JP2017537626A5 - - Google Patents

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JP2017537626A5
JP2017537626A5 JP2017529610A JP2017529610A JP2017537626A5 JP 2017537626 A5 JP2017537626 A5 JP 2017537626A5 JP 2017529610 A JP2017529610 A JP 2017529610A JP 2017529610 A JP2017529610 A JP 2017529610A JP 2017537626 A5 JP2017537626 A5 JP 2017537626A5
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Japan
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guide rna
synthetic guide
linkage
rna
sequence
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JP2017529610A
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JP7068821B2 (ja
JP2017537626A (ja
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JP2017529610A 2014-12-03 2015-12-03 化学修飾を有するガイドrna Active JP7068821B2 (ja)

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JP2022020615A JP7579820B2 (ja) 2014-12-03 2022-02-14 化学修飾を有するガイドrna

Applications Claiming Priority (7)

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US201462087211P 2014-12-03 2014-12-03
US62/087,211 2014-12-03
US201562146189P 2015-04-10 2015-04-10
US62/146,189 2015-04-10
US201562256095P 2015-11-16 2015-11-16
US62/256,095 2015-11-16
PCT/US2015/000143 WO2016089433A1 (en) 2014-12-03 2015-12-03 Guide rna with chemical modifications

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JP2017537626A JP2017537626A (ja) 2017-12-21
JP2017537626A5 true JP2017537626A5 (https=) 2019-01-17
JP7068821B2 JP7068821B2 (ja) 2022-05-17

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JP2022020615A Active JP7579820B2 (ja) 2014-12-03 2022-02-14 化学修飾を有するガイドrna

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US (5) US10900034B2 (https=)
EP (2) EP3227447B1 (https=)
JP (2) JP7068821B2 (https=)
KR (2) KR102629128B1 (https=)
CN (1) CN107250148B (https=)
AU (1) AU2015355546B2 (https=)
CA (1) CA2969619A1 (https=)
WO (1) WO2016089433A1 (https=)

Families Citing this family (188)

