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US 9,447,415 B2Granted
Antisense oligonucleotides for inducing exon skipping and methods of use thereof
AssigneeThe University of Western Australia
InventorsStephen Donald Wilton, Sue Fletcher, Graham McClorey
Life of this patent
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Abstract
An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.
Claims2
An antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of exon 45 of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of CCA AUG CCA UCC UGG AGU UCC UGU AA (SEQ ID NO:
- 207), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 45 skipping;
- or a pharmaceutically acceptable salt thereof.
A pharmaceutical composition comprising:
- (i) an antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of exon 45 of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of CCA AUG CCA UCC UGG AGU UCC UGU AA (SEQ ID NO:
- 207), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 45 skipping;
- or a pharmaceutically acceptable salt thereof, and (ii) a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.