KR102629128B1 - 화학적 변형을 갖는 가이드 rna - Google Patents

화학적 변형을 갖는 가이드 rna Download PDF

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KR102629128B1
KR102629128B1 KR1020177018311A KR20177018311A KR102629128B1 KR 102629128 B1 KR102629128 B1 KR 102629128B1 KR 1020177018311 A KR1020177018311 A KR 1020177018311A KR 20177018311 A KR20177018311 A KR 20177018311A KR 102629128 B1 KR102629128 B1 KR 102629128B1
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guide rna
sequence
methyl
modification
modifications
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KR20170084334A (ko
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다니엘 이 라이언
더글라스 제이 델링저
제프리 알 샘슨
로버트 카이저
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애질런트 테크놀로지스, 인크.
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KR1020177018311A 2014-12-03 2015-12-03 화학적 변형을 갖는 가이드 rna Active KR102629128B1 (ko)

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KR1020247002012A KR102763527B1 (ko) 2014-12-03 2015-12-03 화학적 변형을 갖는 가이드 rna

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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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KR102629128B1 true KR102629128B1 (ko) 2024-01-25

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US (5) US10900034B2 (enExample)
EP (2) EP3227447B1 (enExample)
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CN (1) CN107250148B (enExample)
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Families Citing this family (187)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
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
DE212015000061U1 (de) 2014-02-11 2017-09-03 The Regents Of The University Of Colorado, A Body Corporate CRISPR-ermöglichtes Multiplex Genom Engineering
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
CA2968336C (en) 2014-12-17 2021-11-23 Proqr Therapeutics Ii B.V. Construct for site directed editing of an adenosine nucleotide in target rna
EP3786296A1 (en) 2014-12-18 2021-03-03 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
BR112017017810A2 (pt) 2015-02-23 2018-04-10 Crispr Therapeutics Ag materiais e métodos para tratamento de hemoglobinopatias
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including 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
US11414657B2 (en) * 2015-06-29 2022-08-16 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
IL295616A (en) 2015-07-31 2022-10-01 Us Health Adapted cells and treatment methods
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
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
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
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会（法人团体） 用于生成条形码化组合文库的方法
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
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018035388A1 (en) * 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3507366B1 (en) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Chemically modified single-stranded rna-editing oligonucleotides
CA3034931A1 (en) 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
US11118179B2 (en) 2016-09-23 2021-09-14 Synthena Ag Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
US10669539B2 (en) * 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
CN110462034A (zh) 2016-10-07 2019-11-15 综合Dna技术公司 化脓链球菌cas9突变基因和由其编码的多肽
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
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
EP3541945A4 (en) * 2016-11-18 2020-12-09 Genedit Inc. COMPOSITIONS AND METHODS OF MODIFICATION OF TARGET NUCLEIC ACIDS
JP2019535287A (ja) 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Ｃｒｉｓｐｒ／ｃｐｆ１システム及び方法
IL267024B2 (en) * 2016-12-08 2023-12-01 Intellia Therapeutics Inc Adapted leader RNAs for genomic editing
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
WO2018118587A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Modified polynucleotides for antiviral therapy
MY204487A (en) * 2016-12-22 2024-08-30 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US20190382751A1 (en) * 2016-12-28 2019-12-19 Ionis Pharmaceuticals, Inc. Modified crispr rna and uses thereof
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
BR112019017751A2 (pt) 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN110678548A (zh) 2017-03-31 2020-01-10 埃吉诺维亚公司 抗病毒治疗剂
