CN111867617A - 用于神经营养因子的诱导型表达的组合物和方法 - Google Patents

用于神经营养因子的诱导型表达的组合物和方法 Download PDF

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CN111867617A
CN111867617A CN201980019683.6A CN201980019683A CN111867617A CN 111867617 A CN111867617 A CN 111867617A CN 201980019683 A CN201980019683 A CN 201980019683A CN 111867617 A CN111867617 A CN 111867617A
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cells
gdnf
expression cassette
promoter
derived
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克莱夫·斯文森
约施华·布罗尼格
阿斯兰·阿赫塔
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Cedars Sinai Medical Center
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CN201980019683.6A 2018-03-16 2019-03-15 用于神经营养因子的诱导型表达的组合物和方法 Pending CN111867617A (zh)

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US201862644332P 2018-03-16 2018-03-16
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PCT/US2019/022595 WO2019178550A1 (en) 2018-03-16 2019-03-15 Methods and compositions for inducible expression of neurotrophic factors

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Cited By (3)

* 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
WO2025002240A1 (en) * 2023-06-29 2025-01-02 The University Of Hong Kong Method for controllable transcription in liver or liver cancer
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 (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190018000A1 (en) 2016-01-12 2019-01-17 Cedars-Sinai Medical Center A method of non destructive monitoring of biological processes in microfluidic tissue culture systems
WO2018140647A1 (en) 2017-01-25 2018-08-02 Cedars-Sinai Medical Center In vitro induction of mammary-like differentiation from human pluripotent stem cells
US11767513B2 (en) 2017-03-14 2023-09-26 Cedars-Sinai Medical Center Neuromuscular junction
US11414648B2 (en) 2017-03-24 2022-08-16 Cedars-Sinai Medical Center Methods and compositions for production of fallopian tube epithelium
EP4700387A2 (en) 2018-03-23 2026-02-25 Cedars-Sinai Medical Center Methods of use of islet cells
EP3775161A4 (en) 2018-04-06 2022-04-06 Cedars-Sinai Medical Center NEURODEGENERATIVE DISEASE MODELS DERIVED FROM HUMAN PLURIPOTENTIC STEM CELLS ON A MICROFLUIDIC CHIP
US11981918B2 (en) 2018-04-06 2024-05-14 Cedars-Sinai Medical Center Differentiation technique to generate dopaminergic neurons from induced pluripotent stem cells
EP3787613B1 (en) 2018-04-30 2026-03-11 Cedars-Sinai Medical Center Pkc activation for use in treating early onset sporadic parkinson's disease
WO2020084168A1 (en) * 2018-10-26 2020-04-30 Icm (Institut Du Cerveau Et De La Moelle Épinière) Tetracycline compound for treating drug-induced dyskinesia
AU2020370288B2 (en) * 2019-10-22 2026-02-12 Cedars-Sinai Medical Center Cortical neural progenitor cells from iPSCs
US20230047254A1 (en) * 2019-12-29 2023-02-16 Gloriana Therapeutics Mammalian Cells Secreting GDNF and Their Therapeutic Use
KR20220061403A (ko) * 2020-11-06 2022-05-13 (주) 에스바이오메딕스 다능성 줄기세포 유래 신경전구세포를 포함하는 퇴행성 뇌질환 치료용 약학적 조성물
GB202312699D0 (en) * 2023-08-18 2023-10-04 Cambridge Entpr Ltd Controllable expression in mammalian cells
WO2025091023A1 (en) * 2023-10-27 2025-05-01 Northwestern University Engineered cell transfected by all-in-one vector for therapeutic agents production and delivery and applications thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20120107284A1 (en) * 2006-06-30 2012-05-03 Elena Kozlova Stem cells for transplantation and methods for production thereof
CN102816793A (zh) * 2012-08-22 2012-12-12 陕西师范大学 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用
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

Family Cites Families (18)

* 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
US20030084468A1 (en) 2001-09-05 2003-05-01 Economides Aris N. Methods of expressing transgenes
GB2404382B (en) 2003-07-28 2008-01-30 Oxitec Ltd Pest control
US20100077495A1 (en) 2006-12-21 2010-03-25 Davis David P 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
US20120107938A1 (en) 2009-06-29 2012-05-03 University Of Basel Methods and kits for high efficiency engineering of conditional mouse alleles
WO2013155222A2 (en) * 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
JP2016508520A (ja) 2013-02-15 2016-03-22 インターナショナル ステム セル コーポレイション 神経変性疾患の治療のための、ヒト多能性幹細胞に由来する神経細胞の使用
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
WO2015143342A1 (en) 2014-03-21 2015-09-24 Cellular Dynamics International, Inc. Production of midbrain dopaminergic neurons and methods for the use thereof
CA2943296C (en) * 2014-03-21 2022-10-18 The Sydney Children's Hospitals Network (Randwick And Westmead) (Incorporating The Royal Alexandra Hospital For Children) Stable gene transfer to proliferating cells
EP3122370A1 (en) 2014-03-28 2017-02-01 Buck Institute for Research on Aging Methods and compositions for modulating the immune 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
JP6928604B2 (ja) * 2015-11-04 2021-09-01 フェイト セラピューティクス，インコーポレイテッド 万能性細胞のゲノム改変
US10221395B2 (en) 2016-06-16 2019-03-05 Cedars-Sinai Medical Center Efficient method for reprogramming blood to induced pluripotent stem cells
US12258571B2 (en) 2018-03-16 2025-03-25 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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20120107284A1 (en) * 2006-06-30 2012-05-03 Elena Kozlova Stem cells for transplantation and methods for production thereof
CN102816793A (zh) * 2012-08-22 2012-12-12 陕西师范大学 单载体双向启动的Tet-on诱导表达系统及其构建方法和应用
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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CHENSHA-SHA 等: "Intrastriatal GDNF gene transfer by inducible lentivirus vectors protects dopaminergic neurons in a rat model of parkinsonism", 《EXPERIMENTAL NEUROLOGY》, vol. 261, pages 88 *
HAKAN ALDSKOGIUS 等: "Regulation of Boundary Cap Neural Crest Stem Cell Differentiation After Transplantation", 《STEMCELLS》, vol. 27, no. 7, pages 1592 - 1603 *
WANG SHUYAN 等: "Inducible regulation of GDNF expression in human neural stem cells", 《SCI CHINA LIFE SCI》, vol. 56, no. 1, pages 32 - 39, XP035163654, DOI: 10.1007/s11427-012-4424-6 *
YANG WEI-HUA 等: "Regulated Expression of Lentivirus-Mediated GDNF in Human Bone Marrow-Derived Mesenchymal Stem Cells and Its Neuroprotection on Dopaminergic Cells In Vitro", 《PLOS ONE》, vol. 8, no. 5, pages 1 - 12 *

Cited By (3)

* 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
WO2025002240A1 (en) * 2023-06-29 2025-01-02 The University Of Hong Kong Method for controllable transcription in liver or liver cancer

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US20210024955A1 (en) 2021-01-28
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