JP2023542132A - Ｆｖｉｉｉ治療薬を発現させるための非ウイルス性ｄｎａベクター及びその使用 - Google Patents

Ｆｖｉｉｉ治療薬を発現させるための非ウイルス性ｄｎａベクター及びその使用 Download PDF

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JP2023542132A
JP2023542132A JP2023517284A JP2023517284A JP2023542132A JP 2023542132 A JP2023542132 A JP 2023542132A JP 2023517284 A JP2023517284 A JP 2023517284A JP 2023517284 A JP2023517284 A JP 2023517284A JP 2023542132 A JP2023542132 A JP 2023542132A
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seq
nucleic acid
acid sequence
cedna
cedna vector
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デブラ クラット，
ラッセル モンズ，
ルーク エス． ハム，
ナサニエル シルバー，
フィリップ サマヨア，
ダグラス アンソニー カー，
ジェシカ リン キーナン，
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ジェネレーション バイオ カンパニー
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JP2023517284A 2020-09-16 2021-09-16 Ｆｖｉｉｉ治療薬を発現させるための非ウイルス性ｄｎａベクター及びその使用 Pending JP2023542132A (ja)

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US202063079349P 2020-09-16 2020-09-16
US63/079,349 2020-09-16
US202063132838P 2020-12-31 2020-12-31
US63/132,838 2020-12-31
PCT/US2021/050715 WO2022061014A1 (en) 2020-09-16 2021-09-16 Non-viral dna vectors and uses thereof for expressing fviii therapeutics

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JP2023542132A true JP2023542132A (ja) 2023-10-05
JP2023542132A5 JP2023542132A5 (https=) 2024-09-26

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US (1) US20230383311A1 (https=)
EP (1) EP4214315A4 (https=)
JP (1) JP2023542132A (https=)
KR (1) KR20230066453A (https=)
AU (1) AU2021342503A1 (https=)
CA (1) CA3191743A1 (https=)
IL (1) IL301197A (https=)
MX (1) MX2023003021A (https=)
WO (1) WO2022061014A1 (https=)

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TW202541849A (zh) * 2023-12-14 2025-11-01 美商聲納席拉公司 用於經由超音波改良核酸遞送之方法及系統

Citations (4)

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JP2009542225A (ja) * 2006-06-30 2009-12-03 ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・ミシガン 組換え法によりｆｖｉｉｉタンパク質を生産する方法
JP2017521049A (ja) * 2014-05-21 2017-08-03 インペリアル イノベーションズ リミテッド レンチウイルスベクター
JP2019506889A (ja) * 2016-03-03 2019-03-14 ユニバーシティ オブ マサチューセッツ 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖ｄｎａ
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED

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GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
AU2013336601B2 (en) * 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
PL231230B1 (pl) * 2015-03-11 2019-02-28 Inst Biochemii I Biofizyki Polskiej Akademii Nauk Czynnik zwiększający efektywność szczepionki DNA skierowanej przeciw wirusowi, preparat plazmidowy DNA, szczepionka DNA, sposób otrzymywania zmodyfikowanego wektora ekspresyjnego oraz zastosowanie sekwencji nukleotydowej rozpoznawanej przez czynnik NF kappa B
NZ762100A (en) * 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
MA52116A (fr) * 2018-03-02 2021-01-06 Generation Bio Co Vecteurs d'adn à extrémité fermée (cedna) pour l'insertion de transgènes au niveau de havres génomiques sécuritaires (gsh) dans des génomes humains et murins
SG11202108357PA (en) * 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression

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Publication number Priority date Publication date Assignee Title
JP2009542225A (ja) * 2006-06-30 2009-12-03 ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・ミシガン 組換え法によりｆｖｉｉｉタンパク質を生産する方法
JP2017521049A (ja) * 2014-05-21 2017-08-03 インペリアル イノベーションズ リミテッド レンチウイルスベクター
JP2019506889A (ja) * 2016-03-03 2019-03-14 ユニバーシティ オブ マサチューセッツ 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖ｄｎａ
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED

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CA3191743A1 (en) 2022-03-24
IL301197A (en) 2023-05-01
KR20230066453A (ko) 2023-05-15
US20230383311A1 (en) 2023-11-30
EP4214315A4 (en) 2025-07-23
MX2023003021A (es) 2023-05-24
WO2022061014A1 (en) 2022-03-24
AU2021342503A1 (en) 2023-04-06
EP4214315A1 (en) 2023-07-26

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