JP4938189B2 - 修飾されたクロマトグラフィー特性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用 - Google Patents

修飾されたクロマトグラフィー特性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用 Download PDF

Info

Publication number
JP4938189B2
JP4938189B2 JP2001511201A JP2001511201A JP4938189B2 JP 4938189 B2 JP4938189 B2 JP 4938189B2 JP 2001511201 A JP2001511201 A JP 2001511201A JP 2001511201 A JP2001511201 A JP 2001511201A JP 4938189 B2 JP4938189 B2 JP 4938189B2
Authority
JP
Japan
Prior art keywords
aav
structural protein
antibody
mutation
receptor
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.)
Expired - Fee Related
Application number
JP2001511201A
Other languages
English (en)
Japanese (ja)
Other versions
JP2003505033A5 (enExample
JP2003505033A (ja
Inventor
ハレク，ミハエル
ジロ，アンヌ
リート，マルティーン
ストーラ，クリストフ・ゲールハルト
メービウス，ウルリヒ
Original Assignee
メディジーン・アクチェンゲゼルシャフト
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by メディジーン・アクチェンゲゼルシャフト filed Critical メディジーン・アクチェンゲゼルシャフト
Publication of JP2003505033A publication Critical patent/JP2003505033A/ja
Publication of JP2003505033A5 publication Critical patent/JP2003505033A5/ja
Application granted granted Critical
Publication of JP4938189B2 publication Critical patent/JP4938189B2/ja
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Images

Classifications

    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2001511201A 1999-07-19 2000-07-18 修飾されたクロマトグラフィー特性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用 Expired - Fee Related JP4938189B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DE19933719A DE19933719A1 (de) 1999-07-19 1999-07-19 Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
DE19933719.5 1999-07-19
PCT/EP2000/006861 WO2001005991A1 (de) 1999-07-19 2000-07-18 Strukturprotein von adeno-assoziiertem virus mit veränderten chromatographischen eigenschaften, seine herstellung und verwendung

Publications (3)

Publication Number Publication Date
JP2003505033A JP2003505033A (ja) 2003-02-12
JP2003505033A5 JP2003505033A5 (enExample) 2007-09-06
JP4938189B2 true JP4938189B2 (ja) 2012-05-23

Family

ID=7915238

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2001511201A Expired - Fee Related JP4938189B2 (ja) 1999-07-19 2000-07-18 修飾されたクロマトグラフィー特性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用

Country Status (9)

