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.
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Citations
22 Claims
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1-20. -20. (canceled)
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21. An antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO:
- 175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping;
or a pharmaceutically acceptable salt thereof.
- 175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping;
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22. A pharmaceutical composition comprising:
- (i) an antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO;
175), in which uracil bases are thymine bases, wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 50 skipping, or a pharmaceutically acceptable salt thereof; and
(ii) a pharmaceutically acceptable carrier.
- (i) an antisense oligonucleotide of 20 to 31 bases comprising a base sequence that is 100% complementary to consecutive bases of the human dystrophin pre-mRNA, wherein the base sequence comprises at least 20 consecutive bases of GGG AUC CAG UAU ACU UAC AGG CUC C (SEQ ID NO;
Specification