WO2018209158A2 (en) 2017-05-10 2018-11-15 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
IL270415B2 (en) 2017-05-12 2024-08-01 Crispr Therapeutics Ag Materials and methods for cell engineering and their uses in immuno-oncology
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
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
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
EP3681637A4 (en) * 2017-09-11 2021-06-02 Synthego Corporation SYSTEM AND PROCEDURE FOR BIOPOLYMER SYNTHESIS
WO2019058253A1 (en) 2017-09-19 2019-03-28 Tropic Biosciences UK Limited MODIFICATION OF THE SPECIFICITY OF NON-CODING RNA MOLECULES FOR SILENCING GENE EXPRESSION IN EUKARYOTIC CELLS
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019084664A1 (en) * 2017-11-02 2019-05-09 The Governors Of The University Of Alberta Chemically-modified guide rnas to improve crispr-cas protein specificity
SG11202005147WA (en) 2017-12-05 2020-06-29 Vertex Pharma 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
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
WO2019150203A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US11976092B2 (en) 2018-02-09 2024-05-07 Ohio State Innovation Foundation RNA nanostructures, methods of making, and uses thereof
EP3765614A1 (en) * 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3092148A1 (en) * 2018-03-19 2019-09-26 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
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
WO2019215500A1 (en) 2018-05-11 2019-11-14 Crispr Therapeutics Ag Methods and compositions for treating cancer
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
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
US11833225B2 (en) * 2018-05-24 2023-12-05 Crispr Therapeutics Ag Methods and compositions for efficient gene deletion
CN112567036A (zh) * 2018-06-08 2021-03-26 因特利亚治疗公司 用于基因编辑的修饰的向导rna
CN112469834A (zh) * 2018-06-25 2021-03-09 生物纳米基因公司 Dna的标记
AU2019291918B2 (en) * 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3106162A1 (en) * 2018-07-13 2020-01-16 Allele Biotechnology And Pharmaceuticals, Inc. Methods of achieving high specificity of genome editing
US11389485B2 (en) * 2018-08-28 2022-07-19 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
WO2020047477A1 (en) * 2018-08-31 2020-03-05 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
WO2020154500A1 (en) 2019-01-23 2020-07-30 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
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
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
GB201903521D0 (en) 2019-03-14 2019-05-01 Tropic Biosciences Uk Ltd No title
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220249558A1 (en) 2019-04-30 2022-08-11 Crispr Therapeutics Ag Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
KR20220035877A (ko) * 2019-05-23 2022-03-22 보르 바이오파마 인크. Cd33 변형을 위한 조성물 및 방법
CN114375334B (zh) 2019-06-07 2026-03-20 斯克里贝治疗公司 工程化CasX系统
CN114258398A (zh) 2019-06-13 2022-03-29 总医院公司 工程化的人内源性病毒样颗粒及使用其递送至细胞的方法
KR20220034717A (ko) * 2019-07-19 2022-03-18 신테고 코포레이션 안정화된 crispr 복합체
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
KR20220047380A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cll1 변형을 위한 조성물 및 방법
KR20220047381A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cd123 변형을 위한 조성물 및 방법
JP2022548957A (ja) * 2019-09-19 2022-11-22 モデルナティエックス インコーポレイテッド キャップガイド及びｒｎａマッピングのためのその使用方法
CA3153197A1 (en) 2019-10-03 2021-04-08 Ryan T. Gill Crispr systems with engineered dual guide nucleic acids
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
KR20220079832A (ko) 2019-10-11 2022-06-14 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
AU2020379046B2 (en) 2019-11-08 2025-03-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy
WO2021107747A1 (ko) * 2019-11-28 2021-06-03 고려대학교 산학협력단 8-옥소구아닌을 포함하는 rna 간섭 유도 핵산, 8-옥소구아닌을 포함하는 마이크로rna와 결합하는 변형 핵산 및 이들의 이용
EP4028521A1 (en) * 2019-12-09 2022-07-20 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
EP4076647A1 (en) 2019-12-20 2022-10-26 CureVac AG Lipid nanoparticles for delivery of nucleic acids
US20230312635A1 (en) 2020-01-29 2023-10-05 Sumitomo Chemical Company, Limited Method for producing nucleic acid oligomer
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
WO2021193954A1 (ja) 2020-03-27 2021-09-30 住友化学株式会社 核酸オリゴマーの製造方法
