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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-0917907 (2001-07-31)
우선권정보 JP-0140930 (1999-05-21)
발명자 / 주소
  • Matsuo, Masafumi
출원인 / 주소
  • JCR Pharmaceuticals Co., Ltd., Matsuo
  • Masafumi
대리인 / 주소
    Greenblum & Bernstein P.L.C.
인용정보 피인용 횟수 : 57  인용 특허 : 7

초록

A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dys

대표청구항

1. A method of manufacturing a composition, the method comprising: forming an antisense oligonucleotide consisting of a nucleotide sequence fully complementary to the sequence represented by SEQ ID NO: 1; dissolving the antisense oligonucleotide in a pharmaceutically acceptable injectable medium

이 특허에 인용된 특허 (7)

  1. Baker Brenda F. ; Cowsert Lex M., Antisense inhibition of Elk-1 expression.
  2. Kole Ryszard (Chapel Hill NC) Dominski Zbigniew (Chapel Hill NC), Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
  3. Kole Ryszard (Orange County NC) Dominski Zbigniew (Orange County NC), Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
  4. Kole Ryszard ; Dominski Zbigniew, Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
  5. Kole Ryszard ; Dominski Zbigniew, Antisense oligonucleotides which combat aberrant splicing and methods of using the same.
  6. Gottschalk Peter (Centerville OH) Neckers Douglas C. (Perrysburg OH) Schuster Gary B. (Champaign IL), Cationic dye-triarylmonoalkylorate anion complexes.
  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.

이 특허를 인용한 특허 (57)

  1. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
  2. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
  3. Wilton, Stephen Donald; Fletcher, Sue; Adams, Abbie; Meloni, Penny, Antisense molecules and methods for treating pathologies.
  4. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  5. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  6. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  7. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  8. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  9. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  10. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  11. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  12. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  13. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  14. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  15. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  16. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  17. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  18. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  19. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  20. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  21. Wilton, Stephen Donald; Fletcher, Sue; McClorey, Graham, Antisense oligonucleotides for inducing exon skipping and methods of use thereof.
  22. Kaye, Edward M., Compositions for treating muscular dystrophy.
  23. Bestwick, Richard K.; Frank, Diane Elizabeth, Exon skipping compositions for treating muscular dystrophy.
  24. van Ommen, Garrit-Jan B.; van Deutekom, Judith C. T.; den Dunnen, Johannes T., Induction of exon skipping in eukaryotic cells.
  25. Aartsma-Rus, Annemieke; Van Deutekom, Judith Christina T.; Van Ommen, Garrit-Jan Boudewijn, Means and method for inducing exon-skipping.
  26. 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.
  27. 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.
  28. 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.
  29. Fahrenkrug, Scott C.; Carlson, Daniel F., Methods and materials for producing transgenic artiodactyls.
  30. 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.
  31. 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.
  32. 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.
  33. de Kimpe, Josephus Johannes; Platenburg, Gerardus Johannes, Methods and means for treating DNA repeat instability associated genetic disorders.
  34. Van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering RNA structure.
  35. Van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
  36. 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.
  37. van Deutekom, Judith Christina Theodora, Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure.
  38. 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.
  39. 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.
  40. Heemskerk, Johannes Antonius; Van Deutekom, Judith Christina Theodora; Van Kuik-Romeijn, Petra; Platenburg, Gerard Johannes, Molecules for targeting compounds to various selected organs or tissues.
  41. Kuik-Romeijn, Petra Van; Platenburg, Gerard Johannes, Molecules for targeting compounds to various selected organs, tissues or tumor cells.
  42. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  43. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  44. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  45. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  46. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  47. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  48. Sazani, Peter; Kole, Ryszard, Multiple exon skipping compositions for DMD.
  49. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAS.
  50. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
  51. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
  52. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
  53. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
  54. Lee, Jeannie T.; Zhao, Jing; Sarma, Kavitha; Borowsky, Mark; Ohsumi, Toshiro Kendrick, Polycomb-associated non-coding RNAs.
  55. de Visser, Peter Christian; van Deutekom, Judith Christina Theodora, RNA modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy.
  56. 't Hoen, Peter Abraham Christiaan; Sterrenburg, Petronella Johanna Elisabeth; den Dunnen, Johannes Theodorus; van Ommen, Garrit Jan Boudewijn, Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly or abnormally expressed gene.
  57. De Kimpe, Josephus Johannes; Platenburg, Gerard Johannes; Wansink, Derick Gert, Treatment of genetic disorders associated with DNA repeat instability.
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