JP2005512569A - 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー - Google Patents

目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー Download PDF

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JP2005512569A
JP2005512569A JP2003554898A JP2003554898A JP2005512569A JP 2005512569 A JP2005512569 A JP 2005512569A JP 2003554898 A JP2003554898 A JP 2003554898A JP 2003554898 A JP2003554898 A JP 2003554898A JP 2005512569 A JP2005512569 A JP 2005512569A
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nucleic acid
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cell
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ペラーボ，ルーカ
ビュンイング，ヒルデガルト
エンスレ，ヨルグ
リート，マルティーン
ハレク，ミヒャエル
フートナー，ナジャ
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メディジーン・アクチェンゲゼルシャフト
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    • 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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    • A61P31/12—Antivirals
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    • A—HUMAN NECESSITIES
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immunology (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2003554898A 2001-12-21 2002-12-23 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー Pending JP2005512569A (ja)

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US34413101P 2001-12-21 2001-12-21
US36234902P 2002-03-07 2002-03-07
US40711602P 2002-08-30 2002-08-30
PCT/EP2002/014750 WO2003054197A2 (en) 2001-12-21 2002-12-23 A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism

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JP2005512569A true JP2005512569A (ja) 2005-05-12
JP2005512569A5 JP2005512569A5 (enExample) 2006-03-02

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US (1) US20050106558A1 (enExample)
EP (2) EP1456383B1 (enExample)
JP (1) JP2005512569A (enExample)
AU (2) AU2002352261B2 (enExample)
CA (1) CA2468882C (enExample)
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WO (1) WO2003054197A2 (enExample)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010530737A (ja) * 2007-05-31 2010-09-16 メディジーン アーゲー ワクチンとしての変異型パルボウイルス構造タンパク質
WO2014103957A1 (ja) * 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
JP2019518458A (ja) * 2016-05-13 2019-07-04 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ関連ウイルス変異キャプシドおよびその使用方法
JP2021521833A (ja) * 2018-04-27 2021-08-30 ウニベルジテート ハイデルベルク 筋疾患の治療のための改変ａａｖカプシドポリペプチド
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
JP2024056895A (ja) * 2016-08-18 2024-04-23 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 モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ－Ｃａｓゲノム編集

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DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
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
WO2007084773A2 (en) * 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
CN101511373B (zh) * 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
EP2158211B1 (en) * 2007-05-31 2016-08-10 Medigene AG Mutated structural protein of a parvovirus
EP2098536A1 (en) * 2008-03-05 2009-09-09 4-Antibody AG Isolation and identification of antigen- or ligand-specific binding proteins
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
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
JP5765790B2 (ja) * 2010-10-21 2015-08-19 ドイッチェス・クレープスフォルシュングスツェントルムＤｅｕｔｓｃｈｅｓ Ｋｒｅｂｓｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ラットパルボウイルスｈ−１ｐｖのそのカプシドの遺伝子操作による癌細胞への再標的化
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
EP2692731A1 (en) * 2012-07-31 2014-02-05 Paul-Ehrlich-Institut Bundesamt für Sera und Impfstoffe Recombinant Adeno-Associated virus (AAV) vector particles displaying high-affinity ligands for cell-type specific gene delivery
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
EP3221456B1 (en) * 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
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
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
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.
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
WO2019084015A1 (en) * 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
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EP3715358A1 (en) 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
CN114269926A (zh) 2019-04-26 2022-04-01 美商圣加莫治疗股份有限公司 Aav工程化
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WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
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Cited By (13)

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JP2010530737A (ja) * 2007-05-31 2010-09-16 メディジーン アーゲー ワクチンとしての変異型パルボウイルス構造タンパク質
JP2015121536A (ja) * 2007-05-31 2015-07-02 メディジーン アーゲー ワクチンとしての変異型パルボウイルス構造タンパク質
JP2018109636A (ja) * 2007-05-31 2018-07-12 メディジーン アーゲー ワクチンとしての変異型パルボウイルス構造タンパク質
WO2014103957A1 (ja) * 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
JPWO2014103957A1 (ja) * 2012-12-25 2017-01-12 タカラバイオ株式会社 Ａａｖ変異体
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11167041B2 (en) 2016-05-13 2021-11-09 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11179477B2 (en) 2016-05-13 2021-11-23 4D Molecular Therapeutics Inc. Method of using adeno-associated virus with variant capsid
JP2019518458A (ja) * 2016-05-13 2019-07-04 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ関連ウイルス変異キャプシドおよびその使用方法
US11419949B2 (en) 2016-05-13 2022-08-23 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
JP2024056895A (ja) * 2016-08-18 2024-04-23 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 モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ－Ｃａｓゲノム編集
JP2021521833A (ja) * 2018-04-27 2021-08-30 ウニベルジテート ハイデルベルク 筋疾患の治療のための改変ａａｖカプシドポリペプチド
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

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WO2003054197A3 (en) 2004-03-18
CA2468882A1 (en) 2003-07-03
AU2008202032A1 (en) 2009-11-26
CA2468882C (en) 2015-09-29
AU2002352261A1 (en) 2003-07-09
WO2003054197A2 (en) 2003-07-03
ES2467156T3 (es) 2014-06-12
EP1456383A2 (en) 2004-09-15
EP1456383B1 (en) 2014-03-12
EP2363487A3 (en) 2012-03-21
US20050106558A1 (en) 2005-05-19
EP2363487A2 (en) 2011-09-07
AU2008202032B2 (en) 2012-09-20
AU2002352261B2 (en) 2008-02-07

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