AU2014228981B2 - Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing - Google Patents

Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing Download PDF

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AU2014228981B2
AU2014228981B2 AU2014228981A AU2014228981A AU2014228981B2 AU 2014228981 B2 AU2014228981 B2 AU 2014228981B2 AU 2014228981 A AU2014228981 A AU 2014228981A AU 2014228981 A AU2014228981 A AU 2014228981A AU 2014228981 B2 AU2014228981 B2 AU 2014228981B2
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nucleotides
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AU2014228981A1 (en
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Yanfang FU
J. Keith Joung
Morgan Maeder
Jeffry D. Sander
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General Hospital Corp
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AU2014228981A 2013-03-15 2014-03-14 Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing Active AU2014228981B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2020201465A AU2020201465B2 (en) 2013-03-15 2020-02-28 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
AU2022209254A AU2022209254B2 (en) 2013-03-15 2022-07-27 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

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US201361799647P 2013-03-15 2013-03-15
US61/799,647 2013-03-15
US201361838148P 2013-06-21 2013-06-21
US201361838178P 2013-06-21 2013-06-21
US61/838,178 2013-06-21
US61/838,148 2013-06-21
US201361921007P 2013-12-26 2013-12-26
US61/921,007 2013-12-26
PCT/US2014/029068 WO2014144592A2 (en) 2013-03-15 2014-03-14 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing

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AU2014228981A1 AU2014228981A1 (en) 2015-10-01
AU2014228981B2 true AU2014228981B2 (en) 2019-11-28

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AU2014227653A Active AU2014227653B2 (en) 2013-03-15 2014-03-14 Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
AU2014228981A Active AU2014228981B2 (en) 2013-03-15 2014-03-14 Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing
AU2014239665A Active AU2014239665B2 (en) 2013-03-15 2014-03-14 RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
AU2017204909A Active AU2017204909B2 (en) 2013-03-15 2017-07-17 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2019204675A Active AU2019204675B2 (en) 2013-03-15 2019-07-01 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2020201465A Active AU2020201465B2 (en) 2013-03-15 2020-02-28 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
AU2020207840A Active AU2020207840B2 (en) 2013-03-15 2020-07-23 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
AU2021203370A Active AU2021203370B2 (en) 2013-03-15 2021-05-25 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2022209254A Active AU2022209254B2 (en) 2013-03-15 2022-07-27 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
AU2022252788A Active AU2022252788B2 (en) 2013-03-15 2022-10-13 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

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AU2014239665A Active AU2014239665B2 (en) 2013-03-15 2014-03-14 RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
AU2017204909A Active AU2017204909B2 (en) 2013-03-15 2017-07-17 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2019204675A Active AU2019204675B2 (en) 2013-03-15 2019-07-01 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2020201465A Active AU2020201465B2 (en) 2013-03-15 2020-02-28 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
AU2020207840A Active AU2020207840B2 (en) 2013-03-15 2020-07-23 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
AU2021203370A Active AU2021203370B2 (en) 2013-03-15 2021-05-25 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
AU2022209254A Active AU2022209254B2 (en) 2013-03-15 2022-07-27 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
AU2022252788A Active AU2022252788B2 (en) 2013-03-15 2022-10-13 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

