Allele-specific rna silencing for the treatment of hypertrophic cardiomyopathy
AssigneesPresident and Fellows of Harvard College, The Brigham and Women's Hospital, Inc.
InventorsJonathan Seidman, Christine Seidman, Jiangmingjian Jiang, Hiroko Wakimoto
Life of this patent
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Abstract
Provided herein are methods and compositions useful for the treatment of hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM) and/or left ventricular non-compaction (LVNC) and other cardiomyopathies through allele-specific RNA silencing.
Claims25
A method of preventing or treating hypertrophic cardiomyopathy (HCM) in a subject having in their genome a first MYH7 allele comprising an HCM-causing mutation and a second MYH7 allele that does not comprise the HCM-causing mutation, the method comprising administering to the subject an interfering RNA molecule that selectively inactivates the transcript encoded by the first MYH7 allele compared to the transcript encoded by the second MYH7 allele.
21-80. (canceled)
A method of preventing or treating hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM) or Left Ventricular Non-Compaction (LVNC) in a subject having in their genome a first MYL3, MYH7, TNNI3, TNNT2, TPM1 or ACTC1 allele comprising an HCM-causing, a DCM-causing or a LVNC-causing mutation and a second MYL3, MYH7, TNNI3, TNNT2, TPM1 or ACTC1 allele that does not comprise the HCM-causing, the DCM-causing or the LVNC-causing mutation, the method comprising administering to the subject an interfering RNA molecule that selectively inactivates the transcript encoded by the first MYL3, MYH7, TNNI3, TNNT2, TPM1 or ACTC1 allele compared to the transcript encoded by the second MYL3, MYH7, TNNI3, TNNT2, TPM1 or ACTC1 allele.
82-260. (canceled)
An interfering RNA molecule or antisense oligonucleotide that selectively inhibits expression of a MYH7, MYL3, TNNI3, TNNT2, TPM1 or ACTC1 transcript comprising a MYH7, MYL3, TNNI3, TNNT2, TPM1 or ACTC1 polymorphism or mutation compared to a MYH7, MYL3, TNNI3, TNNT2, TPM1 or ACTC1 transcript not comprising the MYH7, MYL3, TNNI3, TNNT2, TPM1 or ACTC1 polymorphism or mutation.
262-341. (canceled)
Dependencies are inferred from the claim text.