Oligonucleotides and Methods for Treatment of Cardiomyopathy Using RNA Interference

Abstract: Compositions and methods for treating cardiomyopathy using RNA interference are disclosed. In particular, embodiments of the invention relate to the use of oligonucleotides for treatment of cardiomyopathy, including small interfering RNAs (siRNAs) and short hairpin RNAs (shRNAs) that silence expression of disease-causing mutant alleles, such as the myosin MYL2 allele encoding human regulatory light chain (hRLC)-N47K and the MYH7 allele encoding human myosin heavy chain (hMHC)-R403Q while retaining expression of the corresponding wild-type allele.
Patent Number: US2016348103(A1)
Public Date:
Inventor: WHEELER MATTHEW [US]; ASHLEY EUAN A [US]; ZALETA-RIVERA KATHEIA M [US]
Applicant: THE BOARD OF TRUSTEE OF THE LELAND STANFORD JUNIOR UNIV [US]; UNIV LELAND STANFORD JUNIOR [US]
Application Number: US201515114063 20150126Global Dossier
Application Date:
Priority Number: US201515114063 20150126;US201461931690P 20140127;WO2015US12966 20150126
International Classification: C12N15/113
Cooperative Classification: A01K67/0275; C12N15/113; A01K2217/052; A01K2227/105; A01K2267/0306; C12N2310/14; C12N2320/31; C12N27
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