CN112823209A - 使用编码高度紧凑的多输入逻辑门的核酸载体治疗疾病的方法 - Google Patents

使用编码高度紧凑的多输入逻辑门的核酸载体治疗疾病的方法 Download PDF

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CN112823209A
CN112823209A CN201980066858.9A CN201980066858A CN112823209A CN 112823209 A CN112823209 A CN 112823209A CN 201980066858 A CN201980066858 A CN 201980066858A CN 112823209 A CN112823209 A CN 112823209A
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acid molecule
polynucleic acid
cassette
transactivator
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Y·本南森
B·安杰利奇
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ETH Zurich
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ETH Zurich
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201980066858.9A 2018-10-11 2019-10-10 使用编码高度紧凑的多输入逻辑门的核酸载体治疗疾病的方法 Pending CN112823209A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862744173P 2018-10-11 2018-10-11
US62/744,173 2018-10-11
PCT/IB2019/001100 WO2020074956A2 (en) 2018-10-11 2019-10-10 A method to treat disease using a nucleic acid vector encoding a highly compact multi-input logic gate

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CN112823209A true CN112823209A (zh) 2021-05-18

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US (1) US20210381001A1 (cg-RX-API-DMAC7.html)
EP (1) EP3864158A2 (cg-RX-API-DMAC7.html)
JP (2) JP2022513347A (cg-RX-API-DMAC7.html)
KR (1) KR20210075126A (cg-RX-API-DMAC7.html)
CN (1) CN112823209A (cg-RX-API-DMAC7.html)
AU (2) AU2019359505B2 (cg-RX-API-DMAC7.html)
CA (1) CA3113325A1 (cg-RX-API-DMAC7.html)
WO (1) WO2020074956A2 (cg-RX-API-DMAC7.html)

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AU2021256845A1 (en) * 2020-04-14 2022-11-24 Eidgenössische Technische Hochschule Zürich Cell classifier circuits and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106103705A (zh) * 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
WO2017064566A2 (en) * 2015-10-16 2017-04-20 Astrazeneca Ab Inducible modification of a cell genome

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7527942B2 (en) 2002-02-08 2009-05-05 The Regents Of The University Of California Transcription amplification system for molecular imaging
DK2002003T3 (en) 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
WO2008070448A2 (en) 2006-11-22 2008-06-12 Board Of Regents, The University Of Texas System Cancer-specific promoters
US9458509B2 (en) 2010-07-22 2016-10-04 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
JP6419082B2 (ja) 2012-12-13 2018-11-07 マサチューセッツ インスティテュート オブ テクノロジー リコンビナーゼに基づく論理/メモリシステム
US11030531B2 (en) 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106103705A (zh) * 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
WO2017064566A2 (en) * 2015-10-16 2017-04-20 Astrazeneca Ab Inducible modification of a cell genome

Non-Patent Citations (13)

* Cited by examiner, † Cited by third party
Title
AMENDOLA 等: ""A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector)"", 《THE AMERICAN SOCIETY OF GENE & CELL THERAPY》 *
AMENDOLA 等: ""A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector)"", 《THE AMERICAN SOCIETY OF GENE & CELL THERAPY》, vol. 21, no. 5, 1 May 2013 (2013-05-01), XP055630166, DOI: 10.1038/mt.2013.12 *
ANGELIC 等: ""Synthetic Biology Platform for Sensing and Integrating Endogenous Transcriptional Inputs in Mammalian Cells"", 《CELL REPORTS》 *
ANGELIC 等: ""Synthetic Biology Platform for Sensing and Integrating Endogenous Transcriptional Inputs in Mammalian Cells"", 《CELL REPORTS》, vol. 16, no. 9, 1 August 2016 (2016-08-01) *
ANGELICI 等: ""Synthetic Biology Platform for Sensing and Integrating Endogenous Transcriptional Inputs in Mammalian Cells"", 《CELL REPORTS》, vol. 16, 30 August 2016 (2016-08-30), pages 2528 - 2532 *
BARTOLOMEO ANGELICI: ""Design and Implementation of Transcription Factor Based Gene Classifier Circuits"", 《ETH ZURICH RESEARCH COLLECTION》 *
BARTOLOMEO ANGELICI: ""Design and Implementation of Transcription Factor Based Gene Classifier Circuits"", 《ETH ZURICH RESEARCH COLLECTION》, 1 January 2017 (2017-01-01), pages 54 - 63 *
HEINZ 等: ""Graded or threshold response of the tet-controlled gene expression: all depends on the concentration of the transactivator"", 《BMC BIOTECHNOLOGY》 *
HEINZ 等: ""Graded or threshold response of the tet-controlled gene expression: all depends on the concentration of the transactivator"", 《BMC BIOTECHNOLOGY》, vol. 13, no. 5, 22 January 2013 (2013-01-22), pages 10 *
SZYMANSKI 等: ""Development and Validation of a Robust and Versatile One-plasmid Regulated Gene Expression System"", 《THE AMERICAN SOCIETY OF GENE THERAPY》 *
SZYMANSKI 等: ""Development and Validation of a Robust and Versatile One-plasmid Regulated Gene Expression System"", 《THE AMERICAN SOCIETY OF GENE THERAPY》, vol. 15, no. 7, 1 July 2007 (2007-07-01), XP002516079, DOI: 10.1038/SJ.MT.6300171 *
VILABOA 等: ""Gene Switches for Oeliberate Regulation of Transgene Expression: Recent Advances in System Oevelopment and Uses"", 《JOURNAL OF GENETIC SYNOROMES & GENE THERAPY》 *
VILABOA 等: ""Gene Switches for Oeliberate Regulation of Transgene Expression: Recent Advances in System Oevelopment and Uses"", 《JOURNAL OF GENETIC SYNOROMES & GENE THERAPY》, vol. 2, no. 3, 1 January 2011 (2011-01-01) *

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US20210381001A1 (en) 2021-12-09
JP2025049296A (ja) 2025-04-03
JP2022513347A (ja) 2022-02-07
AU2019359505B2 (en) 2026-03-19
AU2026201687A1 (en) 2026-03-26
WO2020074956A3 (en) 2020-05-22
WO2020074956A2 (en) 2020-04-16
KR20210075126A (ko) 2021-06-22
EP3864158A2 (en) 2021-08-18
AU2019359505A1 (en) 2021-04-29
CA3113325A1 (en) 2020-04-16

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