JP2016501036A5 - - Google Patents

Download PDF

Info

Publication number
JP2016501036A5
JP2016501036A5 JP2015549528A JP2015549528A JP2016501036A5 JP 2016501036 A5 JP2016501036 A5 JP 2016501036A5 JP 2015549528 A JP2015549528 A JP 2015549528A JP 2015549528 A JP2015549528 A JP 2015549528A JP 2016501036 A5 JP2016501036 A5 JP 2016501036A5
Authority
JP
Japan
Prior art keywords
nucleic acid
guide rna
sequence
eukaryotic cell
cas9
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2015549528A
Other languages
English (en)
Japanese (ja)
Other versions
JP6700788B2 (ja
JP2016501036A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2013/075317 external-priority patent/WO2014099744A1/en
Publication of JP2016501036A publication Critical patent/JP2016501036A/ja
Publication of JP2016501036A5 publication Critical patent/JP2016501036A5/ja
Application granted granted Critical
Publication of JP6700788B2 publication Critical patent/JP6700788B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2015549528A 2012-12-17 2013-12-16 Ｒｎａ誘導性ヒトゲノム改変 Active JP6700788B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261738355P 2012-12-17 2012-12-17
US61/738,355 2012-12-17
US201361779169P 2013-03-13 2013-03-13
US61/779,169 2013-03-13
PCT/US2013/075317 WO2014099744A1 (en) 2012-12-17 2013-12-16 Rna-guided human genome engineering

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018230081A Division JP2019076097A (ja) 2012-12-17 2018-12-07 Ｒｎａ誘導性ヒトゲノム改変

Publications (3)

Publication Number Publication Date
JP2016501036A JP2016501036A (ja) 2016-01-18
JP2016501036A5 true JP2016501036A5 (enExample) 2017-06-29
JP6700788B2 JP6700788B2 (ja) 2020-05-27

Family

ID=50979072

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2015549528A Active JP6700788B2 (ja) 2012-12-17 2013-12-16 Ｒｎａ誘導性ヒトゲノム改変
JP2018230081A Pending JP2019076097A (ja) 2012-12-17 2018-12-07 Ｒｎａ誘導性ヒトゲノム改変
JP2021000280A Pending JP2021048882A (ja) 2012-12-17 2021-01-04 Ｒｎａ誘導性ヒトゲノム改変
JP2023173277A Active JP7749631B2 (ja) 2012-12-17 2023-10-04 Ｒｎａ誘導性ヒトゲノム改変
JP2025158193A Pending JP2025175149A (ja) 2012-12-17 2025-09-24 Ｒｎａ誘導性ヒトゲノム改変

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2018230081A Pending JP2019076097A (ja) 2012-12-17 2018-12-07 Ｒｎａ誘導性ヒトゲノム改変
JP2021000280A Pending JP2021048882A (ja) 2012-12-17 2021-01-04 Ｒｎａ誘導性ヒトゲノム改変
JP2023173277A Active JP7749631B2 (ja) 2012-12-17 2023-10-04 Ｒｎａ誘導性ヒトゲノム改変
JP2025158193A Pending JP2025175149A (ja) 2012-12-17 2025-09-24 Ｒｎａ誘導性ヒトゲノム改変

Country Status (16)

Country Link
US (19) US9970024B2 (enExample)
EP (4) EP4481048A3 (enExample)
JP (5) JP6700788B2 (enExample)
KR (2) KR20220139433A (enExample)
CN (2) CN121022900A (enExample)
AU (5) AU2013363194B2 (enExample)
CA (2) CA3081054A1 (enExample)
DK (2) DK3553174T3 (enExample)
ES (2) ES2741951T3 (enExample)
IL (2) IL239326B2 (enExample)
MX (3) MX383326B (enExample)
MY (1) MY170059A (enExample)
RU (2) RU2699523C2 (enExample)
SG (3) SG10201704932UA (enExample)
WO (2) WO2014099744A1 (enExample)
ZA (2) ZA201504739B (enExample)