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
BR112023001272A2 (pt) 2020-07-24 2023-04-04 Massachusetts Gen Hospital Partículas semelhantes a vírus aprimoradas e métodos de uso das mesmas para entrega às células
EP4204565A1 (en) 2020-08-28 2023-07-05 Vor Biopharma Inc. Compositions and methods for cll1 modification
JP2023540277A (ja) 2020-08-28 2023-09-22 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ１２３改変のための組成物および方法
US20240041932A1 (en) 2020-09-14 2024-02-08 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
JP2023543351A (ja) 2020-09-18 2023-10-13 アーティザン ディヴェロップメント ラブス インコーポレイテッド 効率的かつ特異的なゲノム編集のためのコンストラクト及びその使用
CN116209668A (zh) 2020-09-24 2023-06-02 住友化学株式会社 核酸寡聚物的制造方法
WO2022067240A1 (en) 2020-09-28 2022-03-31 Vor Biopharma, Inc. Compositions and methods for cd6 modification
US20230364146A1 (en) 2020-09-30 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
JP2023544826A (ja) * 2020-10-08 2023-10-25 ゲンコレ インコーポレイテッド ＣＲＩＳＰＲ／Ｃａｓ１２ｆ１（Ｃａｓ１４ａ１）システム効率化のためのエンジニアリングされたガイドＲＮＡおよびその用途
CA3199623A1 (en) 2020-10-27 2022-05-05 Vor Biopharma Inc. Compositions and methods for treating hematopoietic malignancy
US20240344058A1 (en) 2020-10-30 2024-10-17 Vor Biopharma Inc. Compositions and methods for bcma modification
WO2022104090A1 (en) 2020-11-13 2022-05-19 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
EP4256054A1 (en) 2020-12-03 2023-10-11 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
US20240366675A1 (en) 2020-12-31 2024-11-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
EP4298212A1 (en) 2021-02-25 2024-01-03 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
WO2022217086A1 (en) 2021-04-09 2022-10-13 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
WO2022266139A2 (en) 2021-06-14 2022-12-22 Graphite Bio, Inc. Methods to genetically engineer hematopoietic stem and progenitor cells for red cell 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
US20240352452A1 (en) * 2021-07-09 2024-10-24 The Brigham And Women's Hospital, Inc. Crispr-based protein barcoding and surface assembly
WO2023015182A1 (en) 2021-08-02 2023-02-09 Vor Biopharma Inc. Compositions and methods for gene modification
EP4395748A1 (en) 2021-09-03 2024-07-10 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
WO2023039586A1 (en) * 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
AU2022346785A1 (en) * 2021-09-14 2024-04-18 Agilent Technologies, Inc. Methods for using guide rnas with chemical modifications
EP4408991A2 (en) 2021-09-27 2024-08-07 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
JPWO2023054350A1 (enExample) 2021-09-28 2023-04-06
AU2022381173A1 (en) * 2021-11-03 2024-05-02 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
WO2023122508A2 (en) * 2021-12-23 2023-06-29 Mammoth Biosciences, Inc. Programmable nuclease-based assay improvements
WO2023164636A1 (en) 2022-02-25 2023-08-31 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
US20250388896A1 (en) 2022-03-01 2025-12-25 Celyntra Therapeutics Sa Composition and methods for transgene insertion
JPWO2023182274A1 (enExample) 2022-03-23 2023-09-28
EP4499829A2 (en) 2022-03-29 2025-02-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
JP2025511728A (ja) 2022-04-04 2025-04-16 ブイオーアール バイオファーマ インコーポレーテッド エピトープを操作することを媒介するための組成物及び方法
EP4526884A2 (en) 2022-05-20 2025-03-26 Artisan Development Labs, Inc. Systems and methods for assessing risk of genome editing events
US20260009051A1 (en) 2022-07-13 2026-01-08 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 (enExample) 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
KR20250092173A (ko) 2022-10-27 2025-06-23 스미또모 가가꾸 가부시끼가이샤 올리고뉴클레오티드의 제조 방법
KR20250153183A (ko) 2022-12-19 2025-10-24 카마우 테라퓨틱스, 인크. P53 결합 단백질인 53bp1의 개선된 펩타이드 저해제
WO2024138131A1 (en) * 2022-12-22 2024-06-27 Trustees Of Tufts College Expanding applications of zgtc alphabet in protein expression and gene editing
WO2024140737A1 (en) * 2022-12-26 2024-07-04 Huidagene Therapeutics Co., Ltd. Modified guide rna and uses thereof
KR20250128304A (ko) 2022-12-26 2025-08-27 스미또모 가가꾸 가부시끼가이샤 올리고뉴클레오티드의 제조 방법
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
AU2024233180A1 (en) 2023-03-08 2025-09-25 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
EP4689101A1 (en) * 2023-04-07 2026-02-11 Genentech, Inc. Modified guide rnas
KR20260007576A (ko) * 2023-04-28 2026-01-14 빔 테라퓨틱스, 인크. 변형된 가이드 rna
