CPC A61K 31/713 (2013.01) [A61K 45/06 (2013.01); A61P 25/28 (2018.01); C12N 15/113 (2013.01); C12N 2310/14 (2013.01)] | 7 Claims |
1. A method of minimizing dysregulation of Staufen1-mediated RNA metabolism in a target cell, comprising:
introducing an amount of a Staufen1-regulating agent to the target cell that is sufficient to minimize said dysregulation, wherein the Staufen1-regulating agent is a member selected from the group consisting of an antisense oligonucleotide (ASO), a small interfering RNA (siRNA), a small molecule, a micro RNA (miRNA), a ribozyme, or a combination thereof, and
wherein minimizing dysregulation of Staufen1-mediated RNA metabolism includes reducing Staufen1 expression, reducing Staufen1 activity, reducing mutant ATXN2 expression, reducing mTOR expression, reducing or blocking Staufen1 interaction with mRNAs resulting in altered mRNA expression or abundance, or a combination thereof,
wherein the Staufen1-regulating agent comprises a nucleotide sequence that is at least 85% homologous to and having the same number of nucleotides as SEQ ID NO: 1, SEQ ID NO: 2, or a combination thereof.
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