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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- 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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- 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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- 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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- 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
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- 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
- 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
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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
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- 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
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| US60/125,974 | 1999-03-24 | ||
| US09/364,862 | 1999-07-30 | ||
| US09/364,862 US6221349B1 (en) | 1998-10-20 | 1999-07-30 | Adeno-associated vectors for expression of factor VIII by target cells |
| PCT/US1999/024495 WO2000023116A1 (en) | 1998-10-20 | 1999-10-19 | Adeno-associated vectors for expression of factor viii by target cells |
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| JP2002527493A true JP2002527493A (ja) | 2002-08-27 |
| JP2002527493A5 JP2002527493A5 (enExample) | 2005-04-07 |
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| EP (1) | EP1124583A4 (enExample) |
| JP (1) | JP2002527493A (enExample) |
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| AU (1) | AU757688B2 (enExample) |
| BR (1) | BR9914643A (enExample) |
| CA (1) | CA2348068A1 (enExample) |
| IL (1) | IL142721A0 (enExample) |
| MX (1) | MXPA01004067A (enExample) |
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Cited By (6)
| 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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- 1999-10-19 CN CN99814128A patent/CN1329509A/zh active Pending
- 1999-10-19 BR BR9914643-6A patent/BR9914643A/pt not_active Application Discontinuation
- 1999-10-19 EP EP99955065A patent/EP1124583A4/en not_active Withdrawn
- 1999-10-19 KR KR1020017004962A patent/KR20010082234A/ko not_active Ceased
- 1999-10-19 WO PCT/US1999/024495 patent/WO2000023116A1/en not_active Ceased
- 1999-10-19 IL IL14272199A patent/IL142721A0/xx unknown
Cited By (10)
| 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 |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2000023116A1 (en) | 2000-04-27 |
| BR9914643A (pt) | 2001-12-11 |
| IL142721A0 (en) | 2002-03-10 |
| EP1124583A4 (en) | 2002-04-17 |
| CA2348068A1 (en) | 2000-04-27 |
| MXPA01004067A (es) | 2003-03-10 |
| AU757688B2 (en) | 2003-03-06 |
| AU1125600A (en) | 2000-05-08 |
| EP1124583A1 (en) | 2001-08-22 |
| KR20010082234A (ko) | 2001-08-29 |
| US6221349B1 (en) | 2001-04-24 |
| CN1329509A (zh) | 2002-01-02 |
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