EP4713453A1 (en) 2023-05-15 2026-03-25 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013142578A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2014093712A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US20140273226A1 (en) 2013-03-15 2014-09-18 System Biosciences, Llc Crispr/cas systems for genomic modification and gene modulation

Family Cites Families (134)

* 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
US20070122906A1 (en) 2003-12-29 2007-05-31 Allan Mishra Method of culturing cells
US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
CN101784556B (zh) 2007-05-10 2014-07-09 安捷伦科技有限公司 用于rna合成的硫代碳保护基团
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
CN104284669A (zh) 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
EP2852602B1 (en) 2012-05-21 2020-08-12 Agilent Technologies, Inc. Method for conjugating oligonucleotides
PL4289948T3 (pl) 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
BR112015004995B1 (pt) 2012-09-07 2023-05-02 Sangamo Biosciences, Inc. Método para modificação do genoma de uma célula, uso de uma célula, semente ou planta obtida pelo referido método e nuclease de dedo de zinco sítio específica
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
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
TWI670004B (zh) 2012-09-07 2019-09-01 美商陶氏農業科學公司 用來產生植物之螢光激活細胞分選富增技術
EP2912175B1 (en) 2012-10-23 2018-08-22 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
MX363017B (es) 2012-11-01 2019-03-04 Factor Bioscience Inc Métodos y productos para la expresión de proteínas en células.
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
CN111321081A (zh) 2012-12-06 2020-06-23 合成基因组股份有限公司 具有不易变化的高光适应表型的藻类突变体
CN105142669B (zh) 2012-12-06 2018-07-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
PL2784162T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Opracowanie systemów, metod oraz zoptymalizowanych kompozycji przewodnikowych do manipulacji sekwencyjnej
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
US9970002B2 (en) 2012-12-12 2018-05-15 Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
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
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
PT2898075E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105658796B (zh) 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP3434776A1 (en) 2012-12-12 2019-01-30 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
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
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
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
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
EP2970997A1 (en) 2013-03-15 2016-01-20 Regents of the University of Minnesota Engineering plant genomes using crispr/cas systems
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
IL289396B2 (en) 2013-03-15 2023-12-01 The General Hospital Coporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
JP2016522679A (ja) 2013-04-04 2016-08-04 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
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
CA2908697C (en) 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
US9890393B2 (en) 2013-05-29 2018-02-13 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided CAS nuclease system
JP7065564B2 (ja) 2013-05-29 2022-05-12 セレクティス Ｃａｓ９ニッカーゼ活性を用いて正確なｄｎａ切断をもたらすための方法
CA2913869C (en) 2013-05-29 2023-01-24 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
MY197877A (en) 2013-06-04 2023-07-22 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
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20240172759A (ko) 2013-06-17 2024-12-10 더 브로드 인스티튜트, 인코퍼레이티드 간의 표적화 및 치료를 위한 ＣＲＩＳＰＲ­Ｃａｓ 시스템, 벡터 및 조성물의 전달 및 용도
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
WO2015006290A1 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Multiplex rna-guided genome engineering
CN105555132B (zh) 2013-07-10 2018-07-17 约瑟夫·A·玛吉朱布 Mrap2敲除
AU2014287397B2 (en) 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
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
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
MX363842B (es) 2013-08-22 2019-04-05 Du Pont Métodos para producir modificaciones genéticas en un genoma vegetal sin incorporar un marcador transgénico seleccionable, y composiciones de éstas.
