CN116064664A - 多重基因组编辑 - Google Patents

多重基因组编辑 Download PDF

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Publication number
CN116064664A
CN116064664A CN202211152246.1A CN202211152246A CN116064664A CN 116064664 A CN116064664 A CN 116064664A CN 202211152246 A CN202211152246 A CN 202211152246A CN 116064664 A CN116064664 A CN 116064664A
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cells
nucleic acid
cell
perv
rna
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Chinese (zh)
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G·M·丘奇
杨璐菡
M·格尔
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Harvard University
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Harvard University
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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CN202211152246.1A 2015-10-08 2016-10-07 多重基因组编辑 Pending CN116064664A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562239239P 2015-10-08 2015-10-08
US62/239,239 2015-10-08
PCT/US2016/055916 WO2017062723A1 (en) 2015-10-08 2016-10-07 Multiplexed genome editing
CN201680071844.2A CN108513580A (zh) 2015-10-08 2016-10-07 多重基因组编辑

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CN201680071844.2A Division CN108513580A (zh) 2015-10-08 2016-10-07 多重基因组编辑

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CN116064664A true CN116064664A (zh) 2023-05-05

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CN202211152246.1A Pending CN116064664A (zh) 2015-10-08 2016-10-07 多重基因组编辑
CN201680071844.2A Pending CN108513580A (zh) 2015-10-08 2016-10-07 多重基因组编辑

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US (6) US10959413B2 (enExample)
EP (2) EP4491732A3 (enExample)
JP (4) JP7059468B2 (enExample)
KR (2) KR102729669B1 (enExample)
CN (2) CN116064664A (enExample)
AU (2) AU2016335698B2 (enExample)
CA (1) CA3001314A1 (enExample)
CL (1) CL2018000902A1 (enExample)
CO (1) CO2018004763A2 (enExample)
IL (2) IL297017A (enExample)
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Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP6700788B2 (ja) * 2012-12-17 2020-05-27 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性ヒトゲノム改変
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
MX2018004263A (es) * 2015-10-08 2019-08-16 Harvard College Edición genómica multiplexada.
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
DK3386550T3 (da) 2015-12-07 2021-04-26 Arc Bio Llc Fremgangsmåder til fremstilling og anvendelse af guide nukleinsyrer
JP7370702B2 (ja) 2015-12-24 2023-10-30 セレクシス エス．エー． タンパク質製造用の改善された真核細胞およびそれらの作製方法
EP4368637A3 (en) 2016-05-20 2024-07-10 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
EP3555285A4 (en) * 2016-12-14 2020-07-08 Dow AgroSciences LLC RECONSTRUCTION OF SITE SPECIFIC NUCLEASE BINDING SITES
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
KR102746223B1 (ko) * 2017-04-20 2024-12-27 이제네시스, 인크. 유전자 변형 동물의 생성 방법
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN107254551B (zh) * 2017-06-06 2020-04-07 中国人民解放军军事科学院军事医学研究院 鉴定和选育PERV-pol基因缺陷型五指山小型猪新品系的方法
AU2018279112B2 (en) * 2017-06-07 2024-11-07 Arc Bio, Llc Creation and use of guide nucleic acids
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
KR102103103B1 (ko) 2018-08-16 2020-04-21 (주)라트바이오 인위적 뉴클레아제를 생산하는 형질전환 동물 및 형질전환 배아
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110358766A (zh) * 2019-06-10 2019-10-22 云南农业大学 一种基于全基因组CRISPR/Cas9文库筛选异种移植抗原基因的方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN111534541A (zh) * 2020-05-07 2020-08-14 西南大学 一种真核生物CRISPR-Cas9双gRNA载体及构建方法
CN111549060A (zh) * 2020-05-07 2020-08-18 西南大学 一种真核生物CRISPR/Cas9全基因组编辑细胞文库及构建方法
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
CN112280802A (zh) * 2020-05-21 2021-01-29 华中农业大学 基于piggyBac转座子系统制备稳定表达BE4蛋白的猪肾上皮细胞系的方法
AU2022375820A1 (en) 2021-11-01 2024-06-13 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
IL313765A (en) 2021-12-22 2024-08-01 Tome Biosciences Inc Joint provision of a gene editor structure and a donor template
CN114438083A (zh) * 2022-01-29 2022-05-06 五邑大学 识别猪PERV基因的sgRNA及其编码DNA和应用
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1972593A (zh) * 2003-11-21 2007-05-30 雷维维科公司 干扰rna在生产转基因动物中的用途
US20150232833A1 (en) * 2012-12-17 2015-08-20 President And Fellows Of Harvard College RNA-Guided Human Genome Engineering

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU3099801A (en) 2000-01-19 2001-07-31 Biotransplant Incorporated Swine defective for transmission of porcine endogenous retrovirus and uses thereof
EP1582591A3 (en) 2004-02-27 2005-12-21 Bundesrepublik Deutschland vertreten durch das Bundesminsterium für Gesundheit, dieses vertr. durch das Robert-Koch-Institut Sirna and their use for knock down expression of porcine endogenous retrovirus
NZ539491A (en) * 2005-04-15 2008-04-30 Living Cell Products Pty Ltd Swine population and uses thereof
EP2077329A1 (en) * 2008-01-04 2009-07-08 MWM Biomodels GmbH Transgenic pig with altered incretin function
WO2014093701A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US10704060B2 (en) 2013-06-05 2020-07-07 Duke University RNA-guided gene editing and gene regulation
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
SG10201804975PA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
MA41382A (fr) 2015-03-20 2017-11-28 Univ Temple Édition génique basée sur le système crispr/endonucléase à induction par tat
DE102015207944A1 (de) 2015-04-29 2016-11-03 Wacker Chemie Ag Verfahren zur Herstellung organisch modifizierter Aerogele
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CN105154471A (zh) 2015-08-11 2015-12-16 南方医科大学珠江医院 低表达或不表达perv的肝细胞的制备方法
MX2018004263A (es) 2015-10-08 2019-08-16 Harvard College Edición genómica multiplexada.
JP7370702B2 (ja) 2015-12-24 2023-10-30 セレクシス エス．エー． タンパク質製造用の改善された真核細胞およびそれらの作製方法
WO2018144546A1 (en) * 2017-02-01 2018-08-09 President And Fellows Of Harvard College Methods for increasing efficiency of gene editing in cells
KR102746223B1 (ko) 2017-04-20 2024-12-27 이제네시스, 인크. 유전자 변형 동물의 생성 방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1972593A (zh) * 2003-11-21 2007-05-30 雷维维科公司 干扰rna在生产转基因动物中的用途
US20150232833A1 (en) * 2012-12-17 2015-08-20 President And Fellows Of Harvard College RNA-Guided Human Genome Engineering

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ZHOU等: ""Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer", CELLULAR AND MOLECULAR LIFE SCIENCES, vol. 72, 2 October 2014 (2014-10-02), pages 1175 - 1184, XP035456224, DOI: 10.1007/s00018-014-1744-7 *

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