US 11,723,912 B2
Staufen1 agents and associated methods
Stefan M. Pulst, Sandy, UT (US); Daniel R. Scoles, Salt Lake City, UT (US); and Sharan Paul, Salt Lake City, UT (US)
Assigned to University of Utah Research Foundation, Salt Lake City, UT (US)
Appl. No. 16/467,945
Filed by University of Utah Research Foundation, Salt Lake City, UT (US)
PCT Filed Dec. 8, 2017, PCT No. PCT/US2017/065421
§ 371(c)(1), (2) Date Jun. 7, 2019,
PCT Pub. No. WO2018/107096, PCT Pub. Date Jun. 14, 2018.
Claims priority of provisional application 62/431,757, filed on Dec. 8, 2016.
Prior Publication US 2020/0069721 A1, Mar. 5, 2020
Int. Cl. C07H 21/02 (2006.01); C07H 21/04 (2006.01); A61K 31/713 (2006.01); A61K 45/06 (2006.01); C12N 15/113 (2010.01); A61P 25/28 (2006.01)
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.