DK3523430T3 - Manipulerede nukleinsyremålrettede nukleinsyrer - Google Patents

Manipulerede nukleinsyremålrettede nukleinsyrer Download PDF

Info

Publication number
DK3523430T3
DK3523430T3 DK17832598.1T DK17832598T DK3523430T3 DK 3523430 T3 DK3523430 T3 DK 3523430T3 DK 17832598 T DK17832598 T DK 17832598T DK 3523430 T3 DK3523430 T3 DK 3523430T3
Authority
DK
Denmark
Prior art keywords
manipulated
nucleic acid
acid targeted
nucleic acids
acids
Prior art date
Application number
DK17832598.1T
Other languages
English (en)
Inventor
Paul Daniel Donohoue
Andrew Paul May
Original Assignee
Caribou Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Caribou Biosciences Inc filed Critical Caribou Biosciences Inc
Application granted granted Critical
Publication of DK3523430T3 publication Critical patent/DK3523430T3/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
DK17832598.1T 2016-12-06 2017-12-05 Manipulerede nukleinsyremålrettede nukleinsyrer DK3523430T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US15/371,188 US9816093B1 (en) 2016-12-06 2016-12-06 Engineered nucleic acid-targeting nucleic acids
US15/787,630 US10590415B2 (en) 2016-12-06 2017-10-18 Engineered nucleic acid-targeting nucleic acids
PCT/US2017/064771 WO2018106727A1 (en) 2016-12-06 2017-12-05 Engineered nuceic acid-targeting nucleic acids

Publications (1)

Publication Number Publication Date
DK3523430T3 true DK3523430T3 (da) 2021-07-26

Family

ID=60255607

Family Applications (1)

Application Number Title Priority Date Filing Date
DK17832598.1T DK3523430T3 (da) 2016-12-06 2017-12-05 Manipulerede nukleinsyremålrettede nukleinsyrer

Country Status (4)

Country Link
US (3) US9816093B1 (da)
EP (1) EP3523430B1 (da)
DK (1) DK3523430T3 (da)
WO (1) WO2018106727A1 (da)

Families Citing this family (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins 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
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US20190225955A1 (en) 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
IL308426A (en) 2016-08-03 2024-01-01 Harvard College Adenosine nuclear base editors and their uses
CA3033327A1 (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
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 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
SG11201908658TA (en) 2017-03-23 2019-10-30 Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592A1 (en) 2017-10-16 2020-06-04 Beam Therapeutics, Inc. Uses of adenosine base editors
WO2019152941A1 (en) 2018-02-05 2019-08-08 Caribou Biosciences, Inc. Engineered gut microbes for reduction of reactivation of detoxified drugs
WO2019173248A1 (en) 2018-03-07 2019-09-12 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
JP2021523745A (ja) 2018-05-16 2021-09-09 シンテゴ コーポレイション ガイドrna設計および使用のための方法およびシステム
JP7428712B2 (ja) * 2018-12-05 2024-02-06 フレッド ハッチンソン キャンサー センター 低/最小操作による遺伝子改変細胞の製造
US20220290120A1 (en) 2019-02-25 2022-09-15 Novome Biotechnologies, Inc. Plasmids for gene editing
WO2020191246A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020218657A1 (ko) * 2019-04-26 2020-10-29 주식회사 툴젠 표적 특이적 crispr 변이체
WO2021007563A1 (en) * 2019-07-11 2021-01-14 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
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.
US20240102007A1 (en) 2021-06-01 2024-03-28 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
WO2023097268A2 (en) * 2021-11-23 2023-06-01 Mantra Bio, Inc. Extracellular vesicles comprising non-naturally occurring modular rna hairpins and uses thereof
CN114685685B (zh) * 2022-04-20 2023-11-07 上海科技大学 编辑rna的融合蛋白及其应用
EP4286401A1 (en) * 2022-06-03 2023-12-06 Technische Universität Dresden Use of an artificial protein or of a nucleic acid encoding the artificial protein for providing a functional ribonucleoprotein complex