Families Citing this family (440)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8053191B2 (en) 2006-08-31 2011-11-08 Westend Asset Clearinghouse Company, Llc Iterative nucleic acid assembly using activation of vector-encoded traits
EP4039363A1 (en) 2010-11-12 2022-08-10 Gen9, Inc. Protein arrays and methods of using and making the same
IL280133B2 (en) 2010-11-12 2023-04-01 Gen9 Inc Methods and devices for the synthesis of nucleic acids
WO2016100976A2 (en) * 2014-12-19 2016-06-23 Apprise Bio, Inc. Methods for identifying multiple epitopes in selected sub-populations of cells
US11560585B2 (en) 2011-01-31 2023-01-24 Roche Sequencing Solutions, Inc. Methods of identifying multiple epitopes in cells
CN103703143B (zh) 2011-01-31 2016-12-14 爱普瑞斯生物公司 鉴定细胞中的多个表位的方法
EA201391373A1 (ru) 2011-03-23 2014-07-30 Пайонир Хай-Бред Интернэшнл, Инк. Способы получения сложного локуса трансгенных признаков
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP3594340B1 (en) 2011-08-26 2021-06-30 Gen9, Inc. Compositions and methods for high fidelity assembly of nucleic acids
JP2014531908A (ja) 2011-10-14 2014-12-04 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 構造アッセンブリによる配列決定
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US9637739B2 (en) * 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
AU2013251701A1 (en) 2012-04-24 2014-10-30 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
EP2847335B1 (en) 2012-04-25 2018-06-27 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
NZ714353A (en) 2012-05-25 2017-05-26 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US20150225734A1 (en) 2012-06-19 2015-08-13 Regents Of The University Of Minnesota Gene targeting in plants using dna viruses
JP6509727B2 (ja) 2012-06-25 2019-05-15 ギンゴー バイオワークス， インコーポレイテッド 核酸アセンブリおよび高処理シークエンシングのための方法
DE202013012597U1 (de) 2012-10-23 2017-11-21 Toolgen, Inc. Zusammensetzung zum Spalten einer Ziel-DNA, umfassend eine für die Ziel-DNA spezifische guide-RNA und eine Cas-Protein-codierende Nukleinsäure oder ein Cas-Protein, sowie deren Verwendung
AU2013355214B2 (en) * 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
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
EP4286403A3 (en) 2012-12-12 2024-02-14 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG11201504523UA (en) 2012-12-12 2015-07-30 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
EP2771468B1 (en) 2012-12-12 2015-02-11 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
PL2931898T3 (pl) 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2898075B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2940140B1 (en) 2012-12-12 2019-03-27 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
KR20220139433A (ko) * 2012-12-17 2022-10-14 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-가이드된 인간 게놈 조작
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
SG10201706741VA (en) 2013-02-20 2017-10-30 Regeneron Pharma Genetic modification of rats
EP2971184B1 (en) 2013-03-12 2019-04-17 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
CA2905432C (en) 2013-03-14 2021-09-14 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
CN105209624A (zh) * 2013-03-15 2015-12-30 明尼苏达大学董事会 采用CRISPR/Cas系统的植物基因组的工程改造
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US20140273230A1 (en) * 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
EP2970208A4 (en) * 2013-03-15 2016-11-30 Gen Hospital Corp SPECIFICITY INCREASES FOR RNA-MEDIATED CHANGES
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CN105518146B (zh) 2013-04-04 2022-07-15 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
BR112015026197B1 (pt) 2013-04-16 2022-12-06 Regeneron Pharmaceuticals, Inc Método para modificação marcada de um lócus genômico de interesse em uma célula de rato pluripotente
HK1223401A1 (zh) * 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
MY177814A (en) * 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
JP6665088B2 (ja) 2013-06-17 2020-03-13 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物
WO2014204729A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
SG11201600060VA (en) * 2013-07-10 2016-02-26 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CA2922089C (en) 2013-08-22 2022-07-05 E. I. Du Pont De Nemours And Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
KR20160060659A (ko) 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CN105637087A (zh) 2013-09-18 2016-06-01 科马布有限公司 方法、细胞与生物体
CN116836957A (zh) * 2013-10-17 2023-10-03 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
WO2015066119A1 (en) 2013-10-30 2015-05-07 North Carolina State University Compositions and methods related to a type-ii crispr-cas system in lactobacillus buchneri
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
SI3080279T1 (sl) 2013-12-11 2019-01-31 Regeneron Pharmaceuticals, Inc. Postopki in sestavki za ciljano spremembo genoma
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3080261B1 (en) 2013-12-12 2019-05-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
EP3079725B1 (en) 2013-12-12 2019-10-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
BR112016013547A2 (pt) 2013-12-12 2017-10-03 Broad Inst Inc Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
KR20160097327A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 유전자 산물, 구조 정보 및 유도성 모듈형 ｃａｓ 효소의 발현의 변경을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 방법
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
