Compositions and methods for regulating expression of atxn2
AssigneeRiboway Therapeutics, Inc.
InventorsQinghong Yan, Sebastien Weyn
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Abstract
The present disclosure provides an antisense oligonucleotide that hybridizes to a target region in an ATXN2 RNA transcript, wherein hybridization of the antisense oligonucleotide to the target region changes (e.g., decreases) the expression of a functional protein encoded by the ATXN2 RNA transcript in a cell. The present disclosure further provides antisense oligonucleotides, pharmaceutical compositions, and methods for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with ATXN2 in a subject in need thereof.
Claims35
An antisense oligonucleotide that hybridizes to a target region in an ATXN2 RNA transcript, wherein hybridization of the antisense oligonucleotide to the target region decreases the expression of a functional protein encoded by the ATXN2 RNA transcript in a cell.
A method of reducing the amount or activity of a target mRNA encoding an ATXN2 isoform in a cell, comprising
- contacting the cell with an antisense oligonucleotide configured to hybridize with the target mRNA;
- and
- causing the target mRNA transcript to be degraded by nonsense-mediated decay.
Dependencies are inferred from the claim text.