JP2002527493A - 標的細胞による第ｖｉｉｉ因子の発現のためのアデノ随伴ベクター - Google Patents
標的細胞による第ｖｉｉｉ因子の発現のためのアデノ随伴ベクターInfo
- Publication number
- JP2002527493A JP2002527493A JP2000576889A JP2000576889A JP2002527493A JP 2002527493 A JP2002527493 A JP 2002527493A JP 2000576889 A JP2000576889 A JP 2000576889A JP 2000576889 A JP2000576889 A JP 2000576889A JP 2002527493 A JP2002527493 A JP 2002527493A
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- Prior art keywords
- factor viii
- aav
- vector
- sequence
- recombinant adeno
- Prior art date
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Classifications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (7)
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| US10499498P | 1998-10-20 | 1998-10-20 | |
| US12597499P | 1999-03-24 | 1999-03-24 | |
| US09/364,862 US6221349B1 (en) | 1998-10-20 | 1999-07-30 | Adeno-associated vectors for expression of factor VIII by target cells |
| US09/364,862 | 1999-07-30 | ||
| US60/125,974 | 1999-07-30 | ||
| US60/104,994 | 1999-07-30 | ||
| PCT/US1999/024495 WO2000023116A1 (en) | 1998-10-20 | 1999-10-19 | Adeno-associated vectors for expression of factor viii by target cells |
Publications (2)
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| JP2002527493A true JP2002527493A (ja) | 2002-08-27 |
| JP2002527493A5 JP2002527493A5 (https=) | 2005-04-07 |
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| JP2000576889A Pending JP2002527493A (ja) | 1998-10-20 | 1999-10-19 | 標的細胞による第ｖｉｉｉ因子の発現のためのアデノ随伴ベクター |
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| Country | Link |
|---|---|
| US (1) | US6221349B1 (https=) |
| EP (1) | EP1124583A4 (https=) |
| JP (1) | JP2002527493A (https=) |
| KR (1) | KR20010082234A (https=) |
| CN (1) | CN1329509A (https=) |
| AU (1) | AU757688B2 (https=) |
| BR (1) | BR9914643A (https=) |
| CA (1) | CA2348068A1 (https=) |
| IL (1) | IL142721A0 (https=) |
| MX (1) | MXPA01004067A (https=) |
| WO (1) | WO2000023116A1 (https=) |
Cited By (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2008537680A (ja) * | 2005-04-14 | 2008-09-25 | ツェー・エス・エル・ベーリング・ゲー・エム・ベー・ハー | 安定性の増大された改変型凝固第ｖｉｉｉ因子およびその誘導体 |
| JP2018078898A (ja) * | 2008-04-22 | 2018-05-24 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| JP2020527333A (ja) * | 2017-06-15 | 2020-09-10 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | パーキンソン病を治療するためのａａｄｃポリヌクレオチド |
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| US5846528A (en) | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| WO1998009524A1 (en) * | 1996-09-06 | 1998-03-12 | Chiron Corporation | Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors |
| US6797505B2 (en) * | 1998-05-27 | 2004-09-28 | Cell Genesys, Inc. | Recombinant AAV vectors for gene therapy of hemophilia A |
| AU4185699A (en) * | 1998-05-27 | 1999-12-13 | Avigen, Inc. | Adeno-associated viral vector-mediated expression of factor viii activity |
-
1999
- 1999-07-30 US US09/364,862 patent/US6221349B1/en not_active Expired - Lifetime
- 1999-10-19 CN CN99814128A patent/CN1329509A/zh active Pending
- 1999-10-19 KR KR1020017004962A patent/KR20010082234A/ko not_active Ceased
- 1999-10-19 JP JP2000576889A patent/JP2002527493A/ja active Pending
- 1999-10-19 AU AU11256/00A patent/AU757688B2/en not_active Expired
- 1999-10-19 EP EP99955065A patent/EP1124583A4/en not_active Withdrawn
- 1999-10-19 MX MXPA01004067A patent/MXPA01004067A/es not_active Application Discontinuation
- 1999-10-19 IL IL14272199A patent/IL142721A0/xx unknown
- 1999-10-19 WO PCT/US1999/024495 patent/WO2000023116A1/en not_active Ceased
- 1999-10-19 BR BR9914643-6A patent/BR9914643A/pt not_active Application Discontinuation
- 1999-10-19 CA CA002348068A patent/CA2348068A1/en not_active Abandoned
Cited By (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2008537680A (ja) * | 2005-04-14 | 2008-09-25 | ツェー・エス・エル・ベーリング・ゲー・エム・ベー・ハー | 安定性の増大された改変型凝固第ｖｉｉｉ因子およびその誘導体 |
| JP2018078898A (ja) * | 2008-04-22 | 2018-05-24 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| JP7347933B2 (ja) | 2016-04-15 | 2023-09-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 血友病ａ治療に対する遺伝子療法 |
| JP2020527333A (ja) * | 2017-06-15 | 2020-09-10 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | パーキンソン病を治療するためのａａｄｃポリヌクレオチド |
| JP2020530999A (ja) * | 2017-08-09 | 2020-11-05 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| JP7374883B2 (ja) | 2017-08-09 | 2023-11-07 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| JP2024010074A (ja) * | 2017-08-09 | 2024-01-23 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| US12168776B2 (en) | 2017-08-09 | 2024-12-17 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof |
| JP7688682B2 (ja) | 2017-08-09 | 2025-06-04 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| US12364774B2 (en) | 2018-08-09 | 2025-07-22 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
| JP2025026612A (ja) * | 2019-02-20 | 2025-02-21 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | がんを標的とする、ウイルスによってコードされる、調節可能なｔ（ｃａｔｖｅｒｔ）またはｎｋ細胞（ｃａｔｖｅｒｎ）リンカー |
| WO2025239324A1 (ja) * | 2024-05-13 | 2025-11-20 | 中外製薬株式会社 | 遺伝子改変非ヒト動物 |
Also Published As
| Publication number | Publication date |
|---|---|
| MXPA01004067A (es) | 2003-03-10 |
| EP1124583A1 (en) | 2001-08-22 |
| AU757688B2 (en) | 2003-03-06 |
| CN1329509A (zh) | 2002-01-02 |
| BR9914643A (pt) | 2001-12-11 |
| AU1125600A (en) | 2000-05-08 |
| CA2348068A1 (en) | 2000-04-27 |
| US6221349B1 (en) | 2001-04-24 |
| IL142721A0 (en) | 2002-03-10 |
| WO2000023116A1 (en) | 2000-04-27 |
| KR20010082234A (ko) | 2001-08-29 |
| EP1124583A4 (en) | 2002-04-17 |
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