KR20200124250A - 폐쇄-말단 dna (cedna) 벡터를 사용한 이식유전자의 제어된 발현 - Google Patents

폐쇄-말단 dna (cedna) 벡터를 사용한 이식유전자의 제어된 발현 Download PDF

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KR20200124250A
KR20200124250A KR1020207026566A KR20207026566A KR20200124250A KR 20200124250 A KR20200124250 A KR 20200124250A KR 1020207026566 A KR1020207026566 A KR 1020207026566A KR 20207026566 A KR20207026566 A KR 20207026566A KR 20200124250 A KR20200124250 A KR 20200124250A
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expression
transgene
itr
cedna
level
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더글라스 에이. 커
매튜 쥐. 스탠턴
매트 치오코
마크 디. 안젤리노
로버트 엠. 코틴
필립 사마요아
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제너레이션 바이오 컴퍼니
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020207026566A 2018-02-22 2019-02-21 폐쇄-말단 dna (cedna) 벡터를 사용한 이식유전자의 제어된 발현 Ceased KR20200124250A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862633757P 2018-02-22 2018-02-22
US201862633795P 2018-02-22 2018-02-22
US201862633882P 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

Publications (1)

Publication Number Publication Date
KR20200124250A true KR20200124250A (ko) 2020-11-02

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KR1020207026566A Ceased KR20200124250A (ko) 2018-02-22 2019-02-21 폐쇄-말단 dna (cedna) 벡터를 사용한 이식유전자의 제어된 발현

Country Status (14)

Country Link
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=)
SG (1) SG11202007621TA (https=)
WO (1) WO2019165050A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
CN113316640A (zh) * 2018-11-09 2021-08-27 世代生物公司 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna)
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
AU2021244559A1 (en) * 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
MX2022011805A (es) * 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
JP2023524010A (ja) * 2020-04-28 2023-06-08 プレジデント アンド フェローズ オブ ハーバード カレッジ 高効率遺伝子送達系
US20230265440A1 (en) * 2020-06-26 2023-08-24 The Board Of Trustees Of The Leland Stanford Junior University Targeting the human ccr5 locus as a safe harbor for the expression of therapeutic proteins
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
US20220090130A1 (en) * 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
US20240026374A1 (en) * 2020-09-16 2024-01-25 Generation Bio Co. Closed-ended dna vectors and uses thereof 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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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003087334A2 (en) * 2002-04-09 2003-10-23 Cornell Research Foundation, Inc. Use of aav integration efficiency element for mediating site-specific integration of a transcription unit
LT2816118T (lt) * 2005-05-31 2018-12-10 The Regents Of The University Of Colorado, A Body Corporate Genų pristatymo būdai
JP2010538675A (ja) * 2007-09-19 2010-12-16 アムステルダム モレキュラー セラピューティクス ビー．ブイ． タンパク質産生の改善のためのａａｖ複製機構の使用
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
AU2015311704B2 (en) * 2014-09-07 2021-12-09 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
EP3889260A1 (en) * 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2017152149A1 (en) * 2016-03-03 2017-09-08 University Of Massachusetts Closed-ended linear duplex dna for non-viral gene transfer

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Publication number Publication date
JP2024028931A (ja) 2024-03-05
BR112020017060A2 (pt) 2020-12-15
PH12020551235A1 (en) 2021-04-19
WO2019165050A1 (en) 2019-08-29
MA51915A (fr) 2020-12-30
EP3755803A1 (en) 2020-12-30
CA3092059A1 (en) 2019-08-29
MX2020008676A (es) 2020-09-25
US20220175970A1 (en) 2022-06-09
CN111886343A (zh) 2020-11-03
JP2021513999A (ja) 2021-06-03
SG11202007621TA (en) 2020-09-29
EP3755803A4 (en) 2022-01-19
IL276658A (en) 2020-09-30
RU2020131041A (ru) 2022-03-22
AU2019225937A1 (en) 2020-08-13

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