IT IS CLAIMED:
`
`Atty Docket No. 50450.8090
`
`1. A composition for use in producing skipping of exon 44 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 1-20, and capable of forming with the complementary sequence in the dsytrophn-gene
`
`exon 44, a heteroduplex structure between said compoundandtranscript having a Tm ofat least 45°C.
`
`10
`
`2. The composition of claim 1, wherein the compound contains a sequence selected from the
`
`group consisting of SEQ ID NOS: 1-5 and 16-20.
`
`3. The composition of claim 1, wherein the compoundis conjugated to an arginine-rich
`
`15
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`4. A composition for use in producing skipping of exon 45 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`20
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 21-76, and capable of forming with the complementary sequencein the dsytrophn-gene
`
`exon 45, a heteroduplex structure between said compound andtranscript having a Tm ofatleast 45°C.
`
`25
`
`5. The composition of claim 4, wherein the compound contains a sequence selected from the
`
`group consisting of SEQ ID NOS: 21-53.
`
`6. The composition of claim 5, wherein the compound contains a sequenceselected from the
`
`group consisting of SEQ ID NOS: 21-25 and 49-53.
`
`30
`
`7. The composition of claim 4, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`8. A composition for use in producing skipping of exon 46 in the processing of human
`
`ATL_IMANAGE-5365690 v1
`
`31
`
`
`
`Atty Docket No. 50450.8090
`
`1. A composition for use in producing skipping of exon 44 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 1-20, and capable of forming with the complementary sequence in the dsytrophn-gene
`
`exon 44, a heteroduplex structure between said compoundandtranscript having a Tm ofat least 45°C.
`
`10
`
`2. The composition of claim 1, wherein the compound contains a sequence selected from the
`
`group consisting of SEQ ID NOS: 1-5 and 16-20.
`
`3. The composition of claim 1, wherein the compoundis conjugated to an arginine-rich
`
`15
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`4. A composition for use in producing skipping of exon 45 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`20
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 21-76, and capable of forming with the complementary sequencein the dsytrophn-gene
`
`exon 45, a heteroduplex structure between said compound andtranscript having a Tm ofatleast 45°C.
`
`25
`
`5. The composition of claim 4, wherein the compound contains a sequence selected from the
`
`group consisting of SEQ ID NOS: 21-53.
`
`6. The composition of claim 5, wherein the compound contains a sequenceselected from the
`
`group consisting of SEQ ID NOS: 21-25 and 49-53.
`
`30
`
`7. The composition of claim 4, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`8. A composition for use in producing skipping of exon 46 in the processing of human
`
`ATL_IMANAGE-5365690 v1
`
`31
`
`
`
`Atty Docket No. 50450.8090
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 77-125, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 46, a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`45°C.
`
`9. The composition of claim 8, wherein the compoundcontains a sequence selected from the
`
`10
`
`group consisting of SEQ ID NOS: 77-105.
`
`10. The composition of clatm 9, wherein the compoundcontains a sequence selected from the
`
`group consisting of SEQ ID NOS: 77-81 and 101-105.
`
`15
`
`11. The composition of claim 8, wherein the compound is conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`12. A composition for use in producing skipping of exon 47 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`20
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 126-169, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 47, a heteroduplex structure between said compoundandtranscript having a Tm of at least
`
`25
`
`45°C.
`
`13. The composition of claim 12, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 126-149.
`
`30
`
`14. The composition of claim 13, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 126-130 and 145-149.
`
`15. The composition of claim 12, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`ATL_IMANAGE-5365690 v1
`
`32
`
`
`
`Atty Docket No. 50450.8090
`
`16. A composition for use in producing skipping of exon 48 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 170-224, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 48, a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`45°C.
`
`10
`
`17. The composition of claim 16, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 170-201.
`
`18. The composition of claim 17, wherein the compound contains a sequenceselected from
`
`15
`
`the group consisting of SEQ ID NOS: 170-174 and 197-201.