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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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3690044B1 (en) 2014-02-11 2024-01-10 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
CA2970683A1 (en) 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
BR112017017812A2 (en) 2015-02-23 2018-04-10 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
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
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
WO2017004261A1 (en) * 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
GB2592821B (en) * 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
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
US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
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
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
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
EP4485466A3 (en) * 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
EP3516061A1 (en) 2016-09-23 2019-07-31 Synthena AG Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
CA3034931A1 (en) 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
US10669539B2 (en) * 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20260019012A (ko) 2016-10-07 2026-02-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. S. 피오게네스 cas9 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
EP3315125A1 (en) 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipid nanoparticle formulation
US11732258B2 (en) 2016-11-02 2023-08-22 President And Fellows Of Harvard College Engineered guide RNA sequences for in situ detection and sequencing
WO2018094356A2 (en) * 2016-11-18 2018-05-24 Genedit Inc. Compositions and methods for target nucleic acid modification
KR20190082318A (ko) 2016-11-22 2019-07-09 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 및 방법
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
TWI851534B (zh) 2016-12-22 2024-08-11 美商英特利亞醫療公司 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法
WO2018118587A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Modified polynucleotides for antiviral therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3562942A4 (en) * 2016-12-28 2020-12-09 Ionis Pharmaceuticals, Inc. MODIFIED CRISPR-RNA AND ITS USES
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
RU2019130504A (ru) 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
US20180258485A1 (en) * 2017-03-07 2018-09-13 The Regents Of The University Of Colorado, A Body Corporate Boranephosphonate Detection Probes and Methods For Producing and Using the Same
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
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
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
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11166985B2 (en) 2017-05-12 2021-11-09 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
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
MX2019013514A (es) 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
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
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
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
GB2582865B (en) * 2017-09-11 2022-05-04 Synthego Corp Biopolymer synthesis system and method
SG11202002481RA (en) 2017-09-19 2020-04-29 Tropic Biosciences Uk Ltd Modifying the specificity of non-coding rna molecules for silencing gene expression in eukaryotic cells
BR112020006601A2 (pt) 2017-10-02 2020-12-08 Genedit Inc. Rna guia de cpf1 modificado
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20210277392A1 (en) * 2017-11-02 2021-09-09 The Governors Of The University Of Alberta Chemically-modified guide rnas to improve crispr-cas protein specificity
AU2018378479B2 (en) 2017-12-05 2025-03-13 Vertex Pharmaceuticals Incorporated CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11268092B2 (en) 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
WO2019147743A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11566236B2 (en) 2018-02-05 2023-01-31 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019156726A1 (en) * 2018-02-09 2019-08-15 Ohio State Innovation Foundation Rna nanostructures, methods of making, and uses thereof
KR102907245B1 (ko) * 2018-03-14 2026-01-05 에디타스 메디신, 인코포레이티드 혈색소병증 치료를 위한 시스템 및 방법
US20210363518A1 (en) * 2018-03-19 2021-11-25 University Of Massachusetts Modified guide rnas for crispr genome editing
CN112512594A (zh) * 2018-03-23 2021-03-16 瑞非生物科技有限公司 经由基因调节多肽的条件性核定位的基因调节
MX2020012028A (es) 2018-05-11 2021-03-29 Crispr Therapeutics Ag Metodos y composiciones para tratar el cancer.
