Antisense oligonucleotides for inducing exon skipping and methods of use thereof
AssigneeThe University of Western Austrailia
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.
Claims43
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides of SEQ ID NO:203, wherein the oligonucleotide specifically hybridizes to an exon 46 target region of the human dystrophin gene inducing exon 46 skipping, and wherein uracil bases are optionally thymine bases.
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 20 consecutive nucleotides complementary to an exon 46 target region of the human dystrophin gene designated as annealing site H46A(+86+115), wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 46 skipping, and wherein uracil bases in the antisense oligonucleotide are optionally thymine bases.
Dependencies are inferred from the claim text.