Antisense oligonucleotides for inducing exon skipping and methods of use thereof
AssigneeThe University of Western Australia
InventorsStephen Donald Wilton, Sue Fletcher, Graham McClorey
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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.
Claims25
An isolated antisense oligonucleotide that is specifically hybridizable to an exon 53 target region of the Dystrophin gene designated as annealing site H53A (+23+47), wherein the antisense oligonucleotide consists of the sequence identified as SEQ ID NO:
- 195, wherein the uracil bases are optionally thymine bases.
A method of inducing exon-skipping of a dystrophin exon 53, comprising delivering an antisense oligonucleotide that is specifically hybridizable to an exon 53 target region of the dystrophin gene designated as annealing site H53A (+23+47), wherein the antisense oligonucleotide consists of the sequence identified as SEQ ID NO:
- 195, wherein the uracil bases are optionally thymine bases, thereby inducing exon-skipping of the dystrophin exon.
Dependencies are inferred from the claim text.