MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
1 Assignment
0 Petitions
Accused Products
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.
43 Citations
69 Claims
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1-65. -65. (canceled)
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66. An antisense oligonucleotide of formula (I):
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67. A pharmaceutical composition comprising:
(a) an antisense oligonucleotide of formula (I);
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68. An antisense oligonucleotide of formula (I):
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69. A pharmaceutical composition comprising:
(a) an antisense oligonucleotide of formula (I);
Specification