Antisense molecules and methods for treating pathologies
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.
1 . 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.
2 . 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.
3 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is chemically linked to one or more moieties or conjugates that enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotide.
4 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain.