CN108884472A - 用于体内双重组酶介导的盒式交换(dRMCE)的系统和方法及其疾病模型 - Google Patents

用于体内双重组酶介导的盒式交换(dRMCE)的系统和方法及其疾病模型 Download PDF

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CN108884472A
CN108884472A CN201680084065.6A CN201680084065A CN108884472A CN 108884472 A CN108884472 A CN 108884472A CN 201680084065 A CN201680084065 A CN 201680084065A CN 108884472 A CN108884472 A CN 108884472A
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cells
rna
transgene
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expression
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约施华·布罗尼格
莫伊兹·丹尼尔鲍尔
金基范
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Cedars Sinai Medical Center
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CN201680084065.6A 2016-01-26 2016-12-30 用于体内双重组酶介导的盒式交换(dRMCE)的系统和方法及其疾病模型 Pending CN108884472A (zh)

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US201662287197P 2016-01-26 2016-01-26
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PCT/US2016/069442 WO2017131926A1 (en) 2016-01-26 2016-12-30 SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF

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US (2) US11466290B2 (enExample)
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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109943564A (zh) * 2019-03-28 2019-06-28 上海市中医老年医学研究所 Amh基因定点敲入2A-Cre的杂合子小鼠模型构建方法及其应用
CN114080451A (zh) * 2019-06-19 2022-02-22 豪夫迈·罗氏有限公司 通过使用Cre mRNA进行的靶向整合来产生蛋白质表达细胞的方法
CN114761035A (zh) * 2019-06-17 2022-07-15 西达-赛奈医疗中心 用于体内双重组酶介导的盒式交换(dRMCE)的系统和方法及其疾病模型
US11466290B2 (en) 2016-01-26 2022-10-11 Cedars-Sinai Medical Center Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof
US12258571B2 (en) 2018-03-16 2025-03-25 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109321599B (zh) * 2018-09-27 2023-08-22 同济大学 人多能干细胞中的谱系示踪系统的构建与应用
WO2023238843A1 (ja) 2022-06-06 2023-12-14 協和発酵バイオ株式会社 3'-シアリルラクトースの生産性が向上した微生物および3'-シアリルラクトースの製造方法

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030084468A1 (en) * 2001-09-05 2003-05-01 Economides Aris N. Methods of expressing transgenes

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7575924B2 (en) * 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
US20030228295A1 (en) 2002-04-25 2003-12-11 Svendsen Clive N. Use of human neural stem cells secreting GDNF for treatment of parkinson's and other neurodegenerative diseases
GB2404382B (en) 2003-07-28 2008-01-30 Oxitec Ltd Pest control
AU2007337263A1 (en) * 2006-12-21 2008-07-03 Genentech, Inc. Compositions and methods for the expression of nucleic acids
US8592211B2 (en) 2009-03-20 2013-11-26 The Rockefeller University Enhanced PiggyBac transposon and methods for transposon mutagenesis
WO2011001247A2 (en) * 2009-06-29 2011-01-06 University Of Basel Methods and kits for high efficiency engineering of conditional mouse alleles
US9528124B2 (en) * 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
EP2961277A4 (en) 2013-02-15 2016-12-28 Int Stem Cell Corp USE OF NERVOUS CELLS OBTAINED FROM HUMAN PLURIPOTENTIAL STEM CELLS FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
US10519421B2 (en) 2013-03-21 2019-12-31 Kyoto University Induction of motor neurons from pluripotent stem cells
WO2014204724A1 (en) * 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
AU2015234242B2 (en) 2014-03-21 2020-09-24 Children's Medical Research Institute Stable gene transfer to proliferating cells
DK3119881T3 (da) 2014-03-21 2023-04-03 Fujifilm Cellular Dynamics Inc Production of midbrain dopaminergic neurons and methods for the use thereof
US20170274048A1 (en) 2014-03-28 2017-09-28 Buck Institute For Research On Aging Methods and compositions for modulating the imune system
AU2015279552B2 (en) 2014-06-27 2021-01-21 Angiocrine Bioscience, Inc. Neural cells expressing adenovirus E4ORF1, and methods of making and using the same
CN108368520B (zh) 2015-11-04 2023-01-17 菲特治疗公司 多能细胞的基因组工程改造
WO2017131926A1 (en) 2016-01-26 2017-08-03 Cedars-Sinai Medical Center SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF
WO2019178550A1 (en) 2018-03-16 2019-09-19 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors
KR20220024527A (ko) 2019-06-17 2022-03-03 세다르스-신나이 메디칼 센터 생체내 이중 재조합효소-매개된 카세트 교환 (dRMCE)을 위한 시스템 및 방법 그리고 이들의 질환 모델

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030084468A1 (en) * 2001-09-05 2003-05-01 Economides Aris N. Methods of expressing transgenes

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BIN GUAN等: "Establishing isogenic inducible cell lines using founder reporter lines and recombinase-mediated cassette exchange", 《BIOTECHNIQUES》 *
MARCO OSTERWALDER等: "Dual RMCE for efficient re-engineering of mouse mutant alleles", 《NATURE METHODS》 *

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11466290B2 (en) 2016-01-26 2022-10-11 Cedars-Sinai Medical Center Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof
US12258571B2 (en) 2018-03-16 2025-03-25 Cedars-Sinai Medical Center Methods and compositions for inducible expression of neurotrophic factors
CN109943564A (zh) * 2019-03-28 2019-06-28 上海市中医老年医学研究所 Amh基因定点敲入2A-Cre的杂合子小鼠模型构建方法及其应用
CN109943564B (zh) * 2019-03-28 2022-03-15 上海市中医老年医学研究所 Amh基因定点敲入2A-Cre的杂合子小鼠模型构建方法及其应用
CN114761035A (zh) * 2019-06-17 2022-07-15 西达-赛奈医疗中心 用于体内双重组酶介导的盒式交换(dRMCE)的系统和方法及其疾病模型
CN114080451A (zh) * 2019-06-19 2022-02-22 豪夫迈·罗氏有限公司 通过使用Cre mRNA进行的靶向整合来产生蛋白质表达细胞的方法
CN114080451B (zh) * 2019-06-19 2024-03-22 豪夫迈·罗氏有限公司 通过使用Cre mRNA进行的靶向整合来产生蛋白质表达细胞的方法

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KR102811061B1 (ko) 2025-05-21
WO2017131926A1 (en) 2017-08-03
CA3012042A1 (en) 2017-08-03
AU2016389495B2 (en) 2023-03-30
AU2016389495A1 (en) 2018-08-16
JP2022091801A (ja) 2022-06-21
US20190390222A1 (en) 2019-12-26
EP3408396A1 (en) 2018-12-05
EP3408396A4 (en) 2019-10-09
JP2019511243A (ja) 2019-04-25
JP2024063065A (ja) 2024-05-10
KR20180101535A (ko) 2018-09-12
US11466290B2 (en) 2022-10-11

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