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US 8,835,402 B2Granted
Compound and method for treating myotonic dystrophy
AssigneeSarepta Therapeutics, Inc.
InventorsRyszard Kole, Gunnar J. Hanson
Life of this patent
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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.
Claims10
An antisense compound for treating myotonic dystrophy DM1, consisting of a 9-base phosphorodiamidate morpholino antisense oligonucleotide,
- wherein at least one and up to about 1 per every 2 intersubunit linkage(s) contains a pendant cationic group, and
- wherein the 9 bases are complementary to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA and have the base sequence of SEQ ID NO:5 or SEQ ID NO:9.
A method of treating myotonic dystrophy DM1 in a mammalian subject, comprising administering to the subject a 9-base phosphorodiamidate morpholino antisense oligonucleotide,
- wherein at least one and up to about 1 per every 2 intersubunit linkage(s) contains a pendant cationic group, and
- wherein the 9 bases are complementary to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA and have the base sequence of SEQ ID NO:5 or SEQ ID NO:9,
- and repeating said administering at least once every one week to 3 months.
Dependencies are inferred from the claim text.