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US 8,865,883 B2Granted
Multiple exon skipping compositions for DMD
AssigneeSarepta Therapeutics, Inc.
InventorsPeter Sazani, Ryszard Kole
Life of this patent
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Abstract
Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
Claims21
An isolated antisense oligonucleotide of 20 to 35 nucleotides in length comprising at least 17 contiguous nucleotides of a nucleotide sequence set forth as SEQ ID NO:
- 2 wherein the oligonucleotide is capable of binding to human dystrophin pre-mRNA to induce exon 44 skipping, and wherein the oligonucleotide comprises a modification to resist degradation of an oligonucleotide:RNA heteroduplex by RNase H, and thymine bases (T) are optionally uracil bases (U).
Dependencies are inferred from the claim text.