US2018179533A1PendingUtilityA1
Modulation of transthyretin expression
Est. expiryApr 5, 2024(expired)· nominal 20-yr term from priority
C12N 2310/341C12N 2310/321Y10T436/143333C12N 2310/315C12N 2310/3341C12Q 1/6883C12N 2310/11C12Q 2600/158C12Q 2600/136C12N 15/113C12N 2310/346C12N 15/1136C12N 2310/3525
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Claims
Abstract
Compounds, compositions and methods are provided for modulating the expression of transthyretin. The compositions comprise oligonucleotides, targeted to nucleic acid encoding transthyretin. Methods of using these compounds for modulation of transthyretin expression and for diagnosis and treatment of diseases and conditions associated with expression of transthyretin are provided.
Claims
exact text as granted — not AI-modified1 .- 18 . (canceled)
19 . A compound comprising a liposome and an oligomeric compound at least 95% complementary within nucleobases 3880-3899 of SEQ ID NO: 11, or within nucleobases 6-25, 59-78, 91-119, 126-152, 170-189, 197-216, 217-236, 232-251, 250-269, 264-297, 323-361, 425-469, 460-532, or 532-619 of SEQ ID NO: 4, wherein the oligomeric compound comprises at least one modified sugar moiety.
20 . The compound of claim 19 , wherein the oligomeric compound is an antisense compound comprising a modified oligonucleotide 20 to 25 nucleobases in length.
21 . The compound of claim 20 , wherein at least one modified sugar moiety comprises a 2′-O—CH 3 modification.
22 . The compound of claim 20 , wherein the antisense compound is single-stranded.
23 . The compound of claim 21 , wherein the antisense compound is single-stranded.
24 . A method of inhibiting the expression of human transthyretin in a cell or tissue comprising contacting said cell or tissue with the compound of claim 19 , thereby inhibiting expression of human transthyretin in the cell or tissue.
25 . A method of inhibiting the expression of human transthyretin in a cell or tissue comprising contacting said cell or tissue with the compound of claim 20 , thereby inhibiting expression of human transthyretin in the cell or tissue.
26 . A method of inhibiting the expression of human transthyretin in a cell or tissue comprising contacting said cell or tissue with the compound of claim 21 , thereby inhibiting expression of human transthyretin in the cell or tissue.
27 . A method of inhibiting the expression of human transthyretin in a cell or tissue comprising contacting said cell or tissue with the compound of claim 22 , thereby inhibiting expression of human transthyretin in the cell or tissue.
28 . A method of inhibiting the expression of human transthyretin in a cell or tissue comprising contacting said cell or tissue with the compound of claim 23 , thereby inhibiting expression of human transthyretin in the cell or tissue.Join the waitlist — get patent alerts
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