Antisense Oligonucleotides
First Claim
1. An antisense oligonucleotide molecule for treating DMD, the antisense oligonucleotide molecule comprising a base sequence selected from the group consisting of:
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Abstract
Embodiments of the present invention are directed generally to antisense compounds and compositions for the treatment of muscular dystrophy, and in particular, Duchenne muscular dystrophy (DMD). In one embodiment, the invention is directed to antisense oligonucleotide molecules, pharmaceutical compositions and formulations comprising antisense oligonucleotide molecules, and methods of treating muscular dystrophy related diseases and disorders wherein the antisense oligonucleotide molecules comprises a base sequence selected from the group consisting of SEQ ID NO: 5-8, 10, 12, 14, 16, 24, 27, 28, 34, 35, 37, 40, 42, 44-46, 79, 97, 100, 101, and 116, and combinations thereof.
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Citations
21 Claims
- 1. An antisense oligonucleotide molecule for treating DMD, the antisense oligonucleotide molecule comprising a base sequence selected from the group consisting of:
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8. A pharmaceutical composition comprising
an antisense oligonucleotide conjugated with a polymer to enhance the delivery of antisense oligonucleotide and antisense effect, wherein the antisense oligonucleotide is selected from the group consisting of
- 14. A method of treating muscular dystrophy in a subject, comprising administering to the subject an effective amount of an antisense oligonucleotide molecule selected from the group consisting of
Specification