KR20190053179A - 유전자 지우개들 - Google Patents

유전자 지우개들 Download PDF

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KR20190053179A
KR20190053179A KR1020197005575A KR20197005575A KR20190053179A KR 20190053179 A KR20190053179 A KR 20190053179A KR 1020197005575 A KR1020197005575 A KR 1020197005575A KR 20197005575 A KR20197005575 A KR 20197005575A KR 20190053179 A KR20190053179 A KR 20190053179A
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cells
cell
engineered
gene construct
nuclease
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Korean (ko)
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티모시 쿠안-타 루
레무스 웡
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센티 바이오사이언시스, 인코포레이티드
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KR1020197005575A 2016-07-26 2017-07-26 유전자 지우개들 Withdrawn KR20190053179A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662366755P 2016-07-26 2016-07-26
US62/366,755 2016-07-26
PCT/US2017/043931 WO2018022747A1 (en) 2016-07-26 2017-07-26 Genetic erasers

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KR20190053179A true KR20190053179A (ko) 2019-05-17

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KR1020197005575A Withdrawn KR20190053179A (ko) 2016-07-26 2017-07-26 유전자 지우개들
KR1020197005579A Ceased KR20190053180A (ko) 2016-07-26 2017-07-26 시공간적 조절자들

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US (2) US20190233844A1 (enExample)
EP (2) EP3491138A1 (enExample)
JP (2) JP2019527563A (enExample)
KR (2) KR20190053179A (enExample)
CN (2) CN110088285A (enExample)
AU (2) AU2017302589A1 (enExample)
CA (2) CA3031673A1 (enExample)
WO (2) WO2018022749A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
EP3235908A1 (en) 2016-04-21 2017-10-25 Ecole Normale Superieure De Lyon Methods for selectively modulating the activity of distinct subtypes of cells
DK3622079T3 (da) 2017-05-08 2025-11-17 Flagship Pioneering Innovations V Inc Sammensætninger til at gøre membranfusion lettere og anvendelser deraf
SG11202011015QA (en) * 2018-05-15 2020-12-30 Flagship Pioneering Innovations V Inc Fusosome compositions and uses thereof
EP3806888B1 (en) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
JP2022513040A (ja) 2018-11-14 2022-02-07 フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド コンパートメント特異的カーゴ送達のための組成物および方法
AU2019474716A1 (en) * 2019-11-19 2022-07-07 Protalix Ltd. Removal of constructs from transformed cells
GB202006462D0 (en) * 2020-05-04 2020-06-17 Mote Res Limited Modifying genomes with integrase
KR20240028975A (ko) 2021-04-08 2024-03-05 사나 바이오테크놀로지, 인크. Cd8-특이적 항체 구조체들 및 이의 조성물
WO2025006468A2 (en) 2023-06-26 2025-01-02 University Of Hawaii Evolved integrases and methods of using the same for genome editing
CN118813656B (zh) * 2024-06-19 2025-06-03 山东大学 一种大片段dna级联组装的方法、系统及应用

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19834430C2 (de) * 1998-07-30 2000-05-31 Harald Von Melchner Selbstdeletierende Vektoren für die Krebstherapie
US7189506B1 (en) * 1999-03-03 2007-03-13 Genelabs Technologies, Inc. DNA binding compound-mediated molecular switch system
US20060265771A1 (en) * 2005-05-17 2006-11-23 Lewis David L Monitoring microrna expression and function
SI2002003T1 (sl) * 2005-05-27 2016-05-31 Ospedale San Raffaele S.R.L. Genski vektor, ki vsebuje mi-RNA
AU2006254732A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
EP2069778A2 (en) * 2006-08-03 2009-06-17 Numira Biosciences, Inc. Methods and compositions for identifying biomarkers
US20090286242A1 (en) * 2007-12-10 2009-11-19 Cold Spring Harbor Laboratory MicroRNA Expression Profiling and Uses Thereof
US20110098346A1 (en) * 2008-05-19 2011-04-28 Agency For Science, Technology And Research Nucleic acid molecule and method of targeting gene expression to gliomas
US20100229254A1 (en) * 2009-03-04 2010-09-09 Kmita Marie Method for targeted cell ablation
US9272053B2 (en) * 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
WO2012056440A1 (en) * 2010-10-28 2012-05-03 Nanodoc Ltd. COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
CN102719556A (zh) * 2011-03-29 2012-10-10 北京五加和分子医学研究所有限公司 基于AAV载体的高通量miRNA活性检测方法及其应用
EP2691525A1 (en) * 2011-03-31 2014-02-05 Royal College of Surgeons in Ireland Treatment and prognosis of solid tumour cancers
CN102358901A (zh) * 2011-11-11 2012-02-22 天津托普泰克生物科技有限公司 Has-miR-520e的抗肿瘤用途
EP2684962A1 (en) * 2012-07-10 2014-01-15 Commissariat A L'energie Atomique Et Aux Energies Alternatives Vector for the selective silencing of a gene in astrocytes
WO2014093852A1 (en) * 2012-12-13 2014-06-19 Massachusetts Institute Of Technology Recombinase-based logic and memory systems
EP2948550A1 (en) * 2013-01-28 2015-12-02 Council of Scientific & Industrial Research METHOD FOR INHIBITING TUMOR GROWTH THROUGH RNA-INTERFERENCE USING LIPOSOMALLY ASSOCIATED CDC20 siRNA
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
RU2738421C2 (ru) * 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
JP2016088884A (ja) * 2014-11-04 2016-05-23 国立大学法人岐阜大学 腫瘍治療用組成物
AU2016279050A1 (en) 2015-06-19 2018-01-04 Massachusetts Institute Of Technology Tumor immunotherapy
US11174495B2 (en) * 2015-12-04 2021-11-16 Board Of Regents, The University Of Texas System Reporter system for detecting and targeting activated cells

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US20190169634A1 (en) 2019-06-06
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JP2019527563A (ja) 2019-10-03
WO2018022747A1 (en) 2018-02-01
EP3491137A1 (en) 2019-06-05
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