JP2021522825A (ja) * 2018-05-11 2021-09-02 ユニベルシテ ラバル CRISPR/Cas9システムおよびその使用
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019224603A1 (en) * 2018-05-24 2019-11-28 Crispr Therapeutics Ag Methods and compositions for efficient gene deletion
AU2019282824C1 (en) * 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing
CN112469834A (zh) * 2018-06-25 2021-03-09 生物纳米基因公司 Dna的标记
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
WO2020014577A1 (en) * 2018-07-13 2020-01-16 Allele Biotechnology And Pharmaceuticals, Inc. Methods of achieving high specificity of genome editing
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
US20210310002A1 (en) * 2018-08-31 2021-10-07 City Of Hope Cationic compounds for delivery of nucleic acids
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
JP7553100B2 (ja) * 2018-12-12 2024-09-18 国立大学法人九州大学 ゲノム編集された細胞の製造方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB201901873D0 (en) * 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
GB201903519D0 (en) 2019-03-14 2019-05-01 Tropic Biosciences Uk Ltd Introducing silencing activity to dysfunctional rna molecules and modifying their specificity against a gene of interest
GB201903520D0 (en) 2019-03-14 2019-05-01 Tropic Biosciences Uk Ltd Modifying the specificity of non-coding rna molecules for silencing genes in eukaryotic cells
GB201903521D0 (en) 2019-03-14 2019-05-01 Tropic Biosciences Uk Ltd No title
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
MX2021013359A (es) 2019-04-30 2022-01-31 Crispr Therapeutics Ag Terapia de celulas alogénicas de neoplasias malignas de células b usando células t modificadas genéticamente dirigidas a cd19.
AU2020280103A1 (en) 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CA3127684A1 (en) * 2019-07-19 2021-01-28 Synthego Corporation Stabilized crispr complexes
JP7630904B2 (ja) * 2019-07-23 2025-02-18 株式会社東芝 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法
AU2020338011A1 (en) 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2021041977A1 (en) 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cd123 modification
WO2021055811A1 (en) * 2019-09-19 2021-03-25 Modernatx, Inc. Cap guides and methods of use thereof for rna mapping
JP7689949B2 (ja) 2019-10-03 2025-06-09 アーティサン ディベロップメント ラブズ インコーポレイテッド 操作されたデュアルガイド核酸を有するcrisprシステム
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP7640461B2 (ja) 2019-10-11 2025-03-05 住友化学株式会社 核酸オリゴマーの製造方法
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
US20230227820A1 (en) * 2019-11-28 2023-07-20 Korea University Research And Business Foundation Rna interference-inducing nucleic acid comprising 8-oxoguanine, modified nucleic acid binding to microrna comprising 8-oxoguanine, and uses thereof
EP4438729A3 (en) * 2019-12-09 2025-03-26 Caribou Biosciences, Inc. Crispr abasic restricted nucleotides and crispr accuracy via analogs
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
CN114901360A (zh) 2019-12-20 2022-08-12 库瑞瓦格股份公司 用于递送核酸的新型脂质纳米颗粒
EP4098655A4 (en) 2020-01-29 2024-02-28 Sumitomo Chemical Company, Limited METHOD FOR PRODUCING NUCLEIC ACID OLIGOMERS
JP7667788B2 (ja) 2020-01-29 2025-04-23 住友化学株式会社 核酸オリゴマーの製造方法
EP4130018A4 (en) 2020-03-27 2024-04-17 Sumitomo Chemical Company, Limited METHOD FOR PRODUCING A NUCLEIC ACID OLIGOMER
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
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
WO2022047168A1 (en) 2020-08-28 2022-03-03 Vor Biopharma Inc. Compositions and methods for cll1 modification
US20240238344A1 (en) 2020-08-28 2024-07-18 Vor Biopharma Inc. Compositions and methods for cd123 modification
WO2022056459A1 (en) 2020-09-14 2022-03-17 Vor Biopharma, Inc. Compositions and methods for cd5 modification
JP2023541457A (ja) 2020-09-14 2023-10-02 ブイオーアール バイオファーマ インコーポレーテッド Cd38修飾のための化合物および方法
WO2022061115A1 (en) 2020-09-18 2022-03-24 Vor Biopharma Inc. Compositions and methods for cd7 modification
CA3193089A1 (en) 2020-09-18 2022-03-24 Jamie KERSHNER Constructs and uses thereof for efficient and specific genome editing
KR20230074205A (ko) 2020-09-24 2023-05-26 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
US20230364233A1 (en) 2020-09-28 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd6 modification
WO2022072643A1 (en) 2020-09-30 2022-04-07 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
CA3198435A1 (en) * 2020-10-08 2022-04-14 Genkore Inc. Engineered guide rna for optimized crispr/cas12f1 (cas14a1) system and use thereof
WO2022093983A1 (en) 2020-10-27 2022-05-05 Vor Biopharma, Inc. Compositions and methods for treating hematopoietic malignancy
