ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
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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.
142 Citations
27 Claims
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1-14. -14. (canceled)
- 15. A method of inducing exon-skipping of dystrophin exon 51, comprising introducing a nucleic acid molecule into a cell by way of an expression vector, wherein the nucleic acid molecule is 10 to about 50 nucleotides in length and is specifically hybridizable to an exon 51 target region of the dystrophin gene designated as annealing site H51A (+66+95).
Specification