EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
First Claim
Patent Images
1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides of a nucleotide sequence selected from the group consisting of:
- SEQ ID NOs;
1-7, wherein the oligonucleotide specifically hybridizes to an exon 44 target region of the human dystrophin gene and induces exon 44 skipping, and wherein thymine bases are optionally uracil bases.
1 Assignment
0 Petitions
Accused Products
Abstract
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
-
Citations
42 Claims
-
1. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides of a nucleotide sequence selected from the group consisting of:
- SEQ ID NOs;
1-7, wherein the oligonucleotide specifically hybridizes to an exon 44 target region of the human dystrophin gene and induces exon 44 skipping, and wherein thymine bases are optionally uracil bases. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 35, 36, 37, 38, 39, 40, 41, 42)
- SEQ ID NOs;
-
18. An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides complementary to an exon 44 target region of the human dystrophin gene designated as an annealing site selected from the group consisting of:
- H44A(−
07+17), H44A(−
07+20), H44A(−
07+22), H44A(+77+101), H44A(+64+91), H44A(+62+89), and H44A(+62+85), wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 44 skipping. - View Dependent Claims (19, 20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34)
- H44A(−
Specification