Exon skipping compositions for treating muscular dystrophy
AssigneeSarepta Therapeutics, Inc.
InventorsRichard K. BESTWICK, Diane Elizabeth FRANK
Life of this patent
Estimating the term…
Abstract
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
Claims30
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, 9, 11, and 14-18,wherein the antisense oligonucleotide comprises a non-naturally occurring backbone, wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene and induces exon 53 skipping, and wherein thymine bases are optionally uracil bases.
-6. (canceled)
-10. (canceled)
-14. (canceled)
(canceled)
An isolated antisense oligonucleotide of 20 to 50 nucleotides in length comprising at least 10 consecutive nucleotides complementary to an exon 53 target region of the human dystrophin gene designated as an annealing site selected from the group consisting of:
- H53A(+36+60), H53A(+33+60), H53A(+31+55), H53A(+22+46), H53A(+30+57), H53A (+30+56), H53A(+30+55) and H53A(+33+57), wherein the antisense oligonucleotide comprises a non-naturally occurring backbone, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 53 skipping.
-23. (canceled)
-27. (canceled)
-31. (canceled)
(canceled)
-38. (canceled)
A pharmaceutical composition, comprising an 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, 9, 11, and 14-18,wherein the antisense oligonucleotide comprises a non-naturally occurring backbone, wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene and induces exon 53 skipping, and wherein thymine bases are optionally uracil bases, and a saline solution that includes a phosphate buffer.
A method of treating Duchenne muscular dystrophy, comprising administering to a patient in need thereof an effective amount of an 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, 9, 11, and 14-18,wherein the antisense oligonucleotide comprises a non-naturally occurring backbone, wherein the antisense oligonucleotide specifically hybridizes to an exon 53 target region of the human dystrophin gene and induces exon 53 skipping, and wherein thymine bases are optionally uracil bases.
-43. (canceled)
Dependencies are inferred from the claim text.