Antisense oligonucleotides for inducing exon skipping and methods of use thereof
First Claim
Patent Images
1. An isolated antisense oligonucleotide of 30 to 50 nucleotides in length comprising SEQ ID NO:
- 181, wherein the uracil bases are optionally thymine bases.
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Accused Products
Abstract
Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.
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Citations
33 Claims
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1. An isolated antisense oligonucleotide of 30 to 50 nucleotides in length comprising SEQ ID NO:
- 181, wherein the uracil bases are optionally thymine bases.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21)
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22. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; anda pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for intravenous administration. - View Dependent Claims (23)
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24. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; andphosphate-buffered saline; wherein the injectable solution is formulated for intravenous administration.
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25. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; anda pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for parenteral administration. - View Dependent Claims (26)
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27. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; andphosphate-buffered saline; wherein the injectable solution is formulated for parenteral administration.
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28. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; anda pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for intramuscular administration. - View Dependent Claims (29)
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30. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; andphosphate-buffered saline; wherein the injectable solution is formulated for intramuscular administration.
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31. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; anda pharmaceutically acceptable carrier or diluent; wherein the injectable solution is formulated for subcutaneous administration. - View Dependent Claims (32)
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33. An injectable solution comprising:
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an antisense oligonucleotide of 30 nucleotides in length comprising the base sequence 5′
-CUCCAACAUCAAGGAAGAUGGCAUUUCUAG-3′
(SEQ ID NO;
181), in which the uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol chain; andphosphate-buffered saline; wherein the injectable solution is formulated for subcutaneous administration.
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Specification