Multiple exon skipping compositions for DMD
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Abstract
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 thereof to treat muscular dystrophy.
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Citations
4 Claims
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1. An antisense oligonucleotide of formula (I):
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2. A pharmaceutical composition comprising:
(a) an antisense oligonucleotide of formula (I);
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3. An antisense oligonucleotide of formula (I):
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4. A pharmaceutical composition comprising:
(a) an antisense oligonucleotide of formula (I);
Specification