RS60492B1 (sr) 2014-02-11 2020-08-31 Univ Colorado Regents Multipleksirani genomski inženjering omogućen pomoću crispr
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
WO2015153780A1 (en) 2014-04-02 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
CA2944978C (en) 2014-04-08 2024-02-13 North Carolina State University Methods and compositions for rna-directed repression of transcription using crispr-associated genes
BR122023023211A2 (pt) 2014-04-28 2024-01-23 Recombinetics, Inc. Método de fazer edições de genes multiplex em uma célula de vertebrado ou de embrião primário não-humano
EP3149171A1 (en) 2014-05-30 2017-04-05 The Board of Trustees of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
ES3019688T3 (en) 2014-06-06 2025-05-21 Regeneron Pharma Methods and compositions for modifying a targeted locus
AU2015280120B2 (en) 2014-06-23 2017-05-25 Regeneron Pharmaceuticals, Inc. Nuclease-mediated DNA assembly
RU2771532C2 (ru) 2014-06-26 2022-05-05 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленных генетических модификаций и способы их применения
US10179932B2 (en) 2014-07-11 2019-01-15 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
BR112017000482A2 (pt) 2014-07-11 2017-11-07 Du Pont métodos para produzir uma planta mutante e para gerar uma planta, planta, semente, rna, métodos para produzir uma célula, para duplicar um fragmento gênico, para substituir uma primeira sequência promotora, para inserir um elemento regulador em uma sequência de nucleotídeos e para inserir um íntron em uma sequência de nucleotídeos, planta de milho e célula vegetal
EP3169776B1 (en) * 2014-07-14 2025-09-03 The Regents of The University of California Crispr/cas transcriptional modulation
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
KR20170036801A (ko) 2014-08-19 2017-04-03 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산의 프로빙 및 맵핑을 위한 ｒｎａ-가이드된 시스템
AU2015308910B2 (en) * 2014-08-27 2017-12-07 Caribou Biosciences, Inc. Methods for increasing Cas9-mediated engineering efficiency
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
US20170298450A1 (en) * 2014-09-10 2017-10-19 The Regents Of The University Of California Reconstruction of ancestral cells by enzymatic recording
WO2016040030A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
EP3998344A1 (en) * 2014-10-09 2022-05-18 Life Technologies Corporation Crispr oligonucleotides and gene editing
US20180250424A1 (en) 2014-10-10 2018-09-06 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
KR20170070136A (ko) 2014-10-15 2017-06-21 리제너론 파마슈티칼스 인코포레이티드 만능성 세포를 생성하거나 유지하는 방법 및 조성물
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
JP6779217B2 (ja) 2014-10-24 2020-11-04 アヴェクタス リミテッド 細胞原形質膜を越える送達方法
WO2016065364A1 (en) * 2014-10-24 2016-04-28 Life Technologies Corporation Compositions and methods for enhancing homologous recombination
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
CN113444747B (zh) 2014-11-21 2025-02-11 瑞泽恩制药公司 使用成对向导rna进行靶向遗传修饰的方法和组合物
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
CN105695485B (zh) * 2014-11-27 2020-02-21 中国科学院上海生命科学研究院 一种用于丝状真菌Crispr-Cas系统的Cas9编码基因及其应用
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
US9888673B2 (en) 2014-12-10 2018-02-13 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
US10196613B2 (en) 2014-12-19 2019-02-05 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
CN107208113A (zh) 2014-12-19 2017-09-26 瑞泽恩制药公司 用于通过单步多重靶向进行靶向遗传修饰的方法和组合物
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
WO2016114972A1 (en) 2015-01-12 2016-07-21 The Regents Of The University Of California Heterodimeric cas9 and methods of use thereof
WO2016123243A1 (en) 2015-01-28 2016-08-04 The Regents Of The University Of California Methods and compositions for labeling a single-stranded target nucleic acid
EP3798302A1 (fr) * 2015-01-29 2021-03-31 Meiogenix Procede pour induire des recombinaisons meiotiques ciblees
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
US11046952B2 (en) * 2015-03-16 2021-06-29 The Broad Institute, Inc. Encoding of DNA vector identity via iterative hybridization detection of a barcode transcript
WO2016160389A1 (en) 2015-03-27 2016-10-06 E I Du Pont De Nemours And Company Soybean u6 small nuclear rna gene promoters and their use in constitutive expression of small rna genes in plants
JP6873911B2 (ja) 2015-04-06 2021-05-19 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 初代細胞において標的核酸の遺伝子調節を誘導するためにインビトロで行う方法
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
HK1254190A1 (zh) 2015-05-08 2019-07-12 President And Fellows Of Harvard College 通用供体干细胞和相关方法
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
US11535871B2 (en) * 2015-05-14 2022-12-27 University Of Southern California Optimized gene editing utilizing a recombinant endonuclease system
EA201792663A1 (ru) 2015-05-29 2018-04-30 Норт Каролина Стейт Юниверсити Способы скрининга бактерий, архей, водорослей и дрожжей с использованием нуклеиновых кислот crispr
WO2016196282A1 (en) 2015-05-29 2016-12-08 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
EA037359B1 (ru) 2015-06-01 2021-03-17 Тэмпл Юниверсити - Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
JP7396783B2 (ja) 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
KR102902492B1 (ko) 2015-06-15 2025-12-18 노쓰 캐롤라이나 스테이트 유니버시티 핵산 및 rna-기반 항미생물제를 효율적으로 전달하기 위한 방법 및 조성물
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
RU2752834C2 (ru) 2015-06-18 2021-08-09 Те Брод Инститьют, Инк. Мутации фермента crispr, уменьшающие нецелевые эффекты
