COMPOUNDS AND METHODS FOR MODULATING EXPRESSION APOB
First Claim
1. A short antisense compound 8 to 16 monomers in length, comprising a 2′
- -deoxyribonucleotide gap region flanked on each side by a wing, wherein each wing independently comprises 1 to 3 high-affinity modified monomers and wherein the short antisense compound is targeted to a nucleotide encoding ApoB.
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Abstract
The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
19 Citations
51 Claims
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1. A short antisense compound 8 to 16 monomers in length, comprising a 2′
- -deoxyribonucleotide gap region flanked on each side by a wing, wherein each wing independently comprises 1 to 3 high-affinity modified monomers and wherein the short antisense compound is targeted to a nucleotide encoding ApoB.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 38, 40, 47, 48, 49, 50, 51)
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19-37. -37. (canceled)
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39. (canceled)
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41. (canceled)
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42. (canceled)
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43. A method of modulating expression of a ApoB by contacting a nucleic acid encoding ApoB with a short antisense compound.
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44. (canceled)
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45. (canceled)
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46. (canceled)
Specification