WO2015033293A1 (en) 2013-09-04 2015-03-12 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
RU2668819C2 (ru) 2013-09-04 2018-10-02 ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи Быстрый направленный анализ сельскохозяйственных культур для определения донорной вставки
WO2015035034A1 (en) 2013-09-04 2015-03-12 Mice With Horns, Llc Materials and methods for correcting recessive mutations in animals
US20150071946A1 (en) 2013-09-06 2015-03-12 The Johns Hopkins University Tumor-specific retrotransposon insertions
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
EP3418379B1 (en) 2013-09-18 2020-12-09 Kymab Limited Methods, cells & organisms
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US20160251675A1 (en) 2013-10-08 2016-09-01 International Rice Research Institute Drought-Resistant Cereal Grasses and Related Materials and Methods
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
KR102269769B1 (ko) 2013-11-04 2021-06-28 코르테바 애그리사이언스 엘엘씨 최적 메이즈 유전자좌
WO2015066634A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal soybean loci
EP3066202B1 (en) 2013-11-04 2021-03-03 Dow AgroSciences LLC Optimal soybean loci
AR098283A1 (es) 2013-11-04 2016-05-26 Dow Agrosciences Llc Polinucleótido donante universal para reconocimiento de genes
EP3066110B1 (en) 2013-11-04 2021-12-29 Corteva Agriscience LLC Optimal maize loci
WO2015070062A1 (en) 2013-11-07 2015-05-14 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
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
WO2015089354A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
EP3653229A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
EP3080260B1 (en) 2013-12-12 2019-03-06 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
BR112016028023A2 (pt) 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
US20150344836A1 (en) 2014-05-30 2015-12-03 Ohio State Innovation Foundation Agrobacterium Strains for Plant Transformation and Related Materials and Methods
MX384887B (es) 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
KR102425438B1 (ko) 2014-06-23 2022-07-27 더 제너럴 하스피탈 코포레이션 서열결정에 의해 평가된 DSB의 게놈 전체에 걸친 비편향된 확인 (GUIDE-Seq)
US10501741B2 (en) 2014-06-25 2019-12-10 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
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3011024B1 (en) 2014-08-27 2018-10-10 New England Biolabs, Inc. Synthon formation
AU2015308910B2 (en) 2014-08-27 2017-12-07 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
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
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
CA3239747A1 (en) 2014-09-26 2016-03-31 Pioneer Hi-Bred International, Inc. Wheat ms1 polynucleotides, polypeptides, and methods of use
JP6921740B2 (ja) 2014-09-26 2021-08-18 フィリップ・モーリス・プロダクツ・ソシエテ・アノニム 硝酸同化経路の変化によるタバコ特異的ニトロソアミンの低減
WO2016073079A2 (en) 2014-09-26 2016-05-12 Yale University Compositions and methods for biocontainment of microorganisms
KR20170064540A (ko) 2014-09-26 2017-06-09 투 포어 가이즈, 인코포레이티드 합성 프로브의 나노포어 감지에 의한 표적 서열 검출
US20170354745A1 (en) 2014-10-09 2017-12-14 The Regents Of The University Of California Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
DK3204022T3 (da) 2014-10-09 2021-03-08 Celularity Inc Placenta-afledte adhærente celle-exosomer og anvendelser deraf
DK3212789T3 (da) 2014-10-31 2020-07-27 Massachusetts Inst Technology Massiv parallel kombinatorisk genetik til crispr
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
AU2015339743C1 (en) 2014-10-31 2021-04-22 The Trustees Of The University Of Pennsylvania Altering gene expression in modified T cells and uses thereof
US10900034B2 (en) 2014-12-03 2021-01-26 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
DK3390632T3 (da) 2015-12-18 2025-12-01 Danisco Us Inc Fremgangsmåder og sammensætninger til polymerase ii (pol-ii)-baseret guide-rna-ekspression
WO2017104404A1 (ja) 2015-12-18 2017-06-22 国立研究開発法人科学技術振興機構 遺伝子改変非ヒト生物、卵細胞、受精卵、及び標的遺伝子の改変方法
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
US11118194B2 (en) 2015-12-18 2021-09-14 The Regents Of The University Of California Modified site-directed modifying polypeptides and methods of use thereof
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013142578A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2014093712A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US20140273232A1 (en) 2012-12-12 2014-09-18 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US20140273226A1 (en) 2013-03-15 2014-09-18 System Biosciences, Llc Crispr/cas systems for genomic modification and gene modulation

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