`
`19. The composition of claim 16, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`20
`
`20. A composition for use in producing skipping of exon 49 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`25
`
`SEQ ID NOS: 225-266, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 49, a heteroduplex structure between said compoundandtranscript having a Tm of at least
`
`45°C.
`
`21. The composition of claim 20, wherein the compound contains a sequence selected from
`
`30
`
`the group consisting of SEQ ID NOS: 225-248.
`
`22. The composition of claim 21, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 225-229 and 244-248.
`
`ATL_IMANAGE-5365690 v1
`
`33
`
`
`
`23. The composition of claim 20, wherein the compound is conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`Atty Docket No. 50450.8090
`
`24. A composition for use in producing skipping of exon 50 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 267-308, and capable of forming with the complementary sequence in the dsytrophn-
`
`10
`
`gene exon 50, a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`45°C.
`
`25. The composition of claim 24, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 267-291.
`
`15
`
`26. The composition of claim 25, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 267-271 and 287-291.
`
`27. The composition of claim 24, wherein the compound is conjugated to an arginine-rich
`
`20
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`28. A composition for use in producing skipping of exon 51 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`25
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 309-371, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 51, a heteroduplex structure between said compoundandtranscript having a Tm of at least
`
`45°C.
`
`30
`
`29. The composition of claim 28, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 309-345.
`
`ATL_IMANAGE-5365690 v1
`
`34
`
`
`
`30. The composition of claim 29, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 309-313 and 341-345.
`
`Atty Docket No. 50450.8090
`
`31. The composition of claim 28, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`32. A composition for use in producing skipping of exon 52 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`10
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 372-415, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 52, a heteroduplex structure between said compoundandtranscript having a Tm of at least
`
`45°C,
`
`15
`
`33. The composition of claim 32, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 372-397.
`
`34. The composition of claim 33, wherein the compound contains a sequenceselected from
`
`20
`
`the group consisting of SEQ ID NOS: 372-376 and 393-397.
`
`35. The composition of claim 32, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`25
`
`36. A composition for use in producing skipping of exon 53 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`30
`
`SEQ ID NOS: 416-475, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 53 a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`45°C,
`
`ATL_IMANAGE-5365690 v1
`
`35
`
`
`
`37. The composition of claim 36, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 416-451.
`
`Atty Docket No. 50450.8090
`
`38. The composition of claim 37, wherein the compound contains a sequenceselected from
`
`the group consisting of SEQ ID NOS: 416-420 and 447-451.
`
`39. The composition of claim 36, wherein the compoundis conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`10
`
`40. A composition for use in producing skipping of exon 54 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`15
`
`SEQ ID NOS: 476-519, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 54 a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`45°C.
`
`41. The composition of claim 40, wherein the compound contains a sequence selected from
`
`20
`
`the group consisting of SEQ ID NOS: 476-499.
`
`42. The composition of claim 41, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 476-480 and 495-499.
`
`25
`
`43. The composition of claim 40, wherein the compound is conjugated to an arginine-rich
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`44. A composition for use in producing skipping of exon 55 in the processing of human
`
`dystrophin pre-processed mRNA, comprising
`
`30
`
`a substantially uncharged antisense compound containing 20-35 morpholino subunits linked
`
`by phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5'
`
`exocyclic carbon of an adjacent subunit, containing a sequence selected from the group consisting
`
`SEQ ID NOS: 520-569, and capable of forming with the complementary sequence in the dsytrophn-
`
`gene exon 55 a heteroduplex structure between said compoundandtranscript having a Tm ofat least
`
`ATL_IMANAGE-5365690 v1
`
`36
`
`
`
`45°C.
`
`Atty Docket No. 50450.8090
`
`45. The composition of claim 44, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 520-546.
`
`46. The composition of claim 45, wherein the compound contains a sequence selected from
`
`the group consisting of SEQ ID NOS: 520-524 and 542-546.
`
`47. The composition of claim 44, wherein the compoundis conjugated to an arginine-rich
`
`10
`
`peptide having a sequence selected from the group consisting of SEQ ID NOS: 570-578.
`
`ATL_IMANAGE-5365690 v1
`
`37
`
`
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