JPH11506318A - 組換えウイルスベクター系 - Google Patents
組換えウイルスベクター系Info
- Publication number
- JPH11506318A JPH11506318A JP8534946A JP53494696A JPH11506318A JP H11506318 A JPH11506318 A JP H11506318A JP 8534946 A JP8534946 A JP 8534946A JP 53494696 A JP53494696 A JP 53494696A JP H11506318 A JPH11506318 A JP H11506318A
- Authority
- JP
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- Prior art keywords
- dna
- recombinant
- sequence
- nucleic acid
- aav
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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Classifications
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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
- 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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- A—HUMAN NECESSITIES
- 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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- 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
- 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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- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Description
Claims (1)
- 【特許請求の範囲】 1.(a) 高度にまたは中程度にストリンジェントなハイブリダイゼーション条件 下で図9に示した配列(配列番号1)にハイブリダイズし、かつ (b) 下記の核酸分子を含有する組換えDNAの複製、およびこの組換えDN AのAAVビリオンへの組立ておよび/または宿主ゲノムへの組込みを指令する ことができる、 DNAヌクレオチド配列を含んでなる、精製および単離された核酸分子。 2.図9に示した165塩基対の逆方向末端反復配列(配列番号1)を含んでな る、精製および単離された核酸分子。 3.目的のタンパク質をコードするDNAヌクレオチド配列および図9に示した 165塩基対の逆方向末端反復配列(配列番号1)を含んでなる、組換えDNA ベクター。 4.目的のタンパク質をコードするDNAヌクレオチド配列および請求項1に記 載の核酸分子を含んでなる、組換えDNAベクター。 5.AAVのREPおよびCAPタンパク質をコードするDNAヌクレオチド配 列をさらに含んでなる、請求項3または4に記載の組換えDNAベクター。 6.細菌のγδリゾルベースにより認識される図10に示したDNAヌクレオチ ド配列(配列番号2)をさらに含んでなる、請求項3または4に記載の組換えD NAベクター。 7.細菌のγδリゾルベースにより認識される図10に示したDNAヌクレオチ ド配列(配列番号2)をさらに含んでなる、請求項5に記載の組換えDNAベク ター。 8.組換えDNA分子を複製し、そのDNA分子をキャプシドで包み込んでAA Vビリオンとする方法であって、 (a)(i)ヘルパーウイルス、(ii)AAVのREPおよびCAPタンパク質を コードする組換え核酸、および(iii)目的のDNA配列および図9に示した16 5塩基対の逆方向末端反復配列(配列番号1)を含む組換え核酸、を含有する真 核細胞を培養し、それにより165塩基対の逆方向末 端反復配列および目的のDNA配列を含む前記核酸を複製させ、かつAAVビリ オンへと組み立て、そして (b) 産生されたビリオンを集める、 ことを含んでなる方法。 9.組換えDNA分子を複製し、そのDNA分子をキャプシドで包み込んでAA Vビリオンとする方法であって、 (a)(i)ヘルパーウイルス、(ii)AAVのREPおよびCAPタンパク質を コードする組換え核酸、および(iii)目的のDNA配列および請求項1に記載の 核酸分子を含む組換え核酸、を含有する真核細胞を培養し、それにより目的のD NA配列および請求項1に記載の核酸分子を含む前記組換え核酸を複製させ、か つAAVビリオンへと組み立て、そして (b) 産生されたビリオンを集める、 ことを含んでなる方法。 10.ヘルパーウイルスがアデノウイルスである、請求項8または9に記載の方法 。 11.ヘルパーウイルスが単純ヘルペスウイルスである、請求項8または9に記載 の方法。 12.AAVのREPおよびCAPタンパク質をコードする組換え核酸が、 (a) AAVのREPおよびCAP RNAの発現を制御するプロモーター、 (b) REPおよびCAPのmRNAの翻訳開始シグナル、 (c) REPおよびCAPタンパク質をコードするDNA配列、および (d) 転写終結シグナル、 を含有するプラスミドベクターである、請求項8または9に記載の方法。 