EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
First Claim
1. An antisense oligomer of 20-50 nucleotides in length capable of binding a selected target to induce exon skipping in the human dystrophin gene, wherein said antisense oligomer comprises a sequence of bases that specifically hybridizes to an exon 53 target region selected from the group consisting of H53A(+36+60), H53A(+30+57), H53A (+30+56), H53A(+30+55) and H53A(+33+57), wherein the bases of said oligomer are linked to morpholino ring structures, and wherein said morpholino ring structures are joined by phosphorous-containing intersubunit linkages joining a morpholino nitrogen of one ring structure to a 5′
- exocyclic carbon of an adjacent ring structure.
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Accused Products
Abstract
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
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Citations
20 Claims
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1. An antisense oligomer of 20-50 nucleotides in length capable of binding a selected target to induce exon skipping in the human dystrophin gene, wherein said antisense oligomer comprises a sequence of bases that specifically hybridizes to an exon 53 target region selected from the group consisting of H53A(+36+60), H53A(+30+57), H53A (+30+56), H53A(+30+55) and H53A(+33+57), wherein the bases of said oligomer are linked to morpholino ring structures, and wherein said morpholino ring structures are joined by phosphorous-containing intersubunit linkages joining a morpholino nitrogen of one ring structure to a 5′
- exocyclic carbon of an adjacent ring structure.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 17, 18, 19, 20)
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15. The antisense oligomer are modified with a pendant cationic group.
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16. An antisense oligomer, wherein the oligomer has a structure selected from the group consisting of:
Specification