| 摘要 |
PROBLEM TO BE SOLVED: To provide multiple exon skipping compositions for DMD.SOLUTION: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use the antisense molecules to treat muscular dystrophy. Embodiments of the present invention relate generally to antisense compounds capable of binding to a selected target to induce exon skipping, and methods of use the antisense compounds to induce exon skipping. In certain embodiments, it is possible to combine two or more antisense oligonucleotides of the present invention together to induce single or multiple exon skipping. |
