AU2015364282B2 - CRISPR-based compositions and methods of use - Google Patents

CRISPR-based compositions and methods of use Download PDF

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Publication number
AU2015364282B2
AU2015364282B2 AU2015364282A AU2015364282A AU2015364282B2 AU 2015364282 B2 AU2015364282 B2 AU 2015364282B2 AU 2015364282 A AU2015364282 A AU 2015364282A AU 2015364282 A AU2015364282 A AU 2015364282A AU 2015364282 B2 AU2015364282 B2 AU 2015364282B2
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modified
rna
tracrrna
base
artificial sequence
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AU2015364282A1 (en
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Mark Aaron Behlke
Michael Allen Collingwood
Ashley Mae Jacobi
Garrett Richard Rettig
Mollie Sue Schubert
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Integrated DNA Technologies Inc
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Integrated DNA Technologies Inc
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2015364282A 2014-12-18 2015-12-18 CRISPR-based compositions and methods of use Active AU2015364282B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2021204304A AU2021204304B2 (en) 2014-12-18 2021-06-24 CRISPR-based compositions and methods of use
AU2024201685A AU2024201685A1 (en) 2014-12-18 2024-03-14 CRISPR-based compositions and methods of use

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462093588P 2014-12-18 2014-12-18
US62/093,588 2014-12-18
US201562239546P 2015-10-09 2015-10-09
US62/239,546 2015-10-09
PCT/US2015/066942 WO2016100951A2 (en) 2014-12-18 2015-12-18 Crispr-based compositions and methods of use

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AU2015364282A1 AU2015364282A1 (en) 2017-05-18
AU2015364282B2 true AU2015364282B2 (en) 2021-04-15

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AU2021204304A Active AU2021204304B2 (en) 2014-12-18 2021-06-24 CRISPR-based compositions and methods of use
AU2024201685A Pending AU2024201685A1 (en) 2014-12-18 2024-03-14 CRISPR-based compositions and methods of use

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AU2024201685A Pending AU2024201685A1 (en) 2014-12-18 2024-03-14 CRISPR-based compositions and methods of use

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US (4) US9840702B2 (https=)
EP (2) EP3234133B1 (https=)
JP (4) JP2017538427A (https=)
CN (3) CN107208096B (https=)
AU (3) AU2015364282B2 (https=)
CA (1) CA2970683A1 (https=)
DK (1) DK3234133T3 (https=)
WO (1) WO2016100951A2 (https=)