WO2022094245A1 (en) 2020-10-30 2022-05-05 Vor Biopharma, Inc. Compositions and methods for bcma modification
EP4243858A1 (en) 2020-11-13 2023-09-20 Vor Biopharma Inc. Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors
CN112266951B (zh) * 2020-11-23 2021-07-20 广东省农业科学院植物保护研究所 一种生物素标记引物延伸方法及其应用
WO2022115878A1 (en) * 2020-11-30 2022-06-02 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas-mediated gene editing of human stem cells
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
JP2024502820A (ja) 2020-12-31 2024-01-23 ブイオーアール バイオファーマ インコーポレーテッド Cd34遺伝子改変のための組成物及び方法
AU2022227650A1 (en) 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
US20240200059A1 (en) 2021-04-09 2024-06-20 Vor Biopharma Inc. Photocleavable guide rnas and methods of use thereof
CN113512767B (zh) * 2021-04-30 2022-09-23 天津诺禾致源生物信息科技有限公司 用于构建small RNA文库的接头、试剂盒及small RNA文库的构建方法
EP4419672A2 (en) 2021-06-01 2024-08-28 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
EP4355879A4 (en) 2021-06-14 2025-07-23 Kamau Therapeutics Inc METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS
EP4370676A2 (en) 2021-06-18 2024-05-22 Artisan Development Labs, Inc. Compositions and methods for targeting, editing or modifying human genes
WO2023278834A1 (en) * 2021-07-02 2023-01-05 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Kinetic barcoding to enhance specificity of crispr/cas reactions
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
WO2023283495A1 (en) * 2021-07-09 2023-01-12 The Brigham And Women's Hospital, Inc. Crispr-based protein barcoding and surface assembly
AU2022324093A1 (en) 2021-08-02 2024-02-08 Vor Biopharma Inc. Compositions and methods for gene modification
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
JP2024534945A (ja) 2021-09-10 2024-09-26 アジレント・テクノロジーズ・インク 化学修飾を有するプライム編集のためのガイドrna
EP4402266A4 (en) * 2021-09-14 2025-11-05 Agilent Technologies Inc GUIDELINES FOR USING RNA WITH CHEMICAL MODIFICATIONS
US20240417755A1 (en) 2021-09-27 2024-12-19 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
CN117980285A (zh) 2021-09-28 2024-05-03 住友化学株式会社 纯化二氯乙酸的制造方法
IL312507A (en) * 2021-11-03 2024-07-01 Intellia Therapeutics Inc Modified guide rnas for gene editing
JP2024543369A (ja) 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Emr2修飾のための化合物及び方法
EP4453245A4 (en) * 2021-12-23 2025-12-31 Mammoth Biosciences Inc DOSING IMPROVEMENTS BASED ON A PROGRAMMABLE NUCLEASE
US20250179531A1 (en) 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
EP4486881A1 (en) 2022-03-01 2025-01-08 Celyntra Therapeutics SA Composition and methods for transgene insertion
US12509485B2 (en) 2022-03-23 2025-12-30 Sumitomo Chemical Company, Limited Method for producing nucleic acid oligomer
WO2023185697A2 (en) 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
AU2023251040A1 (en) 2022-04-04 2024-10-17 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
US20250308637A1 (en) 2022-05-20 2025-10-02 Celyntra Therapeutics Sa Systems and methods for assessing risk of genome editing events
WO2024015925A2 (en) 2022-07-13 2024-01-18 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
EP4559925A1 (en) 2022-07-22 2025-05-28 Sumitomo Chemical Company, Limited Production method of oligonucleotide
JPWO2024024873A1 (https=) 2022-07-28 2024-02-01
WO2024061296A2 (en) 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2024089953A1 (ja) 2022-10-27 2024-05-02 住友化学株式会社 オリゴヌクレオチドの製造方法
EP4638477A1 (en) 2022-12-19 2025-10-29 Kamau Therapeutics, Inc. Improved peptide inhibitors of p53 binding protein 53bp1
WO2024138131A1 (en) * 2022-12-22 2024-06-27 Trustees Of Tufts College Expanding applications of zgtc alphabet in protein expression and gene editing
JPWO2024143276A1 (https=) 2022-12-26 2024-07-04
WO2024140737A1 (en) * 2022-12-26 2024-07-04 Huidagene Therapeutics Co., Ltd. Modified guide rna and uses thereof
WO2024163787A1 (en) * 2023-02-01 2024-08-08 University Hospitals Cleveland Medical Center Crispr technologies for diagnostic applications
WO2024168312A1 (en) 2023-02-09 2024-08-15 Vor Biopharma Inc. Methods for treating hematopoietic malignancy
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
CN121241136A (zh) * 2023-04-07 2025-12-30 基因泰克公司 经修饰的指导rna
CN121079412A (zh) * 2023-04-28 2025-12-05 比姆医疗股份有限公司 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
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025076141A1 (en) 2023-10-03 2025-04-10 Inari Agriculture Technology, Inc. Viral delivery of grna to the scion
WO2026020120A1 (en) * 2024-07-19 2026-01-22 University Of Massachusetts Increasing cas9 genome editing fidelity through attenuation of guide rna watson-crick base pairing potential