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WO2008027558A2 (en) 2006-08-31 2008-03-06 Codon Devices, Inc. Iterative nucleic acid assembly using activation of vector-encoded traits
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
AU2011338841B2 (en) 2010-11-12 2017-02-16 Gen9, Inc. Methods and devices for nucleic acids synthesis
EP2637780B1 (en) 2010-11-12 2022-02-09 Gen9, Inc. Protein arrays and methods of using and making the same
BR112013024337A2 (pt) 2011-03-23 2017-09-26 Du Pont locus de traço transgênico complexo em uma planta, planta ou semente, método para produzir em uma planta um locus de traço transgênico complexo e construto de expressão
EP3461896B1 (en) 2011-07-15 2023-11-29 The General Hospital Corporation Methods of transcription activator like effector assembly
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
AU2012300401B2 (en) 2011-08-26 2018-02-08 Ginkgo Bioworks, 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
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
EP2841601B1 (en) 2012-04-24 2019-03-06 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
US20150191719A1 (en) 2012-06-25 2015-07-09 Gen9, Inc. Methods for Nucleic Acid Assembly and High Throughput Sequencing
EP2906602B1 (en) * 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
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
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
US10676749B2 (en) 2013-02-07 2020-06-09 The General Hospital Corporation Tale transcriptional activators
US10138509B2 (en) 2013-03-12 2018-11-27 President And Fellows Of Harvard College Method for generating a three-dimensional nucleic acid containing matrix
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
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
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
RU2690935C2 (ru) 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
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
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
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
EP3611268A1 (en) 2013-08-22 2020-02-19 E. I. du Pont de Nemours and Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9737604B2 (en) * 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
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
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
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
EP3080266B1 (en) * 2013-12-12 2021-02-03 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
CA2935032C (en) * 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
AU2015217208B2 (en) 2014-02-11 2018-08-30 The Regents Of The University Of Colorado, A Body Corporate CRISPR enabled multiplexed genome engineering
WO2015134812A1 (en) 2014-03-05 2015-09-11 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
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
CN106460003A (zh) 2014-04-08 2017-02-22 北卡罗来纳州立大学 用于使用crispr相关基因rna引导阻遏转录的方法和组合物
US20160060655A1 (en) 2014-05-30 2016-03-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
IL286474B2 (en) 2014-06-23 2023-11-01 Massachusetts Gen Hospital Genome-wide random identification of DSBS assessed by sequencing (guide-sequence)
US20150376586A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
WO2015200697A1 (en) 2014-06-25 2015-12-30 The General Hospital Corporation Targeting human satellite ii (hsatii)
EP3167071B1 (en) * 2014-07-09 2020-10-07 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
WO2016007347A1 (en) 2014-07-11 2016-01-14 E. I. Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
WO2016033298A1 (en) 2014-08-28 2016-03-03 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
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
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
EP3204496A1 (en) * 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
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
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
US11352666B2 (en) 2014-11-14 2022-06-07 Institute For Basic Science Method for detecting off-target sites of programmable nucleases in a genome
JP6621820B2 (ja) * 2014-11-14 2019-12-18 インスティチュート フォー ベーシック サイエンスＩｎｓｔｉｔｕｔｅ Ｆｏｒ Ｂａｓｉｃ Ｓｃｉｅｎｃｅ ゲノムでプログラマブルヌクレアーゼの非標的位置を検出する方法
CN104531633A (zh) * 2014-11-18 2015-04-22 李云英 Cas9-scForkI融合蛋白及其应用
US10858662B2 (en) 2014-11-19 2020-12-08 Institute For Basic Science Genome editing with split Cas9 expressed from two vectors
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
CN107208079B (zh) * 2014-12-05 2021-06-29 应用干细胞有限公司 整合转基因的位点定向crispr/重组酶组合物和方法
AU2015360502A1 (en) 2014-12-10 2017-06-29 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3889260A1 (en) * 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3234133B1 (en) * 2014-12-18 2020-11-11 Integrated DNA Technologies, Inc. Crispr-based compositions and methods of use
