Exon skipping compositions for treating muscular dystrophy
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
1. A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an antisense oligonucleotide of 28 bases comprising the base sequence of SEQ ID NO: 6, wherein the oligonucleotide specifically hybridizes to an exon 44 target region of the Dystrophin pre-mRNA and induces exon 44 skipping, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide and wherein the thymine bases are optionally uracil bases;
or a pharmaceutically acceptable salt thereof.
2. The method of claim 1 , 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.
3. The method of claim 1 , wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol moiety.
4. The method of claim 1 , wherein the antisense oligonucleotide is administered intravenously.