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
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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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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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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 (enExample) 2022-07-25
JP7451497B2 true JP7451497B2 (ja) 2024-03-18

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US (1) US12195745B2 (enExample)
EP (1) EP3841109A4 (enExample)
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AU (1) AU2019325332B2 (enExample)
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IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & 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
WO2021205028A1 (en) 2020-04-09 2021-10-14 University Of Sheffield Gene therapy treatment
US20230295658A1 (en) * 2020-11-10 2023-09-21 The Children's Hospital Of Philadelphia Compositions and methods for in vivo gene transfer
TW202325840A (zh) * 2021-08-30 2023-07-01 美商艾維克羅姆公司 與人肝臟嵌合性組合的腺相關病毒受體（ａａｖｒ）剔除小鼠及彼之使用方法和產製
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
EP4762358A1 (en) 2023-11-30 2026-06-24 Cytomos Limited Cell selection
WO2025188755A1 (en) 2024-03-04 2025-09-12 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
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the 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
DE23193606T1 (de) * 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
WO2016065001A1 (en) * 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
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
EP3448874A4 (en) * 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
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HUE053747T2 (hu) * 2016-08-15 2021-07-28 Genzyme Corp Eljárások AAV kimutatására
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Patent Citations (4)

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

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