JP2018519811A (ja) 2015-06-29 2018-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ｃｒｉｓｐｒ ｒｎａ及び修飾単一ｃｒｉｓｐｒ ｒｎａならびにその使用
CA2992580C (en) 2015-07-15 2022-09-20 Rutgers, The State University Of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
GB2595063B (en) 2015-07-31 2022-03-09 Univ Minnesota Modified cells and methods of therapy
AU2016301196B2 (en) 2015-08-06 2022-09-08 Dana-Farber Cancer Institute, Inc. Tunable endogenous protein degradation
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
HK1257676A1 (zh) 2015-08-28 2019-10-25 The General Hospital Corporation 工程化 crispr-cas 9 核酸酶
CN107922949A (zh) * 2015-08-31 2018-04-17 安捷伦科技有限公司 用于通过同源重组的基于crispr/cas的基因组编辑的化合物和方法
US20180251789A1 (en) * 2015-09-04 2018-09-06 Massachusetts Institute Of Technology Multilayer genetic safety kill circuits based on single cas9 protein and multiple engineered grna in mammalian cells
WO2017049129A2 (en) * 2015-09-18 2017-03-23 President And Fellows Of Harvard College Methods of making guide rna
WO2017053312A1 (en) * 2015-09-21 2017-03-30 The Regents Of The University Of California Compositions and methods for target nucleic acid modification
EP3786294A1 (en) 2015-09-24 2021-03-03 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US11286480B2 (en) 2015-09-28 2022-03-29 North Carolina State University Methods and compositions for sequence specific antimicrobials
AU2016335698B2 (en) * 2015-10-08 2022-12-01 President And Fellows Of Harvard College Multiplexed genome editing
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
WO2017074962A1 (en) * 2015-10-30 2017-05-04 Brandeis University Modified cas9 compositions and methods of use
HK1252878A1 (zh) 2015-11-03 2019-06-06 President And Fellows Of Harvard College 用於含有基质的三维核酸的体积成像的方法和设备
BR112018009515A2 (pt) * 2015-11-11 2018-11-06 Univ Minnesota sistema e métodos de biocontenção/biocontrole
CN106893739A (zh) 2015-11-17 2017-06-27 香港中文大学 用于靶向基因操作的新方法和系统
AU2016355178B9 (en) 2015-11-19 2019-05-30 Massachusetts Institute Of Technology Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity
KR20250044471A (ko) 2015-11-30 2025-03-31 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
JP7449646B2 (ja) 2015-12-30 2024-03-14 アヴェクタス リミテッド 細胞および組織への遺伝子編集タンパク質および組成物の、ベクターなしでの送達
EA201891619A1 (ru) 2016-01-11 2019-02-28 Те Борд Оф Трастиз Оф Те Лилэнд Стэнфорд Джуниор Юниверсити Химерные белки и способы регулирования экспрессии генов
MY194127A (en) 2016-01-11 2022-11-14 Univ Leland Stanford Junior Chimeric proteins and methods of immunotherapy
CN105624187A (zh) * 2016-02-17 2016-06-01 天津大学 酿酒酵母基因组定点突变的方法
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
WO2017151444A1 (en) 2016-02-29 2017-09-08 Agilent Technologies, Inc. Methods and compositions for blocking off-target nucleic acids from cleavage by crispr proteins
WO2017161043A1 (en) 2016-03-16 2017-09-21 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
CA3016504A1 (en) 2016-03-23 2017-09-28 Dana-Farber Cancer Institute, Inc. Methods for enhancing the efficiency of gene editing
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
AU2017253089B2 (en) 2016-04-19 2023-07-20 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
CN116200465A (zh) 2016-04-25 2023-06-02 哈佛学院董事及会员团体 用于原位分子检测的杂交链反应方法
EP3450570B1 (en) * 2016-04-28 2021-03-10 Industry-Academic Cooperation Foundation, Yonsei University Method for evaluating, in vivo, activity of rna-guided nuclease in high-throughput manner
CN109475109B (zh) 2016-05-20 2021-10-29 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
EP3910068A1 (en) * 2016-05-24 2021-11-17 The Translational Genomics Research Institute Molecular tagging methods and sequencing libraries
CN116064529A (zh) 2016-06-02 2023-05-05 西格马-奥尔德里奇有限责任公司 使用可编程dna结合蛋白增强靶向基因组修饰
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
US12214056B2 (en) 2016-07-19 2025-02-04 Duke University Therapeutic applications of CPF1-based genome editing
JP7179715B2 (ja) * 2016-07-27 2022-11-29 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 核酸送達用の崩壊性細胞透過性複合体
CN109563505A (zh) * 2016-07-28 2019-04-02 帝斯曼知识产权资产管理有限公司 用于真核细胞的组装系统
WO2018023014A1 (en) 2016-07-29 2018-02-01 Regeneron Pharmaceuticals, Inc. Mice comprising mutations resulting in expression of c-truncated fibrillin-1
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
CA3032699A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 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
WO2018049009A2 (en) * 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
EP3523426A4 (en) 2016-09-30 2020-01-22 The Regents of The University of California RNA GUIDED NUCLEIC ACID MODIFYING ENZYMES AND METHOD FOR USE THEREOF
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
US11306324B2 (en) 2016-10-14 2022-04-19 President And Fellows Of Harvard College AAV delivery of nucleobase editors
CA3040481A1 (en) 2016-10-14 2018-04-19 The General Hospital Corporation Epigenetically regulated site-specific nucleases
GB2604416B (en) 2016-10-18 2023-03-15 Univ Minnesota Tumor infiltating lymphocytes and methods of therapy
WO2018089386A1 (en) 2016-11-11 2018-05-17 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action
EP4520828A3 (en) 2016-11-15 2025-07-09 The Schepens Eye Research Institute, Inc. Compositions and methods for the treatment of aberrant angiogenesis
KR20210035022A (ko) 2016-12-14 2021-03-31 리간달 인코포레이티드 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
