JP2019517268A5 - - Google Patents

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JP2019517268A5
JP2019517268A5 JP2018564259A JP2018564259A JP2019517268A5 JP 2019517268 A5 JP2019517268 A5 JP 2019517268A5 JP 2018564259 A JP2018564259 A JP 2018564259A JP 2018564259 A JP2018564259 A JP 2018564259A JP 2019517268 A5 JP2019517268 A5 JP 2019517268A5
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crrna
synthetic
guide rna
guide
sequence
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JP7093728B2 (ja
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JP2018564259A 2016-06-08 2017-06-08 化学的に修飾されたガイドrnaを使用する高特異性ゲノム編集 Active JP7093728B2 (ja)

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US201662347553P 2016-06-08 2016-06-08
US62/347,553 2016-06-08
US15/493,129 2017-04-20
US15/493,129 US10767175B2 (en) 2016-06-08 2017-04-20 High specificity genome editing using chemically modified guide RNAs
PCT/US2017/036648 WO2017214460A1 (en) 2016-06-08 2017-06-08 High specificity genome editing using chemically modified guide rnas

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JP2019517268A5 true JP2019517268A5 (enExample) 2020-07-09
JP7093728B2 JP7093728B2 (ja) 2022-06-30

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JP2022098789A Active JP7431891B2 (ja) 2016-06-08 2022-06-20 化学的に修飾されたガイドrnaを使用する高特異性ゲノム編集

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US (2) US10767175B2 (enExample)
EP (1) EP3469084A4 (enExample)
JP (2) JP7093728B2 (enExample)
KR (1) KR102382772B1 (enExample)
CN (2) CN109563514B (enExample)
AU (1) AU2017277918B2 (enExample)
WO (1) WO2017214460A1 (enExample)

Families Citing this family (112)

* 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
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
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
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
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
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
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
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
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
WO2018136758A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hsd17b13 variants and uses thereof
BR112019017751A2 (pt) 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
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
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
MX2019012169A (es) 2017-04-11 2019-12-11 Regeneron Pharma Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b).
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
EP3663310A4 (en) 2017-08-04 2021-08-11 Peking University TALE RVD SPECIFICALLY RECOGNIZING A DNA BASE MODIFIED BY METHYLATION AND CORRESPONDING APPLICATION
US11624077B2 (en) 2017-08-08 2023-04-11 Peking University Gene knockout method
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
US12181452B2 (en) 2017-09-18 2024-12-31 Waters Technologies Corporation Use of vapor deposition coated flow paths for improved chromatography of metal interacting analytes
US11709156B2 (en) 2017-09-18 2023-07-25 Waters Technologies Corporation Use of vapor deposition coated flow paths for improved analytical analysis
US12180581B2 (en) 2017-09-18 2024-12-31 Waters Technologies Corporation Use of vapor deposition coated flow paths for improved chromatography of metal interacting analytes
US11709155B2 (en) 2017-09-18 2023-07-25 Waters Technologies Corporation Use of vapor deposition coated flow paths for improved chromatography of metal interacting analytes
JP7434151B2 (ja) 2017-10-11 2024-02-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Pnpla3 i148mの変異を発現している患者の肝疾患の治療におけるhsd17b13の阻害
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019175328A1 (en) 2018-03-14 2019-09-19 Imba - Institut Für Molekulare Biotechnologie Gmbh Bh4pathwayactivationandusethereoffortreatingcancer
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
KR20250134703A (ko) 2018-05-11 2025-09-11 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
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
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
US11389485B2 (en) 2018-08-28 2022-07-19 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
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
CN111349654B (zh) * 2018-12-20 2023-01-24 北京大学 使用加标签的向导rna构建体进行高效基因筛选的组合物和方法
CA3123981A1 (en) * 2018-12-20 2020-06-25 Peking University Compositions and methods for highly efficient genetic screening using barcoded guide rna constructs
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
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
KR20220035877A (ko) 2019-05-23 2022-03-22 보르 바이오파마 인크. Cd33 변형을 위한 조성물 및 방법
AU2020289581B2 (en) 2019-06-07 2024-11-21 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
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
KR20220047381A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cd123 변형을 위한 조성물 및 방법
KR20220047380A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cll1 변형을 위한 조성물 및 방법
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
AU2020379046B2 (en) 2019-11-08 2025-03-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy
EP4028521A1 (en) * 2019-12-09 2022-07-20 Caribou Biosciences, Inc. Crispr abasic restricted nucleotides and crispr accuracy via analogs
US11918936B2 (en) 2020-01-17 2024-03-05 Waters Technologies Corporation Performance and dynamic range for oligonucleotide bioanalysis through reduction of non specific binding
MX2022012683A (es) * 2020-04-09 2023-01-11 Verve Therapeutics Inc Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades.
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
CN111690720B (zh) * 2020-06-16 2021-06-15 山东舜丰生物科技有限公司 利用修饰的单链核酸进行靶核酸检测的方法
WO2022010286A1 (ko) * 2020-07-08 2022-01-13 경상국립대학교산학협력단 미세상동성 기반 말단 결합을 통한 유전자 교정에 이용되는 공여자 핵산 및 이의 용도
GB202010692D0 (en) * 2020-07-10 2020-08-26 Horizon Discovery Ltd RNA scaffolds
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
EP4189073A1 (en) * 2020-08-03 2023-06-07 The Board of Trustees of the Leland Stanford Junior University Gene correction for scid-x1 in long-term hematopoietic stem cells
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
US12352734B2 (en) 2020-09-24 2025-07-08 Waters Technologies Corporation Chromatographic hardware improvements for separation of reactive molecules
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
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
US20240366675A1 (en) 2020-12-31 2024-11-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CN114836399B (zh) * 2021-01-14 2024-07-05 天津大学 异鸟嘌呤脱氧核糖核苷酸的制备方法
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
EP4419672A2 (en) 2021-06-01 2024-08-28 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
EP4370676A2 (en) 2021-06-18 2024-05-22 Artisan Development Labs, Inc. Compositions and methods for targeting, editing or modifying human genes
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
WO2023015182A1 (en) 2021-08-02 2023-02-09 Vor Biopharma Inc. Compositions and methods for gene modification
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
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
CN114410752B (zh) * 2022-01-24 2024-06-25 华南师范大学 一种基于光控的CRISPR-Cas核酸检测试剂盒及检测方法
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
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
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2024168312A1 (en) 2023-02-09 2024-08-15 Vor Biopharma Inc. Methods for treating hematopoietic malignancy
CN116083536B (zh) * 2023-02-17 2025-12-23 之江实验室 一种前间区序列邻近基序的鉴定方法
EP4689101A1 (en) * 2023-04-07 2026-02-11 Genentech, Inc. Modified guide rnas
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
WO2025240795A1 (en) * 2024-05-15 2025-11-20 President And Fellows Of Harvard College End-modified grnas for improved base editing

