COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
First Claim
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1. An antisense compound for treating myotonic dystrophy DM1, comprising a 9-base morpholino antisense oligonucleotide, where the 9 bases are complementary to polyCUG repeats in the 3′
- UTR region of dystrophia myotonica protein kinase (DMPK) mRNA.
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Abstract
Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
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14 Claims
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1. An antisense compound for treating myotonic dystrophy DM1, comprising a 9-base morpholino antisense oligonucleotide, where the 9 bases are complementary to polyCUG repeats in the 3′
- UTR region of dystrophia myotonica protein kinase (DMPK) mRNA.
- View Dependent Claims (2, 3, 4, 5)
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6. A method of treating myotonic dystrophy DM1 in a mammalian subject, comprising administering to the subject a 9-base morpholino antisense oligonucleotide, where the 9 bases are complementary to polyCUG repeats in the 3′
- UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and repeating said administering at least once every one week to 3 months.
- View Dependent Claims (7, 8, 9, 10, 11, 12, 13, 14)
Specification