JP7465090B2 (ja) 2016-12-22 2024-04-10 アヴェクタス リミテッド 可逆的透過処理によるベクターフリー細胞内送達法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018129129A1 (en) 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
JP7094963B2 (ja) 2017-01-23 2022-07-04 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 Ｈｓｄ１７ｂ１３バリアントおよびその使用
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
JP7227912B2 (ja) 2017-02-08 2023-02-24 ダナ－ファーバー キャンサー インスティテュート，インコーポレイテッド キメラ抗原受容体の調節
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
WO2018195129A1 (en) 2017-04-17 2018-10-25 University Of Maryland, College Park Embryonic cell cultures and methods of using the same
SG11201909797QA (en) 2017-04-20 2019-11-28 Egenesis Inc Methods for generating genetically modified animals
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11834665B2 (en) 2017-05-10 2023-12-05 Regents Of The University Of Minnesota Programmable transcription factors and methods
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
AU2018273986A1 (en) 2017-05-25 2019-12-12 The General Hospital Corporation Bipartite base editor (BBE) architectures and type-II-C-Cas9 zinc finger editing
ES2875579T3 (es) 2017-06-06 2021-11-10 Zymergen Inc Plataforma de ingeniería genómica de HTP para mejorar Escherichia coli
WO2018226900A2 (en) 2017-06-06 2018-12-13 Zymergen Inc. A htp genomic engineering platform for improving fungal strains
CN111065736A (zh) * 2017-06-07 2020-04-24 国立大学法人东京大学 针对颗粒状角膜变性症的基因治疗药物
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
SG11201912179SA (en) 2017-06-15 2020-01-30 Univ California Targeted non-viral dna insertions
EP4527460A3 (en) 2017-06-21 2025-06-11 ProKidney Immunoprivileged bioactive renal cells for the treatment of kidney disease
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
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
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
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
JP2020533957A (ja) 2017-07-31 2020-11-26 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Ｒｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｃｒｉｓｐｒリポーター非ヒト動物およびその使用
KR20200032117A (ko) 2017-07-31 2020-03-25 리제너론 파마슈티칼스 인코포레이티드 생체 내에서 외인성 공여체 핵산과의 CRISPR/Cas-유도된 재조합의 평가
EP3585159B1 (en) 2017-07-31 2025-05-14 Regeneron Pharmaceuticals, Inc. Method of testing the ability of a crispr/cas9 nuclease to modify a target genomic locus in vivo, by making use of a cas-transgenic mouse or rat which comprises a cas9 expression cassette in its genome
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR102544051B1 (ko) 2017-09-29 2023-06-16 리제너론 파마슈티칼스 인코포레이티드 인간화된 ttr 유전자좌를 포함하는 비인간 동물 및 사용 방법
IL311170A (en) 2017-09-29 2024-04-01 Intellia Therapeutics Inc Compositions and methods for ttr gene editing and treating attr amyloidosis
EA202090873A1 (ru) 2017-09-29 2020-08-17 Интеллиа Терапьютикс, Инк. Полинуклеотиды, композиции и способы редактирования генома
AU2018338967B2 (en) 2017-09-29 2025-04-24 Intellia Therapeutics, Inc. In vitro method of mRNA delivery using lipid nanoparticles
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
JP2021500036A (ja) 2017-10-16 2021-01-07 ザ ブロード インスティテュート, インコーポレーテッドThe Broad Institute, Inc. アデノシン塩基編集因子の使用
IL305485B2 (en) 2017-10-27 2025-10-01 Univ California Targeted replacement of endogenous T cell receptors
WO2019099560A1 (en) 2017-11-14 2019-05-23 The Schepens Eye Research Institute, Inc. Runx1 inhibition for treatment of proliferative vitreoretinopathy and conditions associated with epithelial to mesenchymal transition
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3737690A1 (en) 2018-01-12 2020-11-18 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOMIC ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYOTES
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
WO2019165168A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
MX2020010974A (es) 2018-04-19 2021-01-20 Univ California Composiciones y métodos para la edición de genes.
CA3100019A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019229722A1 (en) 2018-06-01 2019-12-05 Avectas Limited Cell engineering platform
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CN112513270B (zh) 2018-07-13 2025-02-25 加利福尼亚大学董事会 基于逆转录转座子的递送媒介物及其使用方法
JP2021531804A (ja) 2018-07-31 2021-11-25 インテリア セラピューティクス，インコーポレイテッド 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法
AU2019314433A1 (en) 2018-07-31 2021-03-25 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
CN110678553B (zh) * 2018-08-21 2023-08-11 中国医学科学院血液病医院(中国医学科学院血液学研究所) 在哺乳动物干细胞中进行基因组编辑的方法
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California Variant type v crispr/cas effector polypeptides and methods of use thereof
TW202028460A (zh) 2018-09-28 2020-08-01 美商英特利亞醫療公司 用於乳酸脫氫酶(ldha)基因編輯之組合物及方法
WO2020072254A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for killing target cells
WO2020072253A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for screening for variant cells
WO2020072250A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for genome modification and alteration of expression
WO2020072248A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
AU2019362879A1 (en) 2018-10-16 2021-05-27 Intellia Therapeutics, Inc. Compositions and methods for immunotherapy
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020092704A1 (en) 2018-10-31 2020-05-07 Zymergen Inc. Multiplexed deterministic assembly of dna libraries
JP2022505440A (ja) * 2018-11-01 2022-01-14 キージーン ナムローゼ フェンノートシャップ 植物細胞におけるＣＲＩＳＰＲ／Ｃａｓゲノム編集のためのデュアルガイドＲＮＡ
