Antisense molecules and methods for treating pathologies
AssigneeThe University of Western Australia
InventorsStephen Donald Wilton, Sue Fletcher, Abbie Adams, Penny Meloni
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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 59.
Claims4
An antisense oligonucleotide of 27 bases in length 100% complementary to a target region of exon 44 of the human dystrophin pre-mRNA, wherein the target region is annealing site H44A(+59+85), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 44 skipping;
- or a pharmaceutically acceptable salt thereof.
An antisense oligonucleotide of 27 bases comprising the base sequence CUG UUC AGC UUC UGU UAG CCA CUG AUU (SEQ ID NO:
- 54), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide and is uniformly modified to comprise a 5-substituted pyrimidine base;
- or a pharmaceutically acceptable salt thereof.
Dependencies are inferred from the claim text.