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Pharmaceutical composition for treatment of Duchenne muscular dystrophy 원문보기

IPC분류정보
국가/구분 United States(US) Patent 등록
국제특허분류(IPC7판)
  • C07H-021/04
  • C12Q-001/68
  • C12P-019/34
출원번호 US-0930251 (2001-08-16)
우선권정보 JP-0256547 (2000-08-25)
발명자 / 주소
  • Matsuo, Masafumi
  • Kamei, Shoichiro
출원인 / 주소
  • JCR Pharmaceuticals Co., Ltd., Matsuo
대리인 / 주소
    Greenblum & Bernstein, P.L.C.
인용정보 피인용 횟수 : 66  인용 특허 : 7

초록 ▼

The invention provides an isolated and purified DNA set forth as SEQ ID NO:15 in the Sequence Listing and an antisense oligonucleotide complementary to the DNA. The DNA represents the splicing enhancer sequence (SES) in exon 45 of human dystrophin gene, and serves as a template in preparation of the

대표청구항 ▼

1. An isolated and purified oligonucleotide selected from the group consisting of: a DNA consisting of a nucleotide sequence set forth as SEQ ID NO:15 in the Sequence Listing, andan RNA consisting of a nucleotide sequence set forth as SEQ ID NO:15 in the Sequence Listing in which all thymine nucleot

이 특허에 인용된 특허 (7)

  1. Baker Brenda F. ; Cowsert Lex M., Antisense inhibition of Elk-1 expression.
    상세보기
  2. Kole Ryszard (Chapel Hill NC) Dominski Zbigniew (Chapel Hill NC), Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
    상세보기
  3. Kole Ryszard (Orange County NC) Dominski Zbigniew (Orange County NC), Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
    상세보기
  4. Kole Ryszard ; Dominski Zbigniew, Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
    상세보기
  5. Kole Ryszard ; Dominski Zbigniew, Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
    상세보기
  6. Gottschalk Peter (Centerville OH) Neckers Douglas C. (Perrysburg OH) Schuster Gary B. (Champaign IL), Cationic dye-triarylmonoalkylorate anion complexes.
    상세보기
  7. Stec Wojciech J. (Lodz PLX) Uznanski Bogdan (Lodz CA PLX) Bergot B. John (Redwood City CA) Hirschbein Bernard L. (San Francisco CA) Fearon Karen L. (Union City CA), Method of synethesizing sulfurized oligonucleotide analogs.
    상세보기

이 특허를 인용한 특허 (66)