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12460189B2 (en) 2018-11-09 2025-11-04 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
BR112021011372A2 (pt) 2018-12-14 2021-08-31 Pioneer Hi-Bred International, Inc. Novos sistemas crispr-cas para edição de genoma
CN113423831B (zh) 2018-12-20 2023-03-10 瑞泽恩制药公司 核酸酶介导的重复扩增
US20220106584A1 (en) 2019-01-14 2022-04-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and kits for generating and selecting a variant of a binding protein with increased binding affinity and/or specificity
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT
WO2020163856A1 (en) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Modified mitochondrion and methods of use thereof
WO2020181101A1 (en) 2019-03-07 2020-09-10 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
US11053515B2 (en) 2019-03-08 2021-07-06 Zymergen Inc. Pooled genome editing in microbes
KR20210136997A (ko) 2019-03-08 2021-11-17 지머젠 인코포레이티드 미생물에서 반복적 게놈 편집
CN113711046B (zh) 2019-03-18 2023-08-29 瑞泽恩制药公司 用于揭示与Tau聚集相关的基因脆弱性的CRISPR/Cas脱落筛选平台
US11845931B2 (en) 2019-03-18 2023-12-19 Regeneron Pharmaceuticals, Inc. CRISPR/Cas screening platform to identify genetic modifiers of tau seeding or aggregation
KR20210143230A (ko) 2019-03-19 2021-11-26 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
JP7636338B2 (ja) 2019-03-28 2025-02-26 インテリア セラピューティクス，インコーポレイテッド コルチコステロイドを含む、ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスを治療するための組成物および方法、またはその使用
CN113874076A (zh) 2019-03-28 2021-12-31 因特利亚治疗公司 包括ttr向导rna和对rna引导的dna结合剂进行编码的多核苷酸的组合物和方法
JP2022527302A (ja) 2019-03-28 2022-06-01 インテリア セラピューティクス，インコーポレイテッド ポリペプチド発現のためのポリヌクレオチド、組成物、および方法
MX2021011956A (es) 2019-04-03 2021-12-15 Regeneron Pharma Metodos y composiciones para la insercion de secuencias codificantes de anticuerpos en un locus de puerto seguro.
IL286917B (en) 2019-04-04 2022-09-01 Regeneron Pharma Methods for scar-free insertion of targeted modifications into targeted vectors
IL286905B2 (en) 2019-04-04 2024-06-01 Regeneron Pharma Non-human animals containing the human coagulation factor 12 locus
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020286382A1 (en) 2019-06-04 2021-11-04 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus with a beta-slip mutation and methods of use
TW202112229A (zh) 2019-06-07 2021-04-01 美商雷傑納榮製藥公司 包含人化白蛋白基因座之非人的動物
JP7664854B2 (ja) 2019-06-14 2025-04-18 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 タウオパチーのモデル
CA3152288A1 (en) 2019-08-27 2021-03-04 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna
KR20220062079A (ko) 2019-09-13 2022-05-13 리제너론 파마슈티칼스 인코포레이티드 지질 나노입자에 의해 전달되는 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
US20230025039A1 (en) 2019-09-20 2023-01-26 The Broad Institute, Inc. Novel type vi crispr enzymes and systems
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021082830A1 (zh) * 2019-11-01 2021-05-06 中国科学院遗传与发育生物学研究所 靶向性修饰植物基因组序列的方法
BR112022008861A2 (pt) 2019-11-08 2022-08-23 Regeneron Pharma Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
CN114945273B (zh) 2019-11-29 2025-08-12 巴斯夫欧洲公司 增加植物对抗真菌感染的抗性
CA3161898A1 (en) 2019-12-19 2021-06-24 Basf Se Increasing space-time-yield, carbon-conversion-efficiency and carbon substrate flexibility in the production of fine chemicals
WO2021122528A1 (en) 2019-12-20 2021-06-24 Basf Se Decreasing toxicity of terpenes and increasing the production potential in micro-organisms
US11060141B1 (en) 2019-12-23 2021-07-13 Stilla Technologies Multiplex drop-off digital polymerase chain reaction methods
AU2021216451A1 (en) * 2020-02-07 2022-09-29 Intellia Therapeutics, Inc. Compositions and methods for kallikrein ( KLKB1) gene editing
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
WO2021191678A1 (en) 2020-03-23 2021-09-30 Avectas Limited Engineering of dendritic cells for generation of vaccines against sars-cov-2
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US12057197B2 (en) 2020-04-03 2024-08-06 Creyon Bio, Inc. Oligonucleotide-based machine learning
EP4133069A2 (en) 2020-04-08 2023-02-15 Astrazeneca AB Compositions and methods for improved site-specific modification
WO2021222287A2 (en) 2020-04-28 2021-11-04 Intellia Therapeutics, Inc. Methods of in vitro cell delivery
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2022008935A1 (en) 2020-07-10 2022-01-13 Horizon Discovery Limited Method for producing genetically modified cells
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
TW202218686A (zh) 2020-09-09 2022-05-16 美商維泰克斯製藥公司 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之組合物及方法
US10947552B1 (en) 2020-09-30 2021-03-16 Alpine Roads, Inc. Recombinant fusion proteins for producing milk proteins in plants
IL301396A (en) 2020-09-30 2023-05-01 Nobell Foods Inc Recombinant milk proteins and food compositions comprising the same
US10894812B1 (en) 2020-09-30 2021-01-19 Alpine Roads, Inc. Recombinant milk proteins
WO2022087235A1 (en) 2020-10-21 2022-04-28 Massachusetts Institute Of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
US20230414648A1 (en) 2020-11-06 2023-12-28 Vertex Pharmaceuticals Incorporated Compositions and Methods for Treatment of DM1 with SLUCAS9 and SACAS9
EP4256052A1 (en) 2020-12-02 2023-10-11 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
WO2022125982A1 (en) 2020-12-11 2022-06-16 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
CA3206284A1 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
AU2021410751A1 (en) 2020-12-23 2023-07-13 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