Family Cites Families (144)

* 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
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
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
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
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
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
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
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
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.
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
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
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
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
PL2784162T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Opracowanie systemów, metod oraz zoptymalizowanych kompozycji przewodnikowych do manipulacji sekwencyjnej
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
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
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
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
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)
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering 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)
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
EP2970997A1 (en) 2013-03-15 2016-01-20 Regents of the University of Minnesota Engineering plant genomes using crispr/cas systems
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
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
WO2014165349A1 (en) 2013-04-04 2014-10-09 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
CA2908697C (en) 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
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
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
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
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
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
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
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
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
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
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
AU2014287397B2 (en) 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
CN105555132B (zh) 2013-07-10 2018-07-17 约瑟夫·A·玛吉朱布 Mrap2敲除
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.
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
WO2015033293A1 (en) 2013-09-04 2015-03-12 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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
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
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
EP3066202B1 (en) 2013-11-04 2021-03-03 Dow AgroSciences LLC Optimal soybean loci
WO2015066634A2 (en) 2013-11-04 2015-05-07 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
KR102269769B1 (ko) 2013-11-04 2021-06-28 코르테바 애그리사이언스 엘엘씨 최적 메이즈 유전자좌
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
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
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
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
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
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
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
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
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
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
US10323073B2 (en) 2014-03-20 2019-06-18 UNIVERSITé LAVAL CRISPR-based methods and products for increasing frataxin levels and uses thereof
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
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
AU2015308910B2 (en) 2014-08-27 2017-12-07 Caribou Biosciences, Inc. Methods for increasing Cas9-mediated engineering efficiency
EP3011024B1 (en) 2014-08-27 2018-10-10 New England Biolabs, Inc. Synthon formation
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
KR20170064540A (ko) 2014-09-26 2017-06-09 투 포어 가이즈, 인코포레이티드 합성 프로브의 나노포어 감지에 의한 표적 서열 검출
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
DK3204022T3 (da) 2014-10-09 2021-03-08 Celularity Inc Placenta-afledte adhærente celle-exosomer og anvendelser deraf
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
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
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
DK3212789T3 (da) 2014-10-31 2020-07-27 Massachusetts Inst Technology Massiv parallel kombinatorisk genetik til crispr
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
EP3786296A1 (en) * 2014-12-18 2021-03-03 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
LT3250691T (lt) 2015-01-28 2023-09-11 Caribou Biosciences, Inc. Crispr hibridiniai dnr/rnr polinukleotidai ir naudojimo būdai
US11414657B2 (en) 2015-06-29 2022-08-16 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
EP3159407A1 (en) 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
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
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
DK3390632T3 (da) 2015-12-18 2025-12-01 Danisco Us Inc Fremgangsmåder og sammensætninger til polymerase ii (pol-ii)-baseret guide-rna-ekspression
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression
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
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
IL267024B2 (en) 2016-12-08 2023-12-01 Intellia Therapeutics Inc Adapted leader RNAs for genomic editing

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