JP7451497B2 - アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 - Google Patents

アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 Download PDF

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JP7451497B2
JP7451497B2 JP2021509223A JP2021509223A JP7451497B2 JP 7451497 B2 JP7451497 B2 JP 7451497B2 JP 2021509223 A JP2021509223 A JP 2021509223A JP 2021509223 A JP2021509223 A JP 2021509223A JP 7451497 B2 JP7451497 B2 JP 7451497B2
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gpr108
cells
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エイチ． ヴァンデンベルゲ，ルク
ドゥデク，アマンダ
ザバレタ ラサルテ，ネレア
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マサチューセッツ アイ アンド イヤー インファーマリー
ザ スケペンス アイ リサーチ インスティテュート，インコーポレイテッド
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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JP2021509223A 2018-08-21 2019-08-21 アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 Active JP7451497B2 (ja)

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Application Number Priority Date Filing Date Title
US201862720859P 2018-08-21 2018-08-21
US62/720,859 2018-08-21
PCT/US2019/047546 WO2020041498A1 (en) 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses

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JP2021533795A JP2021533795A (ja) 2021-12-09
JP2021533795A5 JP2021533795A5 (https=) 2022-07-25
JP7451497B2 true JP7451497B2 (ja) 2024-03-18

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US (1) US12195745B2 (https=)
EP (1) EP3841109A4 (https=)
JP (1) JP7451497B2 (https=)
AU (1) AU2019325332B2 (https=)
WO (1) WO2020041498A1 (https=)

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CA3100006A1 (en) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
AU2021251429B2 (en) 2020-04-09 2025-12-18 University Of Sheffield Gene therapy treatment
EP4244365A4 (en) * 2020-11-10 2024-10-16 The Children's Hospital Of Philadelphia Compositions and methods for in vivo gene transfer
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
CN118207258A (zh) * 2024-03-12 2024-06-18 浙江恒驭生物科技有限公司 一种检测腺相关病毒转导滴度的敏感细胞系、构建方法及其应用
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids

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WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
WO2017083423A1 (en) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
WO2018022608A2 (en) 2016-07-26 2018-02-01 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins

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US6855314B1 (en) * 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
CN107073051B (zh) * 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
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US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
WO2017083423A1 (en) 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
WO2018022608A2 (en) 2016-07-26 2018-02-01 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins

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Publication number Publication date
AU2019325332A1 (en) 2021-03-18
JP2021533795A (ja) 2021-12-09
EP3841109A4 (en) 2022-09-21
US20210317478A1 (en) 2021-10-14
EP3841109A1 (en) 2021-06-30
US12195745B2 (en) 2025-01-14
AU2019325332B2 (en) 2025-11-13
WO2020041498A1 (en) 2020-02-27

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