Agents for modulating expression
AssigneeKicho Inc.
InventorsOliver S.C. Quigley, Ethan Perlstein
Life of this patent
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Abstract
Agents that target a nucleic acid (e.g., processed mRNA) can modulate expression of a protein that is encoded by the nucleic acid, e.g., via modulation of the level of the nucleic acid. In aspects, provided herein are compositions, methods, kits, and systems related to agents that modulate protein expression by targeting a nucleic acid molecule that encodes the protein.
Claims20
1-126. (canceled)
A pharmaceutical composition for use in a method of reducing expression of a UBE3A protein in a mammalian cell having duplication, overexpression, or a gain-of-function mutation of a UBE3A gene that encodes the UBE3A protein, the method comprising contacting the pharmaceutical composition to the mammalian cell, wherein the pharmaceutical composition comprises an antisense oligomer or a vector encoding the antisense oligomer, and wherein the antisense oligomer reduces a level of a processed mRNA encoding the UBE3A protein in the mammalian cell.
An antisense oligomer that comprises a sequence with at least 80% identity to the sequence set forth in any one of SEQ ID NOs:
- 1-92.
A method of treating a disease or condition in a subject in need thereof by reducing expression of a UBE3A protein in cells of the subject, comprising contacting to the cells of the subject the pharmaceutical composition comprising an antisense oligomer that comprises a sequence with at least 80% identity to the sequence set forth in any one of SEQ ID NOs:
- 1-92.
Dependencies are inferred from the claim text.