  1. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
    상세보기
  2. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
    상세보기
  3. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
    상세보기
  4. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  5. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  6. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  7. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  8. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  9. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  10. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  11. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  12. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  13. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  14. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  15. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  16. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  17. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  18. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  19. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  20. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  21. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
    상세보기
  22. Krieg, Arthur M.; Subramanian, Romesh; McSwiggen, James; Lee, Jeannie T., Compositions and methods for modulating ATP2A2 expression.
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  23. Krieg, Arthur M.; Subramanian, Romesh; McSwiggen, James; Lee, Jeannie T., Compositions and methods for modulating SMN gene family expression.
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  24. Krieg, Arthur M.; Subramanian, Romesh; McSwiggen, James; Lee, Jeannie T., Compositions and methods for modulating UTRN expression.
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  25. Krieg, Arthur M.; Subramanian, Romesh; McSwiggen, James; Lee, Jeannie T., Compositions and methods for modulating hemoglobin gene family expression.
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  26. Dale, Roderic M. K., Compositions and methods for the treatment of muscle wasting.
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  27. Kaye, Edward M., Compositions for treating muscular dystrophy.
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  28. Bestwick, Richard K.; Frank, Diane Elizabeth, Exon skipping compositions for treating muscular dystrophy.
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  29. van Ommen, Garrit-Jan B.; van Deutekom, Judith C. T.; den Dunnen, Johannes T., Induction of exon skipping in eukaryotic cells.
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  30. De Kimpe, Josephus Johannes; Platenburg, Gerard Johannes; Van Deutekom, Judith Christina Theodora; Aartsma-Rus, Annemieke; Van Ommen, Garrit-Jan Boudewijn, Means and methods for counteracting muscle disorders.
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  31. Platenburg, Gerard Johannes; de Kimpe, Josephus Johannes; van Deutekom, Judith Christina Theodora; van Ommen, Garrit-Jan Boudewijn; Aartsma-Rus, Annemieke, Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means.
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  32. Platenburg, Gerard Johannes; de Kimpe, Josephus Johannes; van Deutekom, Judith Christina Theodora; van Ommen, Garrit-Jan Boudewijn; Aartsma-Rus, Annemieke, Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means.
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  33. van Deutekom, Judith Christina Theodora, Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene.
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  34. De Kimpe, Josephus Johannes; Platenburg, Gerardus Johannes; Van Deutekom, Judith Christina Theodora; Aartsma-Rus, Annemieke; Van Ommen, Garrit-Jan Boudewijn, Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA.
    상세보기
  35. De Kimpe, Josephus Johannes; Rus, Adriana Marie; Platenburg, Gerard Johannes; Van Deutekom, Judith Christina Theodora; Van Ommen, Garrit-Jan Boudewijn, Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA.
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  36. Uhlmann, Eugen; Weber, Markus; Subramanian, Romesh; Rockel, Thomas Dino; Krieg, Arthur M., Methods of delivering multiple targeting oligonucleotides to a cell using cleavable linkers.
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  37. Van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering RNA structure.
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  38. Van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
    상세보기
  39. Van Deutekom, Judith Christina Theodora; van Ommen, Garrit-Jan B.; Aartsma-Rus, Annemieke; den Dunnen, Johannes Theodorus, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
    상세보기
  40. van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
    상세보기
  41. van Ommen, Garrit-Jan Boudewijn; van Deutekom, Judith Christina Theodora; den Dunnen, Johannes Theodorus; Aartsma-Rus, Annemieke, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
    상세보기
  42. Van Ommen, Garrit-Jan Boudewijn; Aartsma-Rus, Annemieke; Van Deutekom, Judith Christina Theodora; De Kimpe, Josephus Johannes; Verbeek, Joseph Stephan; Ylmaz-Elis, Aliye Seda, Molecule for treating an inflammatory disorder.
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  43. Kuik-Romeijn, Petra Van; Platenburg, Gerard Johannes, Molecules for targeting compounds to various selected organs, tissues or tumor cells.
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  44. Uhlmann, Eugen; Subramanian, Romesh; Krieg, Arthur M., Multimeric oligonucleotide compounds having non-nucleotide based cleavable linkers.
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  45. Uhlmann, Eugen; Weber, Markus; Subramanian, Romesh; Rockel, Thomas Dino; Krieg, Arthur M., Multimeric oligonucleotides compounds.
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  46. Uhlmann, Eugen; Weber, Markus; Subramanian, Romesh; Rockel, Thomas Dino; Krieg, Arthur M., Multimeric oligonucleotides compounds having cleavable linkers.
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  47. Uhlmann, Eugen; Weber, Markus; Subramanian, Romesh; Rockel, Thomas Dino; Krieg, Arthur M., Multimetric oligonucleotide compounds.
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  48. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  49. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  50. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  51. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  52. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  53. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
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  54. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
    상세보기
  55. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAS.
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  56. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
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  57. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
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  58. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
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  59. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
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  60. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
    상세보기
  61. de Visser, Peter Christian; van Deutekom, Judith Christina Theodora, RNA modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy.
    상세보기
  62. Iversen, Patrick L.; Hudziak, Robert, Splice-region antisense composition and method.
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  63. Iversen, Patrick L.; Hudziak, Robert, Splice-region antisense composition and method.
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  64. Iversen, Patrick L.; Hudziak, Robert, Splice-region antisense composition and method.
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  65. Iversen, Patrick L.; Hudziak, Robert; Weller, Dwight D., Splice-region antisense composition and method.
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  66. De Kimpe, Josephus Johannes; Platenburg, Gerard Johannes; Wansink, Derick Gert, Treatment of genetic disorders associated with DNA repeat instability.
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