US20240067954A1 (en) 2021-01-05 2024-02-29 Horizon Discovery Limited Method for producing genetically modified cells
CN117203319A (zh) 2021-02-01 2023-12-08 阿维塔斯有限公司 递送平台
JP2024505678A (ja) 2021-02-08 2024-02-07 インテリア セラピューティクス，インコーポレイテッド 免疫療法のためのリンパ球活性化遺伝子３（ｌａｇ３）組成物及び方法
JP2024505672A (ja) 2021-02-08 2024-02-07 インテリア セラピューティクス，インコーポレイテッド 免疫療法のためのナチュラルキラー細胞受容体２ｂ４組成物及び方法
WO2022170193A2 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy
WO2022182959A1 (en) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
EP4298222A1 (en) 2021-02-26 2024-01-03 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/sacas9
EP4320234A2 (en) 2021-04-07 2024-02-14 Astrazeneca AB Compositions and methods for site-specific modification
AU2022253891A1 (en) * 2021-04-07 2023-08-24 Century Therapeutics, Inc. Gene transfer vectors and methods of engineering cells
WO2022221696A1 (en) 2021-04-17 2022-10-20 Intellia Therapeutics, Inc. Inhibitors of dna-dependent protein kinase and compositions and uses thereof
BR112023021477A2 (pt) 2021-04-17 2024-01-30 Intellia Therapeutics Inc Composições de nanopartículas lipídicas
KR20240017793A (ko) 2021-04-17 2024-02-08 인텔리아 테라퓨틱스, 인크. 지질 나노입자 조성물
WO2022229851A1 (en) 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions and methods for using slucas9 scaffold sequences
WO2022234519A1 (en) 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions and methods for using sacas9 scaffold sequences
WO2022240846A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4347826A1 (en) 2021-06-02 2024-04-10 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
WO2023028471A1 (en) 2021-08-24 2023-03-02 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
WO2023039444A2 (en) 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy
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
EP4408996A2 (en) 2021-09-30 2024-08-07 Astrazeneca AB Use of inhibitors to increase efficiency of crispr/cas insertions
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados y sistemas de compuerta lógica.
CN118119702A (zh) 2021-10-14 2024-05-31 隆萨销售股份有限公司 用于细胞外囊泡产生的经修饰的生产者细胞
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
EP4426338A2 (en) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Cd38 compositions and methods for immunotherapy
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
TW202325848A (zh) 2021-11-03 2023-07-01 美商英特利亞醫療公司 用於基因體編輯之多核苷酸、組合物及方法
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
EP4457342A1 (en) 2021-12-29 2024-11-06 Bristol-Myers Squibb Company Generation of landing pad cell lines
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US20250230412A1 (en) 2022-02-01 2025-07-17 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023150620A1 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
EP4490291A1 (en) 2022-03-08 2025-01-15 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exons for treatment of duchenne muscular dystrophy
WO2023172927A1 (en) 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
US20250302998A1 (en) 2022-05-09 2025-10-02 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
EP4525892A1 (en) 2022-05-19 2025-03-26 Lyell Immunopharma, Inc. Polynucleotides targeting nr4a3 and uses thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
JP2025521154A (ja) 2022-05-31 2025-07-08 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 Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
WO2024020352A1 (en) 2022-07-18 2024-01-25 Vertex Pharmaceuticals Incorporated Tandem guide rnas (tg-rnas) and their use in genome editing
CA3262406A1 (en) 2022-07-18 2024-01-25 Renagade Therapeutics Management Inc. Gene editing components, systems and methods of use
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
JP2025525745A (ja) 2022-08-05 2025-08-07 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 Ｔｄｐ－４３の凝集耐性バリアント
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
IL319122A (en) 2022-09-28 2025-04-01 Regeneron Pharma Different antibody-resistant receptors improve cell-based therapies
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4615424A1 (en) 2022-11-10 2025-09-17 Sail Biomedicines, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
KR20250124819A (ko) 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
KR20250160369A (ko) 2023-01-27 2025-11-12 리제너론 파마슈티칼스 인코포레이티드 변형된 랩도바이러스 당단백질 및 이의 용도
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
TW202503051A (zh) 2023-03-07 2025-01-16 美商英特利亞醫療公司 用於免疫療法之ｃｉｓｈ組合物及方法
CN121127605A (zh) 2023-03-29 2025-12-12 阿斯利康(瑞典)有限公司 使用抑制剂来提高CRISPR/Cas插入的效率
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024275548A1 (en) 2023-05-19 2026-01-08 Intellia Therapeutics, Inc. Ionizable amine lipids
WO2024259309A1 (en) 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders
US20250002946A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods And Compositions For Increasing Homology-Directed Repair
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
TW202513801A (zh) 2023-07-28 2025-04-01 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入
WO2025027165A1 (en) 2023-08-01 2025-02-06 Basf Plant Science Company Gmbh Increased resistance by expression of an ics protein
WO2025027166A1 (en) 2023-08-01 2025-02-06 Basf Plant Science Company Gmbh Increased resistance by expression of msbp1 protein
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
TW202521564A (zh) 2023-08-14 2025-06-01 美商英特利亞醫療公司 用於基於細胞之療法的ｃｄ７０ ｃａｒ－ｔ組合物及方法
TW202515992A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對轉形生長因子β受體２型（ＴＧＦβＲ２）進行基因修飾之組合物及方法
