JP2021513999A - 閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現 - Google Patents

閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現 Download PDF

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JP2021513999A
JP2021513999A JP2020544445A JP2020544445A JP2021513999A JP 2021513999 A JP2021513999 A JP 2021513999A JP 2020544445 A JP2020544445 A JP 2020544445A JP 2020544445 A JP2020544445 A JP 2020544445A JP 2021513999 A JP2021513999 A JP 2021513999A
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transgene
expression
itr
dose
cedna
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ダグラス エー． カー，
ダグラス エー． カー，
マシュー ジー． スタントン，
マシュー ジー． スタントン，
マット チオッコ，
マット チオッコ，
マーク ディー． アンジェリーノ，
マーク ディー． アンジェリーノ，
ロバート エム． コーティン，
ロバート エム． コーティン，
フィリップ サマヨア，
フィリップ サマヨア，
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ジェネレーション バイオ カンパニー
ジェネレーション バイオ カンパニー
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Publication of JP2021513999A publication Critical patent/JP2021513999A/ja
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JP2020544445A 2018-02-22 2019-02-21 閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現 Pending JP2021513999A (ja)

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Application Number Priority Date Filing Date Title
JP2023207938A JP2024028931A (ja) 2018-02-22 2023-12-08 閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862633882P 2018-02-22 2018-02-22
US201862633795P 2018-02-22 2018-02-22
US201862633757P 2018-02-22 2018-02-22
US62/633,882 2018-02-22
US62/633,795 2018-02-22
US62/633,757 2018-02-22
US201862746762P 2018-10-17 2018-10-17
US62/746,762 2018-10-17
PCT/US2019/018927 WO2019165050A1 (en) 2018-02-22 2019-02-21 Controlled expression of transgenes using close-ended dna (cedna) vectors

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JP2023207938A Division JP2024028931A (ja) 2018-02-22 2023-12-08 閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現

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JP2021513999A true JP2021513999A (ja) 2021-06-03
JP2021513999A5 JP2021513999A5 (https=) 2022-03-01

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JP2023207938A Pending JP2024028931A (ja) 2018-02-22 2023-12-08 閉端ｄｎａ（ｃｅｄｎａ）ベクターを使用した導入遺伝子の制御された発現

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US (1) US20220175970A1 (https=)
EP (1) EP3755803A4 (https=)
JP (2) JP2021513999A (https=)
KR (1) KR20200124250A (https=)
CN (1) CN111886343A (https=)
AU (1) AU2019225937A1 (https=)
BR (1) BR112020017060A2 (https=)
CA (1) CA3092059A1 (https=)
IL (1) IL276658A (https=)
MA (1) MA51915A (https=)
MX (1) MX2020008676A (https=)
PH (1) PH12020551235A1 (https=)
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Cited By (1)

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Publication number Priority date Publication date Assignee Title
JP2022506771A (ja) * 2018-11-09 2022-01-17 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）

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NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
JP2023520763A (ja) * 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー ゴーシェ治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
US20230138409A1 (en) * 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
EP4142758A4 (en) * 2020-04-28 2025-02-26 President and Fellows of Harvard College High efficiency gene delivery system
IL299422A (en) * 2020-06-26 2023-02-01 Univ Leland Stanford Junior Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
WO2022046665A1 (en) * 2020-08-23 2022-03-03 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
EP4214309A4 (en) * 2020-09-16 2025-07-16 Generation Bio Co CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules
CN121662213B (zh) * 2026-02-06 2026-04-21 湖南博极生命科技有限公司 基于注意力权重追溯的中药活性成分可解释性分析方法及系统

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Publication number Priority date Publication date Assignee Title
JP2022506771A (ja) * 2018-11-09 2022-01-17 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）
JP7590963B2 (ja) 2018-11-09 2024-11-27 ジェネレーション バイオ カンパニー 対称的な修飾型逆位末端反復配列を含む修飾型閉端ｄｎａ（ｃｅｄｎａ）

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CA3092059A1 (en) 2019-08-29
CN111886343A (zh) 2020-11-03
RU2020131041A (ru) 2022-03-22
WO2019165050A1 (en) 2019-08-29
BR112020017060A2 (pt) 2020-12-15
IL276658A (en) 2020-09-30
US20220175970A1 (en) 2022-06-09
PH12020551235A1 (en) 2021-04-19
AU2019225937A1 (en) 2020-08-13
EP3755803A1 (en) 2020-12-30
MX2020008676A (es) 2020-09-25
EP3755803A4 (en) 2022-01-19
MA51915A (fr) 2020-12-30
JP2024028931A (ja) 2024-03-05
SG11202007621TA (en) 2020-09-29
KR20200124250A (ko) 2020-11-02

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