SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
First Claim
1. A oligomeric compound comprising a modified oligonucleotide consisting of 10 to 30 linked nucleosides, wherein the modified oligonucleotide has a modification motif comprising:
- a 5′
-region consisting of 1-9 linked 5′
-region nucleosides, each independently selected from among a modified nucleoside and an unmodified deoxynucleoside, provided that at least one 5′
-region nucleoside is a modified nucleoside and wherein the 3′
-most 5′
-region nucleoside is a modified nucleoside;
a 3′
-region consisting of 2-10 linked 3′
-region nucleosides, wherein each 3′
-region nucleoside comprises a modified nucleoside; and
a central region between the 5′
-region and the 3′
-region consisting of 6-10 linked central region nucleosides, each independently selected from among;
a modified nucleoside and an unmodified deoxynucleoside, wherein the 5′
-most central region nucleoside is an unmodified deoxynucleoside and the 3′
-most central region nucleoside is an unmodified deoxynucleoside; and
wherein the modified oligonucleotide has a nucleobase sequence complementary to the nucleobase sequence of a target region of a nucleic acid associated with a huntingtin transcript.
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Accused Products
Abstract
The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, certain oligomeric compounds selectively reduce the expression of a target nucleic acid transcript relative to a non-target nucleic acid transcript.
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Citations
168 Claims
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1. A oligomeric compound comprising a modified oligonucleotide consisting of 10 to 30 linked nucleosides, wherein the modified oligonucleotide has a modification motif comprising:
- a 5′
-region consisting of 1-9 linked 5′
-region nucleosides, each independently selected from among a modified nucleoside and an unmodified deoxynucleoside, provided that at least one 5′
-region nucleoside is a modified nucleoside and wherein the 3′
-most 5′
-region nucleoside is a modified nucleoside;a 3′
-region consisting of 2-10 linked 3′
-region nucleosides, wherein each 3′
-region nucleoside comprises a modified nucleoside; anda central region between the 5′
-region and the 3′
-region consisting of 6-10 linked central region nucleosides, each independently selected from among;
a modified nucleoside and an unmodified deoxynucleoside, wherein the 5′
-most central region nucleoside is an unmodified deoxynucleoside and the 3′
-most central region nucleoside is an unmodified deoxynucleoside; and
wherein the modified oligonucleotide has a nucleobase sequence complementary to the nucleobase sequence of a target region of a nucleic acid associated with a huntingtin transcript. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 48, 49, 50, 51, 52, 53, 54, 55, 56, 57, 58, 59, 60, 61, 62, 63, 64, 65, 66, 67, 68, 69, 70, 71, 72, 73, 74, 75, 76, 77, 78, 79, 80, 81, 82, 83, 84, 85, 86, 87, 88, 89, 90, 91, 92, 93, 94, 95, 96, 97, 98, 99, 100, 101, 102, 103, 104, 105, 106, 107, 108, 109, 110, 111, 112, 113, 114, 115, 116, 117, 118, 119, 120, 121, 122, 123, 124, 125, 126, 127, 128, 129, 130, 131, 132, 133, 134, 135, 136, 137, 138, 139, 140, 141, 142, 143, 144, 145, 146, 147, 148, 149, 150, 151, 152, 153, 154, 155, 156, 157, 158, 159, 160, 161, 162, 163, 164, 165, 166, 167, 168)
- a 5′
Specification