Antisense molecules and methods for treating pathologies
AssigneeThe University of Western Australia
InventorsStephen Donald Wilton, Sue Fletcher, Abbie Adams, Penny Meloni
Life of this patent
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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 59.
Claims36
An antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, or a pharmaceutically acceptable salt thereof.
An antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping.
A pharmaceutically acceptable salt of an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, or a pharmaceutically acceptable salt thereof, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient a pharmaceutically acceptable salt of an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 exon 9 skipping, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
A pharmaceutical composition comprising (i) an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, or a pharmaceutically acceptable salt thereof, and (ii) a pharmaceutically acceptable carrier.
A pharmaceutical composition comprising (i) an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, and (ii) a pharmaceutically acceptable carrier.
A pharmaceutical composition comprising (i) a pharmaceutically acceptable salt of an antisense oligonucleotide of 25 bases in length wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, and (ii) a pharmaceutically acceptable carrier.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient a pharmaceutical composition comprising (i) an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, or a pharmaceutically acceptable salt thereof, and (ii) a pharmaceutically acceptable carrier, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient a pharmaceutical composition comprising (i) an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, and (ii) a pharmaceutically acceptable carrier, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
A method for restoring an mRNA reading frame to induce dystrophin protein production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 8 and exon 9 skipping, comprising administering to the patient a pharmaceutical composition comprising (i) a pharmaceutically acceptable salt of an antisense oligonucleotide of 25 bases in length, wherein the antisense oligonucleotide is 100% complementary to a target region of exon 8 of the human dystrophin pre-mRNA, wherein the target region is annealing site H8A(+42+66), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide specifically hybridizes to the annealing site inducing exon 8 and exon 9 skipping, and (ii) a pharmaceutically acceptable carrier, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient.
Dependencies are inferred from the claim text.