Methods for enhancing utrophin production via inhibition of microRNA
AssigneeThe Trustees of the University of Pennsylvania
InventorsTejvir S. Khurana, Steve Wilton
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Abstract
This invention provides a method for enhancing utrophin protein production in a cell by inhibiting an utrophin microRNA molecule. Moreover, the invention provides that methods for enhancing utrophin protein production in a muscle cell are used for treating a muscular dystrophy and/or other myopathies.
Claims6
A pharmaceutical composition comprising an antisense oligonucleotide that specifically hybridizes to a Let-7c microRNA binding sequence in a 3′-UTR of a utrophin mRNA 3′ untranslated region (UTR) and inhibits the binding of the Let-7c microRNA to the utrophin mRNA 3′-UTR and at least one pharmaceutically acceptable excipient, wherein the antisense oligonucleotide is present in an amount effective in a human subject to inhibit the binding of Let-7 micro RNA with its utrophin mRNA 3′-UTR binding sequence, and wherein the antisense oligonucleotide comprises the sequence of SEQ ID NO:
- 25 (5′-CUG AGG UAG AAA GGU GGU CAU GGC UU-3′).
Dependencies are inferred from the claim text.