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.
Claims24
An antisense oligonucleotide of 24 bases comprising the base sequence GAU AGG UGG UAU CAA CAU CUG UAA (SEQ ID NO:
- 1), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide.
An antisense oligonucleotide of 21 bases comprising the base sequence GAU AGG UGG UAU CAA CAU CUG (SEQ ID NO:
- 2), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide.
An antisense oligonucleotide of 25 bases comprising the base sequence GAU AGG UGG UAU CAA CAU CUG UAA G (SEQ ID NO:
- 3), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide.
An antisense oligonucleotide of 20 bases comprising the base sequence GGU GGU AUC AAC AUC UGU AA (SEQ ID NO:
- 4), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide.
A pharmaceutical composition comprising an antisense oligonucleotide of 20 to 50 bases comprising at least 17 consecutive bases of the base sequence GAU AGG UGG UAU CAA CAU CUG UAA (SEQ ID NO:
- 1), in which the uracil bases are optionally thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to a target region in exon 8 of the human dystrophin gene inducing exon 8 skipping, and a pharmaceutically acceptable carrier.
A pharmaceutical composition comprising an antisense oligonucleotide of 21 bases comprising the base sequence AGG UGG UAU CAA CAU CUG (SEQ ID NO:
- 2), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier.
A pharmaceutical composition comprising an antisense oligonucleotide of 25 bases comprising the base sequence GAU AGG UGG UAU CAA CAU CUG UAA G (SEQ ID NO:
- 3), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier.
A pharmaceutical composition comprising an antisense oligonucleotide of 20 bases comprising the base sequence GGU GGU AUC AAC AUC UGU AA (SEQ ID NO:
- 4), in which the uracil bases are thymine bases, and wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.