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025049481A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025078978A1 (en) 2023-10-09 2025-04-17 Avectas Limited Transfection of cells via reversible permeabilization
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025111340A1 (en) * 2023-11-20 2025-05-30 William Marsh Rice University Engineered extrachromosomal nucleic acids and methods of use thereof
WO2025149984A2 (en) 2024-01-12 2025-07-17 Avectas Limited Delivery platform with flow-through system (fts)
WO2025149983A2 (en) 2024-01-12 2025-07-17 Avectas Limited Delivery platform with integrated non-viral transfection and cell processing
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025162985A1 (en) 2024-01-30 2025-08-07 Basf Plant Science Company Gmbh Increased plant disease resistance by expression of a glycine-rich protein
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025202473A1 (en) 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025259669A1 (en) 2024-06-10 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and systems for characterizing modified oligonucleotides
US20250388890A1 (en) 2024-06-20 2025-12-25 Regeneron Pharmaceuticals, Inc. ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6039784A (en) 1997-03-12 2000-03-21 Hoeganaes Corporation Iron-based powder compositions containing green strength enhancing lubricants
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US7090976B2 (en) 1999-11-10 2006-08-15 Rigel Pharmaceuticals, Inc. Methods and compositions comprising Renilla GFP
US6203986B1 (en) 1998-10-22 2001-03-20 Robert H. Singer Visualization of RNA in living cells
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
DK2927318T3 (da) 2003-08-08 2020-08-03 Sangamo Therapeutics Inc Fremgangsmåde og sammensætninger til målrettet spaltning og rekombination
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
US20050233994A1 (en) * 2004-04-16 2005-10-20 Ajamete Kaykas Methods and vectors for expressing siRNA
PL2325332T3 (pl) 2005-08-26 2013-03-29 Dupont Nutrition Biosci Aps Zastosowanie genów związanych z CRISPR (genów CAS)
BRPI0808704B1 (pt) 2007-03-02 2022-01-18 Dupont Nutrition Biosciences Aps Método para gerar uma cultura inicial compreendendo pelo menos duas cepas variantes resistentes a bacteriófagos, cultura iniciadora e método de fermentação
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
EP2414718B1 (en) 2009-03-31 2014-03-26 Nippon Steel & Sumitomo Metal Corporation Threaded joint for pipes
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
DK2569425T3 (en) 2010-05-10 2016-10-03 Univ California Endoribonuclease COMPOSITIONS AND METHODS OF USE THEREOF
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
US20140113376A1 (en) * 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013126794A1 (en) 2012-02-24 2013-08-29 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
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
NZ714353A (en) 2012-05-25 2017-05-26 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
HK1207111A1 (en) 2012-08-03 2016-01-22 加利福尼亚大学董事会 Methods and compositions for controlling gene expression by rna processing
DE202013012597U1 (de) 2012-10-23 2017-11-21 Toolgen, Inc. Zusammensetzung zum Spalten einer Ziel-DNA, umfassend eine für die Ziel-DNA spezifische guide-RNA und eine Cas-Protein-codierende Nukleinsäure oder ein Cas-Protein, sowie deren Verwendung
AU2013355214B2 (en) 2012-12-06 2017-06-15 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
SG11201504523UA (en) 2012-12-12 2015-07-30 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP2940140B1 (en) 2012-12-12 2019-03-27 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions 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
EP4286403A3 (en) * 2012-12-12 2024-02-14 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
EP2771468B1 (en) 2012-12-12 2015-02-11 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
PL2931898T3 (pl) * 2012-12-12 2016-09-30 Le Cong Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2898075B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
KR20220139433A (ko) 2012-12-17 2022-10-14 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-가이드된 인간 게놈 조작
CA2905432C (en) 2013-03-14 2021-09-14 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
MY177814A (en) * 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
JP6665088B2 (ja) 2013-06-17 2020-03-13 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物
AU2014287393B2 (en) * 2013-07-09 2020-10-22 President And Fellows Of Harvard College Multiplex RNA-guided genome engineering
SG11201600060VA (en) * 2013-07-10 2016-02-26 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
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
EP4219699A1 (en) * 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CA2936312A1 (en) * 2014-01-08 2015-07-16 President And Fellows Of Harvard College Rna-guided gene drives
AU2016335698B2 (en) * 2015-10-08 2022-12-01 President And Fellows Of Harvard College Multiplexed genome editing
US11746349B2 (en) * 2016-02-09 2023-09-05 President And Fellows Of Harvard College DNA-guided gene editing and regulation

Similar Documents

Publication Publication Date Title
JP2016501036A5 (enExample)
US11512325B2 (en) RNA-guided human genome engineering
HK40014881B (en) Rna-guided human genome engineering
NZ709429B2 (en) Rna-guided human genome engineering
HK1212376B (en) Rna-guided human genome engineering