Families Citing this family (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US10040048B1 (en) 2014-09-25 2018-08-07 Synthego Corporation Automated modular system and method for production of biopolymers
CA2970683A1 (en) 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
WO2017004261A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
WO2017136794A1 (en) 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
US10767175B2 (en) * 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
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
IL247752A0 (en) 2016-09-11 2016-11-30 Yeda Res & Dev Compositions and methods for modulating gene expression for site-directed mutagenesis
CA3034931A1 (en) 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
DE102016118109A1 (de) * 2016-09-26 2018-03-29 Newfrey Llc Fügeverfahren zum vorlochfreien Verbinden von wenigstens einem ersten Bauteil mit einem zweiten Bauteil
KR20260019012A (ko) 2016-10-07 2026-02-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Ｓ. 피오게네스 ｃａｓ９ 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR20190082318A (ko) 2016-11-22 2019-07-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 및 방법
US20190032131A1 (en) 2016-12-12 2019-01-31 Integrated Dna Technologies, Inc. Genome editing detection
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
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
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018175636A2 (en) * 2017-03-22 2018-09-27 Novartis Ag Compositions and methods for immunooncology
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN110678548A (zh) * 2017-03-31 2020-01-10 埃吉诺维亚公司 抗病毒治疗剂
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
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
US20210130817A1 (en) 2017-07-14 2021-05-06 Cure Genetics Co., Ltd. Gene Editing System and Gene Editing Method
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US20190076814A1 (en) * 2017-09-11 2019-03-14 Synthego Corporation Biopolymer synthesis system and method
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
EP3724216A1 (en) 2017-12-15 2020-10-21 Dana Farber Cancer Institute, Inc. Stabilized peptide-mediated targeted protein degradation
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126037A1 (en) * 2017-12-19 2019-06-27 City Of Hope Modified tracrrnas grnas, and uses thereof
WO2019147275A1 (en) * 2018-01-26 2019-08-01 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
WO2019147743A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
WO2019156475A1 (ko) * 2018-02-06 2019-08-15 주식회사 진씨커 돌연변이 세포 유리 유전자 분리 키트 및 이를 이용한 돌연변이 세포 유리 유전자 분리 방법
KR102086689B1 (ko) 2018-02-06 2020-03-09 주식회사 진씨커 돌연변이 세포 유리 유전자 분리 키트 및 이를 이용한 돌연변이 세포 유리 유전자 분리 방법
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
US20210363518A1 (en) * 2018-03-19 2021-11-25 University Of Massachusetts Modified guide rnas for crispr genome editing
WO2019213910A1 (en) * 2018-05-10 2019-11-14 Syngenta Participations Ag Methods and compositions for targeted editing of polynucleotides
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
AU2019282824C1 (en) * 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing
CN110616187B (zh) * 2018-06-20 2021-12-03 西安桑尼赛尔生物医药有限公司 CRISPR-Cas9高效敲入嵌合抗原受体基因到T细胞特定基因组位点的方法及应用
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020033774A1 (en) 2018-08-08 2020-02-13 Integrated Dna Technologies, Inc. Novel mutations that enhance the dna cleavage activity of acidaminococcus sp. cpf1
EP3617325B1 (en) 2018-08-29 2021-10-20 Leibniz-Institut für Pflanzengenetik und Kulturpflanzenforschung (IPK) Rna-guided endonuclease dna labeling
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN109660518B (zh) * 2018-11-22 2020-12-18 北京六方云信息技术有限公司 网络的通信数据检测方法、装置以及机器可读存储介质
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN113811608B (zh) 2019-02-22 2024-10-29 合成Dna技术公司 毛螺菌科细菌nd2006 cas12a突变型基因和由其编码的多肽
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
LU101172B1 (en) 2019-04-10 2020-10-12 Technische Univ Hamburg Method for in vivo screening of engineered enzyme variants
EP3953465A1 (en) 2019-04-10 2022-02-16 Technische Universität Hamburg Novel dhap synthase
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022547699A (ja) 2019-09-12 2022-11-15 グラクソスミスクライン、インテレクチュアル、プロパティー、ディベロップメント、リミテッド ライブラリーのスクリーニング方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4438729A3 (en) * 2019-12-09 2025-03-26 Caribou Biosciences, Inc. Crispr abasic restricted nucleotides and crispr accuracy via analogs
US12012433B1 (en) 2020-02-27 2024-06-18 Integrated Dna Technologies Inc. Expression and purification of Cas enzymes
CN116096878A (zh) 2020-05-01 2023-05-09 合成Dna技术公司 在非经典tttt前间区序列邻近基序处具有增强的切割活性的毛螺菌科菌种cas12a突变体
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CN115956120A (zh) * 2020-05-12 2023-04-11 马萨诸塞大学 用于crispr基因组编辑的修饰的指导rna
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
CA3265664A1 (en) * 2022-08-25 2024-02-29 Life Edit Therapeutics, Inc. CHEMICAL MODIFICATION OF GUIDE RNA WITH A LOCKED NUCLEIC ACID FOR RNA-GUIDED NUCLEASE-MEDIATED GENE EDITING
US20260071238A1 (en) 2022-09-02 2026-03-12 Janssen Biotech, Inc. Materials and processes for engineering hypoimmunogenicity
CN121241136A (zh) * 2023-04-07 2025-12-30 基因泰克公司 经修饰的指导rna
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014124226A1 (en) * 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US20140273232A1 (en) * 2012-12-12 2014-09-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014144592A2 (en) * 2013-03-15 2014-09-18 The General Hospital Corporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
CN103906848B (zh) 2011-08-18 2016-03-02 雀巢产品技术援助有限公司 用于检测等位变体的组合物和方法
US9895451B2 (en) * 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
PL3360964T3 (pl) * 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
CN113355357B (zh) * 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140212869A1 (en) * 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
EP2922393B2 (en) * 2013-02-27 2022-12-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Gene editing in the oocyte by cas9 nucleases
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
WO2014194190A1 (en) * 2013-05-30 2014-12-04 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
CN120574876A (zh) 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
JP6361121B2 (ja) 2013-11-29 2018-07-25 三菱ケミカル株式会社 粘着剤樹脂組成物
US20150376586A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
WO2016080097A1 (ja) * 2014-11-17 2016-05-26 国立大学法人東京医科歯科大学 簡便で高効率の遺伝子改変非ヒト哺乳動物の作製方法
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
CA2970683A1 (en) 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
JP6799058B2 (ja) 2015-09-21 2020-12-09 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． アレル選択的な遺伝子編集およびその使用
WO2019147275A1 (en) 2018-01-26 2019-08-01 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
US20140273232A1 (en) * 2012-12-12 2014-09-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014124226A1 (en) * 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
WO2014144592A2 (en) * 2013-03-15 2014-09-18 The General Hospital Corporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
M. JINEK ET AL, "A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity", SCIENCE, US, (2012-08-17), vol. 337, no. 6096, doi:10.1126/science.1225829, ISSN 0036-8075, pages 816 - 821 *

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