13.目的のDNA配列および165塩基対の末端反復配列を含む前記組換え核酸 が、 (a) プロモーター、 (b) このプロモーターの制御下で転写される目的のDNA配列、および (c) 図9に示した165塩基対の逆方向末端反復配列(配列番号1)、 を含有するプラスミドベクターである、請求項8に記載の方法。 14.目的のDNA配列および165塩基対の末端反復配列を含む前記組換え核酸 が、 (a) プロモーター、 (b) このプロモーターの制御下で転写される目的のDNA配列、および (c) 請求項1に記載の核酸分子、 を含有するプラスミドベクターである、請求項8に記載の方法。 15.組換えDNA分子を複製し、そのDNA分子をキャプシドで包み込んでウイ ルス粒子とする方法であって、 (a)(i)目的の遺伝子を含む組換えウイルスベクター配列、(ii)ヘルパー機 能を与えるウイルス遺伝子、および(iii)前記ウイルスベクター配列の両端に隣 接する2つの図10に示した細菌γδリゾルベース認識配列(配列番号2)、を 含有する組換え核酸を増殖し、 (b) 増殖した核酸を細菌γδリゾルベースで処理し、そして (c) ヘルパーウイルスおよび前記のリゾルベース処理した組換え核酸を含有 する真核細胞を培養し、それにより分割された組換えDNAを複製させ、かつウ イルス粒子へと包膜させる、 ことを含んでなる方法。 16.組換えDNA分子を複製し、そのDNA分子をキャプシドで包み込んでAA V粒子とする方法であって、 (a) 図9に示した165塩基対の末端反復配列(配列番号1)および図10 に示した細菌のγδリゾルベース認識配列(配列番号2)を含有する組換え核酸 を、細菌のγδリゾルベース酵素で処理し、そして (b) ヘルパーウイルスおよび前記のリゾルベース処理した組換え核酸を含有 する真核細胞を培養し、それにより分割された組換え核酸を複製させ、かつAA V粒子へと包膜させる、 ことを含んでなる方法。 17.ヘルパーウイルスがアデノウイルスである、請求項16に記載の方法。 18.ヘルパーウイルスが単純ヘルペスウイルスである、請求項16に記載の方法。 19.細菌のγδリゾルベース認識配列を含有する組換え核酸が、 (a) 細菌のγδリゾルベース認識配列、 (b) プロモーター、 (c) プロモーターの制御下で転写される目的のDNA配列、および (d) 図9に示した165塩基対の逆方向末端反復配列(配列番号1)、 を含有するプラスミドベクターである、請求項16に記載の方法。 20.細菌のγδリゾルベース認識配列を含有する組換え核酸が、 (a) 細菌のγδリゾルベース認識配列、 (b) プロモーター、 (c) プロモーターの制御下で転写される目的のDNA配列、 (d) 図9に示した165塩基対の逆方向末端反復配列(配列番号1)、およ び (e) AAVのREPおよびCAPタンパク質をコードするDNAヌクレオチ ド配列、 を含有するプラスミドベクターである、請求項16に記載の方法。 21.請求項1または2に記載の核酸分子を含有する組換えDNA分子を用いて真 核細胞をトランスフェクションすることを含んでなる、真核細胞に遺伝情報を伝 達する方法。 22.宿主に遺伝情報を伝達する方法であって、 (a) 請求項1または2に記載の核酸分子を含有する組換えDNA分子をリポ ソーム内に保持させ、そして (b) そのリポソーム調製物を宿主に投与する、 ことを含んでなる方法。 23.AAVのREPタンパク質をコードする核酸分子を前記の組換えDNA分子 と一緒にリポソーム内に保持させる、請求項22に記載の方法。 24.AAVのREPタンパク質を前記の組換えDNA分子と一緒にリポソーム内 に保持させる、請求項22に記載の方法。 25.請求項8または9に記載の方法にしたがって調製された組換えアデノ随伴ウ イルスのストック。 26.組換えアデノ随伴ウイルスのストックを産生する宿主細胞であって、 (a)(i)ヘルパーウイルス、(ii)AAVのREPおよびCAPタンパク質を コードする組換え核酸、および(iii)目的のDNA配列および図9に示した16 5塩基対の逆方向末端反復配列(配列番号1)を含む組換え核酸、を含有する前 記の宿主細胞を培養し、それにより165塩基対の逆方向末端反復配列および目 的のDNA配列を含む前記核酸を複製させ、かつAAVビリオンへと組み立て、 そして (b) 産生されたビリオンを集める、 ことを含んでなる方法により組換えアデノ随伴ウイルスのストックを産生する 宿主細胞。 27.請求項1または2に記載の核酸分子を含有する組換えDNA分子を含んでな る真核細胞。
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US08/440,738 US5869305A (en) | 1992-12-04 | 1995-05-15 | Recombinant viral vector system |
US440,738 | 1995-05-15 | ||
PCT/US1996/006786 WO1996036364A1 (en) | 1995-05-15 | 1996-05-14 | Recombinant viral vector system |
Publications (2)
Publication Number | Publication Date |
---|---|
JPH11506318A true JPH11506318A (ja) | 1999-06-08 |
JP4063319B2 JP4063319B2 (ja) | 2008-03-19 |
Family
ID=23749972
