AU2016335698B2 - Multiplexed genome editing - Google Patents

Multiplexed genome editing Download PDF

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Publication number
AU2016335698B2
AU2016335698B2 AU2016335698A AU2016335698A AU2016335698B2 AU 2016335698 B2 AU2016335698 B2 AU 2016335698B2 AU 2016335698 A AU2016335698 A AU 2016335698A AU 2016335698 A AU2016335698 A AU 2016335698A AU 2016335698 B2 AU2016335698 B2 AU 2016335698B2
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cell
perv
porcine
genetically engineered
sequence
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AU2016335698A1 (en
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George M. Church
Marc Guell
Luhan Yang
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Harvard University
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Harvard University
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    • A—HUMAN NECESSITIES
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    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
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    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
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    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A01K2207/05—Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
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AU2016335698A 2015-10-08 2016-10-07 Multiplexed genome editing Active AU2016335698B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023201178A AU2023201178B2 (en) 2015-10-08 2023-02-28 Multiplexed genome editing

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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

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AU2016335698A1 AU2016335698A1 (en) 2018-05-10
AU2016335698B2 true AU2016335698B2 (en) 2022-12-01

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US (6) US10959413B2 (OSRAM)
EP (2) EP3359661B1 (OSRAM)
JP (4) JP7059468B2 (OSRAM)
KR (2) KR102729669B1 (OSRAM)
CN (2) CN108513580A (OSRAM)
AU (2) AU2016335698B2 (OSRAM)
CA (1) CA3001314A1 (OSRAM)
CL (1) CL2018000902A1 (OSRAM)
CO (1) CO2018004763A2 (OSRAM)
IL (2) IL297017A (OSRAM)
MX (2) MX2018004263A (OSRAM)
NZ (1) NZ741778A (OSRAM)
SA (2) SA518391294B1 (OSRAM)
WO (1) WO2017062723A1 (OSRAM)
ZA (1) ZA201802461B (OSRAM)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
EP4481048A3 (en) * 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
IL301900A (en) 2014-11-21 2023-06-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
KR102729669B1 (ko) 2015-10-08 2024-11-12 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 다중화 게놈 편집
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
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
JP7324583B2 (ja) 2016-05-20 2023-08-10 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018111640A1 (en) * 2016-12-14 2018-06-21 Dow Agrosciences Llc Reconstruction of site specific nuclease binding sites
US10745677B2 (en) 2016-12-23 2020-08-18 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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN116555353A (zh) * 2017-04-20 2023-08-08 E开创生物技术股份有限公司 产生基因修改的动物的方法
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CA3065384A1 (en) * 2017-06-07 2018-12-13 Arc Bio, Llc Creation and use of guide nucleic acids
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 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
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 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
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
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
CA3237300A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct 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
US20050266561A1 (en) * 2003-11-21 2005-12-01 Revivicor, Inc. Use of interfering RNA in the production of transgenic animals
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection

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
EP4481048A3 (en) * 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
KR102733409B1 (ko) 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
CA2932479A1 (en) * 2013-12-12 2015-06-18 The Rockefeller University 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
KR102729669B1 (ko) 2015-10-08 2024-11-12 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 다중화 게놈 편집
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
CN110546267B (zh) * 2017-02-01 2024-09-13 哈佛学院董事及会员团体 提高细胞基因编辑效率的方法
CN116555353A (zh) * 2017-04-20 2023-08-08 E开创生物技术股份有限公司 产生基因修改的动物的方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050266561A1 (en) * 2003-11-21 2005-12-01 Revivicor, Inc. Use of interfering RNA in the production of transgenic animals
WO2015031775A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MARWAN SEMAAN, IVANUSIC DANIEL, DENNER JOACHIM: "Cytotoxic Effects during Knock Out of Multiple Porcine Endogenous Retrovirus (PERV) Sequences in the Pig Genome by Zinc Finger Nucleases (ZFN)", PLOS ONE, vol. 10, no. 4, 24 April 2015 (2015-04-24), pages e0122059, XP055576508, DOI: 10.1371/journal.pone.0122059 *

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