting of an antisense sequence selected from the antisense sequences disclosed in Tables 2 and 3.
A cell containing the dsRNA of any one of the preceding claims.
A composition for use in a method of treating a LECT2 amyloidosis comprising administering to a subject in need of such treatment a double-stranded ribonucleic acid (dsRNA), wherein said dsRNA comprises a sense strand that is 15-30 base pairs in length and an antisense strand that is 15-30 base pairs in length and the antisense strand is complementary to at least 15 contiguous nucleotides of SEQ ID NO:
- 1 or a nucleotide sequence having a A to G substitution at nucleotide position 373 of SEQ ID NO:
- 1.
A vector encoding at least one strand of a dsRNA, wherein said dsRNA comprises a region of complementarity to at least a part of an mRNA encoding LECT2, wherein said dsRNA is 30 base pairs or less in length, and wherein said dsRNA targets said mRNA for cleavage.
Dependencies are inferred from the claim text.