Trophin genome editing for treating Duchenne Muscular Dystrophy (DMD)
AssigneeThe Trustees of the University of Pennsylvania
InventorTejvir S. Khurana
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Abstract
The invention relates to compositions and methods for improving a dystrophic phenotype in a human subject having myopathies, such as Duchenne Muscular Dystrophy (DMD). In one embodiment, the invention relates to compositions comprising an adenoviral vector targeting the let-7c miRNA binding sequence in 3′-UTR genome editing of the utrophin gene and methods of treatment comprising administration thereof.
Claims28
A method for improving a dystrophic phenotype in a human subject having Duchenne Muscular Dystrophy (DMD), the method comprising:
- CRISPR-cas9 based genome editing a 3′ untranslated region (UTR) of a utrophin gene to delete a let-7c microRNA (miRNA) binding sequence in it,
- wherein deletion of the let-7c miRNA binding sequence alleviates miRNA-mediated repression and upregulates utrophin expression, thereby improving the dystrophic phenotype in the human subject.
A method for treating Duchenne Muscular Dystrophy (DMD) in a human subject, the method comprising:
- CRISPR-cas9 based genome editing a 3′ untranslated region (UTR) of a utrophin gene to delete a let-7c microRNA (miRNA) binding sequence in it,
- wherein deletion of the let-7c miRNA binding sequence alleviates miRNA-mediated repression and upregulates utrophin expression, thereby treating DMD in the human subject.
Dependencies are inferred from the claim text.