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP53494696A Expired - Fee Related JP4063319B2 (ja) | 1995-05-15 | 1996-05-14 | 組換えウイルスベクター系 |
Country Status (10)
Country | Link |
---|---|
US (2) | US5869305A (ja) |
EP (1) | EP0828519B1 (ja) |
JP (1) | JP4063319B2 (ja) |
AT (1) | ATE411818T1 (ja) |
AU (1) | AU699973B2 (ja) |
CA (1) | CA2221292A1 (ja) |
DE (1) | DE69637723D1 (ja) |
IL (1) | IL118259A0 (ja) |
WO (1) | WO1996036364A1 (ja) |
ZA (1) | ZA963863B (ja) |
Cited By (2)
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---|---|---|---|---|
JPWO2018139637A1 (ja) * | 2017-01-30 | 2019-11-21 | 学校法人日本医科大学 | 核酸封入aav中空粒子 |
CN114269940A (zh) * | 2019-07-03 | 2022-04-01 | 学校法人日本医科大学 | 用于产生包封核酸的aav中空颗粒的方法 |
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IT1297074B1 (it) | 1997-11-21 | 1999-08-03 | Angeletti P Ist Richerche Bio | Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le |
US6783972B1 (en) | 1998-09-22 | 2004-08-31 | University Of Florida Research Foundation | Methods for large-scale production of recombinant AAV vectors |
US6759237B1 (en) * | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
US6491907B1 (en) * | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
DE19905501B4 (de) * | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
US20040043488A1 (en) * | 1999-02-17 | 2004-03-04 | University Of Pittsburgh Of The Commonwealth System Of Higher Education | Adeno-associated viral gene-transfer vector system |
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US7517887B2 (en) * | 2003-04-09 | 2009-04-14 | General Atomics | Reversible inhibitors of S-adenosyl-L-homocysteine hydrolase and uses thereof |
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Cited By (2)
Publication number | Priority date | Publication date | Assignee | Title |
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JPWO2018139637A1 (ja) * | 2017-01-30 | 2019-11-21 | 学校法人日本医科大学 | 核酸封入aav中空粒子 |
CN114269940A (zh) * | 2019-07-03 | 2022-04-01 | 学校法人日本医科大学 | 用于产生包封核酸的aav中空颗粒的方法 |
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ATE411818T1 (de) | 2008-11-15 |
EP0828519B1 (en) | 2008-10-22 |
JP4063319B2 (ja) | 2008-03-19 |
DE69637723D1 (de) | 2008-12-04 |
IL118259A0 (en) | 1996-09-12 |
EP0828519A4 (en) | 2001-11-28 |
EP0828519A1 (en) | 1998-03-18 |
US5869305A (en) | 1999-02-09 |
CA2221292A1 (en) | 1996-11-21 |
AU5857796A (en) | 1996-11-29 |
WO1996036364A1 (en) | 1996-11-21 |
US6057152A (en) | 2000-05-02 |
ZA963863B (en) | 1998-02-20 |
AU699973B2 (en) | 1998-12-17 |
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