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US 11,932,851 B2Granted
Exon skipping compositions for treating muscular dystrophy
AssigneeSarepta Therapeutics, Inc.
InventorsRichard K. Bestwick, Diane Elizabeth Frank
Life of this patent
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Abstract
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
Claims6
An antisense oligonucleotide of 28 bases comprising the base sequence of SEQ ID NO:
- 6, in which thymine bases are optionally uracil bases;
- wherein the sugar moieties of the oligonucleotide backbone are replaced with morpholinos;
- and
- wherein the antisense oligonucleotide is conjugated to an arginine-rich cell penetrating peptide;
- or a pharmaceutically acceptable salt thereof.
2 more elements
Dependencies are inferred from the claim text.