(12) Patent Application Publication (10) Pub. No.: US 2016/0348103 A1 WHEELER Et Al
US 201603481 03A1 (19) United States (12) Patent Application Publication (10) Pub. No.: US 2016/0348103 A1 WHEELER et al. (43) Pub. Date: Dec. 1, 2016 (54) OLIGONUCLEOTIDES AND METHODS FOR Related U.S. Application Data TREATMENT OF CARDIOMYOPATHY USING RNA INTERFERENCE (60) 27,Provisional 2014. application No. 61/931,690, filed on Jan. (71) Applicant: THE BOARD OF TRUSTEE OF Publication Classification THE LELAND STANFORD JUNOR UNIVERSITY, Palo Alto, CA (US) (51) Int. Cl. (72) Inventors: Matthew WHEELER, Sunnyvale, CA CI2N IS/II3 (2006.01) US); Euan A. ASHLEY. Menlo Park (52) U.S. Cl. CA(US), (US); Eua Katheia AA M. , Menlo Park, CPC ......... CI2N 15/113 (2013.01); C12N 2310/14 ZALETA-RIVERA, Stanford, CA (US) (2013.01) (73) Assignee: The Board of Trustees of the Leland (57) ABSTRACT Stanford Junior University, Stanford, Compositions and methods for treating cardiomyopathy CA (US) using RNA interference are disclosed. In particular, embodi ments of the invention relate to the use of oligonucleotides (21) Appl. No.: 15/114,063 for treatment of cardiomyopathy, including Small interfering RNAs (siRNAs) and short hairpin RNAs (shRNAs) that (22) PCT Filed: Jan. 26, 2015 silence expression of disease-causing mutant alleles, such as the myosin MYL2 allele encoding human regulatory light (86). PCT No.: PCT/US2O15/O12966 chain (hRLC)-N47K and the MYH7 allele encoding human S 371 (c)(1), myosin heavy chain (hMHC)-R403O while retaining (2) Date: Jul. 25, 2016 expression of the corresponding wild-type allele. Patent Application Publication Dec. 1, 2016 Sheet 1 of 38 US 2016/0348103 A1 Patent Application Publication Dec.
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