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
EP2569425B1 (en) 2010-05-10 2016-07-06 The Regents of The University of California Endoribonuclease compositions and methods of use thereof
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN104204225A (zh) 2012-02-29 2014-12-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
DK3241902T3 (da) 2012-05-25 2018-05-07 Univ California Fremgangsmåder og sammensætninger til rna-rettet target-dna-modificering og til rna-rettet modulering af transkription
US9688971B2 (en) 2012-06-15 2017-06-27 The Regents Of The University Of California Endoribonuclease and methods of use thereof
EP2880171B1 (en) 2012-08-03 2018-10-03 The Regents of The University of California Methods and compositions for controlling gene expression by rna processing
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
IL239344B1 (en) 2012-12-12 2024-02-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
PL2931898T3 (pl) 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL300461A (en) 2012-12-12 2023-04-01 Harvard College Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
JP2016519652A (ja) 2013-03-14 2016-07-07 カリブー・バイオサイエンシーズ・インコーポレイテッド 核酸ターゲティング核酸の組成物および方法
CN105829536A (zh) 2013-08-22 2016-08-03 纳幕尔杜邦公司 用于在不掺入选择性转基因标记的情况下,在植物基因组中产生基因修饰的方法,以及用于这种方法的组合物
AU2014350051A1 (en) 2013-11-18 2016-07-07 Crispr Therapeutics Ag CRISPR-Cas system materials and methods
US20150376587A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
KR20170126875A (ko) 2015-01-28 2017-11-20 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 Crispr 하이브리드 dna/rna 폴리뉴클레오티드 및 사용 방법
EP3277816B1 (en) 2015-04-01 2020-06-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US20160362667A1 (en) 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods

Also Published As

Publication number Publication date
EP3523430A1 (en) 2019-08-14
US9816093B1 (en) 2017-11-14
US20200208155A1 (en) 2020-07-02
US20180155720A1 (en) 2018-06-07
EP3523430B1 (en) 2021-06-30
US10590415B2 (en) 2020-03-17
WO2018106727A1 (en) 2018-06-14
US11001843B2 (en) 2021-05-11

Similar Documents

Publication Publication Date Title
DK3523430T3 (da) Manipulerede nukleinsyremålrettede nukleinsyrer
DK3371310T3 (da) Nucleinsyrekonstruerede targetnucleinsyrer
DK3234132T3 (da) Ligand-modificerede dobbeltstrengede nukleinsyrer
DK3433368T3 (da) Transreplikerende rna
DK3341482T3 (da) Kunstige nukleinsyremolekyler
DK3448990T3 (da) Forbedrede fremgangsmåder til modificering af målnukleinsyrer
HK1248284A1 (zh) 多重乳液核酸擴增
BR112017018054A2 (pt) métodos para a cobertura de sequências de ácidos nucleicos direcionadas
DK3234134T3 (da) Målrettet rna-redigering
BR112017008877A2 (pt) métodos e composições para sequenciamento de ácido nucleico-alvo
MA42846A (fr) Identification universelle
DK4023755T3 (da) Kunstige nukleinsyremolekyler til forbedret proteinudtrykkelse
DK3620534T3 (da) Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
ES2966831T3 (es) Técnicas de indexación de ácidos nucleicos
BR112016030439A2 (pt) ácidos nucléicos para o tratamento de alergias ao amendoim.
HK1243958A1 (zh) 核酸構建體
BR112015023398A2 (pt) métodos para amplificar ácidos nucléicos
GB201507119D0 (en) Nucleic Acid Construct
DK3262066T4 (da) Genterapi
DK3094731T3 (da) Saccharidmodificerede nukleinsyremolekyler
HK1244021A1 (zh) 核酸構建體
DK3351633T3 (da) Antisense-nukleinsyre
GB201507115D0 (en) Nucleic Acid Construct
CL2018000520A1 (es) Proteinas inhibidoras de insectos novedosas
GB201507111D0 (en) Nucleic acid construct