CN111886343A - 使用闭合端dna（cedna）载体控制转基因的表达 - Google Patents

使用闭合端dna（cedna）载体控制转基因的表达 Download PDF

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CN111886343A
CN111886343A CN201980019861.5A CN201980019861A CN111886343A CN 111886343 A CN111886343 A CN 111886343A CN 201980019861 A CN201980019861 A CN 201980019861A CN 111886343 A CN111886343 A CN 111886343A
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expression
transgene
itr
cedna
itrs
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Chinese (zh)
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D·A·科尔
M·G·斯坦顿
M·基奥科
M·D·安格里诺
R·M·科廷
P·萨马约亚
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Generational Biology Co
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Generational Biology Co
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    • A—HUMAN NECESSITIES
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    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0069—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201980019861.5A 2018-02-22 2019-02-21 使用闭合端dna（cedna）载体控制转基因的表达 Pending CN111886343A (zh)

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

Publications (1)

Publication Number Publication Date
CN111886343A true CN111886343A (zh) 2020-11-03

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CN201980019861.5A Pending CN111886343A (zh) 2018-02-22 2019-02-21 使用闭合端dna（cedna）载体控制转基因的表达

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

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WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules

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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
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
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
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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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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WO2006130581A2 (en) * 2005-05-31 2006-12-07 Avigen, Inc. Methods for delivering genes
US20090208563A1 (en) * 2005-05-31 2009-08-20 Linda May Rothblum Watkins Method for delivering genes
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
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 Priority date Publication date Assignee Title
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules

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Publication number Publication date
CA3092059A1 (en) 2019-08-29
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
JP2021513999A (ja) 2021-06-03
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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