Family Cites Families (137)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5032401A (en) 1989-06-15 1991-07-16 Alpha Beta Technology Glucan drug delivery system and adjuvant
US7371580B2 (en) 2001-08-24 2008-05-13 Agilent Technologies, Inc. Use of unstructured nucleic acids in assaying nucleic acid molecules
WO2005065419A2 (en) 2003-12-29 2005-07-21 Am Biosolutions Method of culturing cells
US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
DK2476689T3 (en) 2007-05-10 2016-01-18 Agilent Technologies Inc Thiocarbonbeskyttende groups for RNA synthesis
US20100076183A1 (en) 2008-09-22 2010-03-25 Dellinger Douglas J Protected monomer and method of final deprotection for rna synthesis
US9585920B2 (en) 2011-02-04 2017-03-07 Katherine Rose Kovarik Method and system for treating cancer cachexia
US9528124B2 (en) 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) * 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
JP6351577B2 (ja) 2012-05-21 2018-07-04 アジレント・テクノロジーズ・インクAgilent Technologies, Inc. 組成物及びオリゴヌクレオチドをコンジュゲートする方法
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
UA118090C2 (uk) 2012-09-07 2018-11-26 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив
US9914930B2 (en) 2012-09-07 2018-03-13 Dow Agrosciences Llc FAD3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
US20140075593A1 (en) 2012-09-07 2014-03-13 Dow Agrosciences Llc Fluorescence activated cell sorting (facs) enrichment to generate plants
AU2013335451C1 (en) 2012-10-23 2024-07-04 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
US20150315576A1 (en) 2012-11-01 2015-11-05 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
KR102121086B1 (ko) 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
MX375143B (es) 2012-12-06 2025-03-06 Synthetic Genomics Inc Mutantes de alga que tienen un fenotipo aclimatado a alta luz bloqueado.
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
IL239344B2 (en) * 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
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
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093688A1 (en) 2012-12-12 2014-06-19 1Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
US20160138027A1 (en) 2013-03-14 2016-05-19 The Board Of Trustees Of The Leland Stanford Junior University Treatment of diseases and conditions associated with dysregulation of mammalian target of rapamycin complex 1 (mtorc1)
WO2014159719A1 (en) 2013-03-14 2014-10-02 Scrips Health Methods of isolating nucleic acids
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
WO2014144155A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota Engineering plant genomes using crispr/cas systems
WO2014145736A2 (en) 2013-03-15 2014-09-18 Transposagen Biopharmaceuticals, Inc. Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
US20160040155A1 (en) 2013-04-16 2016-02-11 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
AU2014273082B2 (en) 2013-05-29 2018-11-08 Cellectis A method for producing precise DNA cleavage using Cas9 nickase activity
SG10201913068PA (en) 2013-06-04 2020-02-27 Harvard College Rna-guided transcriptional regulation
US9982277B2 (en) 2013-06-11 2018-05-29 The Regents Of The University Of California Methods and compositions for target DNA modification
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
ES2929143T3 (es) 2013-07-09 2022-11-25 Harvard College Ingeniería genómica guiada por ARN múltiplex
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
EP3666892A1 (en) 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US20160143256A1 (en) 2013-07-10 2016-05-26 Joseph A. MAJZOUB Mrap2 knockouts
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in 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
US20150064149A1 (en) 2013-09-04 2015-03-05 Mice With Horns, LLC. Materials and methods for correcting recessive mutations in animals
US9765404B2 (en) 2013-09-04 2017-09-19 Dow Agrosciences Llc Rapid assay for identifying transformants having targeted donor insertion
EP3636750A1 (en) 2013-09-04 2020-04-15 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150071946A1 (en) 2013-09-06 2015-03-12 The Johns Hopkins University Tumor-specific retrotransposon insertions
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
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
AU2014331939A1 (en) 2013-10-08 2016-04-28 Amelia HENRY Drought-resistant cereal grasses and related materials and methods
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
CN105980395A (zh) 2013-11-04 2016-09-28 美国陶氏益农公司 最优大豆座位
NZ746567A (en) 2013-11-04 2019-09-27 Dow Agrosciences Llc Optimal soybean loci
JP6560203B2 (ja) 2013-11-04 2019-08-14 ダウ アグロサイエンシィズ エルエルシー 遺伝子ターゲッティングのためのユニバーサルドナー系
NZ719494A (en) 2013-11-04 2017-09-29 Dow Agrosciences Llc Optimal maize loci
UY35812A (es) 2013-11-04 2015-05-29 Dow Agrosciences Llc ?loci de maíz óptimos?.
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US11326209B2 (en) 2013-11-07 2022-05-10 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
WO2015075056A1 (en) 2013-11-19 2015-05-28 Thermo Fisher Scientific Baltics Uab Programmable enzymes for isolation of specific dna fragments
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法
WO2015089427A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
US20150344836A1 (en) 2014-05-30 2015-12-03 Ohio State Innovation Foundation Agrobacterium Strains for Plant Transformation and Related Materials and Methods
WO2015184259A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
CN106604994B (zh) 2014-06-23 2021-12-14 通用医疗公司 通过测序评估的DSBs的全基因组无偏鉴定(GUIDE-Seq)
ES2781323T3 (es) 2014-06-23 2020-09-01 Regeneron Pharma Montaje de ADN mediado por nucleasas
EP3164148A4 (en) 2014-06-25 2017-12-13 Cold Spring Harbor Laboratory Methods and compositions for inhibiting growth and epithelial to mesenchymal transition (emt) in cancer cells
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
EP3778891A1 (en) 2014-08-27 2021-02-17 New England Biolabs, Inc. Synthon formation
SG11201701245QA (en) 2014-08-27 2017-03-30 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
CN107072165B (zh) 2014-09-26 2021-05-28 先锋国际良种公司 小麦Ms1多核苷酸、多肽以及使用方法
EP3198015A1 (en) 2014-09-26 2017-08-02 Philip Morris Products S.a.s. Reducing tobacco specific nitrosamines through alteration of the nitrate assimilation pathway
KR20170064540A (ko) 2014-09-26 2017-06-09 투 포어 가이즈, 인코포레이티드 합성 프로브의 나노포어 감지에 의한 표적 서열 검출
US11408007B2 (en) 2014-09-26 2022-08-09 Yale University Compositions and methods for biocontainment of microorganisms
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
JP6723230B2 (ja) 2014-10-09 2020-07-15 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 神経障害性疼痛を治療するためのcsf1−dap12経路のメンバー遺伝子の標的破壊
WO2016057755A1 (en) 2014-10-09 2016-04-14 Anthrogenesis Corporation Placenta-derived adherent cell exosomes and uses thereof
TWI716367B (zh) 2014-10-31 2021-01-21 麻省理工學院 用於常間回文重複序列叢集(crispr)之大量平行組合性基因學
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
US20170335331A1 (en) 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
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
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
EP3390624A4 (en) 2015-12-18 2019-07-10 The Regents of The University of California Modified site-directed modifying polypeptides and methods of use thereof
WO2017106414A1 (en) 2015-12-18 2017-06-22 Danisco Us Inc. Methods and compositions for polymerase ii (pol-ii) based guide rna expression
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
US20190075770A1 (en) 2015-12-18 2019-03-14 Japan Science And Technology Agency Genetic modification non-human organism, egg cells, fertilized eggs, and method for modifying target genes
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression

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