JP2024516655A - 原発性高シュウ酸尿症１型の治療のためのｉｎ ｖｉｖｏレンチウイルス遺伝子療法 - Google Patents

原発性高シュウ酸尿症１型の治療のためのｉｎ ｖｉｖｏレンチウイルス遺伝子療法 Download PDF

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JP2024516655A
JP2024516655A JP2023565998A JP2023565998A JP2024516655A JP 2024516655 A JP2024516655 A JP 2024516655A JP 2023565998 A JP2023565998 A JP 2023565998A JP 2023565998 A JP2023565998 A JP 2023565998A JP 2024516655 A JP2024516655 A JP 2024516655A
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lentiviral vector
sequence
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mice
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サンス ホセ カルロス セゴヴィア
ブラヴォ マリア ガルシア
ヴィセンテ アンドレア モリノス
トッラルバ アイーダ ガルシア
ロメロ ヴィルヒニア ニエト
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セントロ デ インベスティガシオネス エネルゲティカス メディオ アムビエンタレス イ テクノロジカス オー．エー．，エム．ピー．
コンソルシオ セントロ デ インベスティガシオン ビオメディカ エン レッド （セイベエエレ）
フンダシオン インスティチュート デ インベスティガシオン サニタリア フンダシオン ヒメネス ディアス
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JP2023565998A 2021-04-26 2022-04-26 原発性高シュウ酸尿症１型の治療のためのｉｎ ｖｉｖｏレンチウイルス遺伝子療法 Pending JP2024516655A (ja)

Applications Claiming Priority (3)

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EP21382363.6 2021-04-26
EP21382363.6A EP4083217A1 (en) 2021-04-26 2021-04-26 In vivo lentiviral gene therapy for the treatment of primary hyperoxaluria type 1
PCT/EP2022/061107 WO2022229223A1 (en) 2021-04-26 2022-04-26 In vivo lentiviral gene therapy for the treatment of primary hyperoxaluria type 1

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JP2024516655A true JP2024516655A (ja) 2024-04-16
JP2024516655A5 JP2024516655A5 (enExample) 2025-05-09

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JP2023565998A Pending JP2024516655A (ja) 2021-04-26 2022-04-26 原発性高シュウ酸尿症１型の治療のためのｉｎ ｖｉｖｏレンチウイルス遺伝子療法

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EP (2) EP4083217A1 (enExample)
JP (1) JP2024516655A (enExample)
KR (1) KR20240004627A (enExample)
CN (1) CN117561336A (enExample)
AU (1) AU2022266057A1 (enExample)
CA (1) CA3218031A1 (enExample)
MX (1) MX2023012684A (enExample)
WO (1) WO2022229223A1 (enExample)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012516325A (ja) * 2009-01-30 2012-07-19 アムステルダム モレキュラー セラピューティクス （エーエムティー） アイピー ビー．ブイ． アラニン−グリオキシル酸アミノトランスフェラーゼ治療薬
US20150118187A1 (en) * 2013-10-24 2015-04-30 Medgenics Medical Israel Ltd. Micro-organs providing sustained delivery of a therapeutic polypeptide and methods of use thereof
US20170260516A1 (en) * 2014-06-30 2017-09-14 Biogen Ma Inc. Optimized factor ix gene

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11976293B2 (en) * 2016-12-05 2024-05-07 The Regents Of The University Of California Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies
US20210038744A1 (en) * 2018-02-01 2021-02-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor viii

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012516325A (ja) * 2009-01-30 2012-07-19 アムステルダム モレキュラー セラピューティクス （エーエムティー） アイピー ビー．ブイ． アラニン−グリオキシル酸アミノトランスフェラーゼ治療薬
US20150118187A1 (en) * 2013-10-24 2015-04-30 Medgenics Medical Israel Ltd. Micro-organs providing sustained delivery of a therapeutic polypeptide and methods of use thereof
US20170260516A1 (en) * 2014-06-30 2017-09-14 Biogen Ma Inc. Optimized factor ix gene

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BRIAN D. BROWN ET AL.: ""A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice"", BLOOD, vol. 110, no. 13, JPN6026009801, 15 December 2007 (2007-12-15), pages 4144 - 4152, ISSN: 0005818164 *
CAROLINA PETRILLO ET AL.: ""Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Ed", CELL STEM CELL, vol. 23, no. 6, JPN6026009805, December 2018 (2018-12-01), pages 820 - 832, ISSN: 0005818167 *
JUDITH AGUDO ET AL.: ""A TLR and Non-TLR Mediated Innate Response to Lentiviruses Restricts Hepatocyte Entry and Can be A", MOLECULAR THERAPY, vol. 20, no. 12, JPN6026009803, December 2012 (2012-12-01), pages 2257 - 2267, ISSN: 0005818166 *
NOEL MESA-TORRES ET AL.: ""The consensus-based approach for gene/enzyme replacement therapies and crystallization strategies:", BIOCHEMICAL JOURNAL, vol. 462, no. 3, JPN6026009802, 22 August 2014 (2014-08-22), pages 453 - 463, ISSN: 0005818165 *

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EP4330415A1 (en) 2024-03-06
WO2022229223A1 (en) 2022-11-03
EP4083217A1 (en) 2022-11-02
CA3218031A1 (en) 2022-11-03
CN117561336A (zh) 2024-02-13
KR20240004627A (ko) 2024-01-11
MX2023012684A (es) 2024-02-15
AU2022266057A1 (en) 2023-11-16

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