Country Link
US (2) US7285381B1 (enExample)
EP (1) EP1198580B1 (enExample)
JP (1) JP4938189B2 (enExample)
AT (1) ATE328101T1 (enExample)
AU (1) AU777885B2 (enExample)
CA (1) CA2379564C (enExample)
DE (2) DE19933719A1 (enExample)
ES (1) ES2263481T3 (enExample)
WO (1) WO2001005991A1 (enExample)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US8313908B2 (en) 2000-02-23 2012-11-20 The Trustees Of The University Of Pennsylvania Regulation of stem cell gene production with specific and selective electric and electromagnetic fields
US7374916B2 (en) 2000-02-23 2008-05-20 The Trustees Of The University Of Pennsylvania Regulation of aggrecan gene expression using specific and selective electrical and electromagnetic signals
US7022506B2 (en) 2000-02-23 2006-04-04 The Trustees Of The University Of Pennsylvania Method and device for treating osteoarthritis, cartilage disease, defects and injuries in the human knee
US7429471B2 (en) 2000-02-23 2008-09-30 The Trustees Of The University Of Pennsylvania Regulation of matrix metalloproteinase gene expression using specific and selective electrical and electromagnetic signals
US7465566B2 (en) 2000-02-23 2008-12-16 The Trustees Of The University Of Pennsylvania Regulation of genes via application of specific and selective electrical and electromagnetic signals
US7465546B2 (en) 2000-02-23 2008-12-16 The Trustees Of The University Of Pennsylvania Regulation of transforming growth factor-beta (TGF-β) gene expression in living cells via the application of specific and selective electric and electromagnetic fields
US7981611B2 (en) 2000-02-23 2011-07-19 The Trustees Of The University Of Pennsylvania Regulation of fibroblastic growth factor-2 (FGF-2) gene expression in living cells with the application of specific and selective electric and electromagnetic fields
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1918285B (zh) 2004-01-12 2013-09-25 宾夕法尼亚大学理事会 通过施加由特异性和选择性的电以及电磁信号产生的场上调骨细胞中骨形态发生蛋白(bmp)基因表达的系统以及方法
US7893985B1 (en) 2004-03-15 2011-02-22 Grandeye Ltd. Wide angle electronic camera with improved peripheral vision
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2158211B1 (en) 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CA2754335C (en) 2009-03-04 2019-12-24 Medigene Ag Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essentially consisting of vp3
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
US9821043B2 (en) 2011-09-15 2017-11-21 Medigene Ag Anti-HER2 vaccine based upon AAV derived multimeric structures
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
SI3645551T1 (sl) * 2017-06-27 2024-06-28 Regeneron Pharmaceuticals, Inc. S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice
KR20250023600A (ko) 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH06507383A (ja) * 1991-01-25 1994-08-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ラミニンのａ鎖のドメイン６のポリペプチド
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5837520A (en) * 1995-03-07 1998-11-17 Canji, Inc. Method of purification of viral vectors
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
WO1997038723A1 (en) * 1996-04-16 1997-10-23 Immusol Incorporated Targeted viral vectors
US20010031463A1 (en) 1997-09-02 2001-10-18 Jurgen Kleinschmidt Method for purifying and concentrating AAV-2 and antigen portions thereof
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
EP1135468B1 (en) * 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods

Also Published As

Publication number Publication date
CA2379564A1 (en) 2001-01-25
US20080241906A1 (en) 2008-10-02
EP1198580B1 (de) 2006-05-31
WO2001005991A1 (de) 2001-01-25
CA2379564C (en) 2012-03-20
EP1198580A1 (de) 2002-04-24
ES2263481T3 (es) 2006-12-16
US20100105123A2 (en) 2010-04-29
JP2003505033A (ja) 2003-02-12
US7285381B1 (en) 2007-10-23
DE19933719A1 (de) 2001-01-25
DE50012863D1 (de) 2006-07-06
US20090098634A2 (en) 2009-04-16
AU777885B2 (en) 2004-11-04
AU6435400A (en) 2001-02-05
ATE328101T1 (de) 2006-06-15
US20090246854A2 (en) 2009-10-01

Similar Documents

Publication Publication Date Title
US7285381B1 (en) Scleroprotein of an adeno-associated virus with modified chromatographic properties, the production thereof and use of the same
US7314912B1 (en) AAv scleroprotein, production and use thereof
JP4652570B2 (ja) Ａａｖの構造タンパク質、その製造及び用途
US7968340B2 (en) Structural protein of adeno-associated virus with modified antigenicity, its production and its use
Rabinowitz et al. Insertional mutagenesis of AAV2 capsid and the production of recombinant virus
AU2016261454B2 (en) Capsid
US7172893B2 (en) Virus vectors and methods of making and administering the same
CA2329060C (en) Aav5 vector and uses thereof
US20030215422A1 (en) Aav4 vector and uses thereof
JP2003505033A5 (enExample)
Zhang et al. Addition of six-His-tagged peptide to the C terminus of adeno-associated virus VP3 does not affect viral tropism or production
CN120641755A (zh) 用于检测抗aav颗粒抗体的方法
AU771545B2 (en) AAV4 vector and uses thereof
Bhrigu Replication of adeno-associated virus in murine fibroblasts with mouse adenovirus provided helper functions
Chen Isolation of infectious Rep/adeno-associated virus complex

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20070718

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20070718

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20100617

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20100917

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20100928

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20101217

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20101220

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20110531

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110830

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20111011

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120106

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20120130

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20120223

FPAY Renewal fee payment (event date is renewal date of database)

Free format text: PAYMENT UNTIL: 20150302

Year of fee payment: 3

R150 Certificate of patent or registration of utility model

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees