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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    • A—HUMAN NECESSITIES
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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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    • 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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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)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
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 (https=) 2006-03-02

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US (1) US20050106558A1 (https=)
EP (2) EP1456383B1 (https=)
JP (1) JP2005512569A (https=)
AU (2) AU2002352261B2 (https=)
CA (1) CA2468882C (https=)
ES (1) ES2467156T3 (https=)
WO (1) WO2003054197A2 (https=)

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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
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

Families Citing this family (34)

* Cited by examiner, † Cited by third party
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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
ES2535877T3 (es) * 2006-06-19 2015-05-18 Asklepios Biopharmaceutical, Inc. Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica
ES2602610T3 (es) * 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
EP2098536A1 (en) * 2008-03-05 2009-09-09 4-Antibody AG Isolation and identification of antigen- or ligand-specific binding proteins
WO2010093784A2 (en) 2009-02-11 2010-08-19 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
AU2011317846B2 (en) 2010-10-21 2015-07-09 Deutsches Krebsforschungszentrum Retargeting of rat parvovirus H-1PV to cancer cells through genetic engineering of its capsid
CN105755044A (zh) 2011-04-22 2016-07-13 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及其使用方法
WO2013037961A1 (en) 2011-09-15 2013-03-21 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
EP3003391B1 (en) 2013-05-31 2021-09-22 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
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
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
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
JP2020534788A (ja) 2017-08-28 2020-12-03 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 アデノ随伴ウイルスカプシド変異体及びその使用方法
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
WO2019084015A1 (en) * 2017-10-23 2019-05-02 Daniel Schmidt PROGRAMMABLE SET OF VIRUS COMPOSITES FOR ADMINISTRATION OF GENES TARGETING A RECEPTOR
CN111770999A (zh) 2017-11-27 2020-10-13 4D分子治疗有限公司 腺相关病毒变体衣壳和用于抑制血管生成的应用
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
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
JP2022530457A (ja) 2019-04-26 2022-06-29 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作ａａｖ
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
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Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6031071A (en) * 1996-01-24 2000-02-29 Biophage, Inc. Methods of generating novel peptides
EP0927044A4 (en) * 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET VIRAL VECTORS
EP1181382B1 (en) * 1999-06-01 2005-03-23 The University of Washington Recombinant adenoviral vectors expressing chimeric fiber proteins for cell specific infection and genome integration
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods

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

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