US20180179523A1 (en) * 2014-12-18 2018-06-28 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. 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
RU2713328C2 (ru) * 2015-01-28 2020-02-04 Пайонир Хай-Бред Интернэшнл, Инк. Гибридные днк/рнк-полинуклеотиды crispr и способы применения
EP3256487A4 (en) * 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
IL254038B2 (en) 2015-02-18 2023-03-01 Univ Iowa State Res Found Inc Modification of the binding site of transcriptional suppressor in the nf-yc4 promoter to increase protein content and stress resistance
KR102598856B1 (ko) 2015-03-03 2023-11-07 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
US10450576B2 (en) 2015-03-27 2019-10-22 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
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
JP2018522249A (ja) 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価
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
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
CA3000155A1 (en) 2015-05-29 2016-12-08 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
EP3303607A4 (en) 2015-05-29 2018-10-10 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
US20180296537A1 (en) 2015-06-05 2018-10-18 Novartis Ag Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
EP3726218B1 (en) * 2015-06-10 2023-08-09 Firmenich Sa Cell lines for screening odorant and aroma receptors
US20160362705A1 (en) 2015-06-12 2016-12-15 Lonza Walkersville, Inc. Methods for Nuclear Reprogramming Using Synthetic Transcription Factors
CN107922918B (zh) 2015-06-15 2022-10-21 北卡罗来纳州立大学 用于有效递送核酸和基于rna的抗微生物剂的方法和组合物
CN109536474A (zh) 2015-06-18 2019-03-29 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
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
US11279928B2 (en) * 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
CA2990699A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
EP3322804B1 (en) 2015-07-15 2021-09-01 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
WO2017024047A1 (en) * 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
WO2017023974A1 (en) * 2015-08-03 2017-02-09 President And Fellows Of Harvard College Cas9 genome editing and transcriptional regulation
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US10526590B2 (en) 2015-08-31 2020-01-07 Agilent Technologies, Inc. Compounds and methods for CRISPR/Cas-based genome editing by homologous recombination
WO2017044776A1 (en) * 2015-09-10 2017-03-16 Texas Tech University System Single-guide rna (sgrna) with improved knockout efficiency
EP3347467B1 (en) 2015-09-11 2021-06-23 The General Hospital Corporation Full interrogation of nuclease dsbs and sequencing (find-seq)
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
JP2018529353A (ja) 2015-09-30 2018-10-11 ザ ジェネラル ホスピタル コーポレイション 配列決定法による切断事象の包括的生体外報告（ＣＩＲＣＬＥ−ｓｅｑ）
AU2016336565B2 (en) * 2015-10-06 2019-07-18 Aict Method for producing genome-modified plants from plant protoplasts at high efficiency
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3350327B1 (en) 2015-10-23 2018-09-26 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
JP6882282B2 (ja) 2015-11-03 2021-06-02 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 三次元核酸含有マトリックスの立体撮像のための方法と装置
EP3374501B1 (en) 2015-11-11 2023-07-12 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
EP3414255B1 (en) 2015-11-20 2021-09-22 Washington University Preparative electrophoretic method for targeted purification of genomic dna fragments
WO2017090724A1 (ja) 2015-11-25 2017-06-01 国立大学法人 群馬大学 Ｄｎａメチル化編集用キットおよびｄｎａメチル化編集方法
MX2018005377A (es) 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
WO2017106251A1 (en) * 2015-12-14 2017-06-22 President And Fellows Of Harvard College Cas discrimination using tuned guide rna
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
EA201891532A1 (ru) * 2015-12-28 2019-01-31 Новартис Аг Композиции и способы лечения гемоглобинопатий
SG10202112024PA (en) 2016-01-11 2021-12-30 Univ Leland Stanford Junior Chimeric proteins and methods of immunotherapy
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
US11845933B2 (en) 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
ES2835101T3 (es) 2016-02-10 2021-06-21 Univ Michigan Regents Detección de ácidos nucleicos
EP3417061B1 (en) 2016-02-18 2022-10-26 The Regents of the University of California Methods and compositions for gene editing in stem cells
US10538750B2 (en) 2016-02-29 2020-01-21 Agilent Technologies, Inc. Methods and compositions for blocking off-target nucleic acids from cleavage by CRISPR proteins
CN109196102B (zh) * 2016-03-15 2022-04-26 阿普斯公司 增加双链rna产生的方法和组合物
CN109414414A (zh) 2016-03-16 2019-03-01 戴维·格拉德斯通研究所 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
CN106701765A (zh) * 2016-04-11 2017-05-24 广东赤萌医疗科技有限公司 用于hiv感染治疗的多核苷酸及其制备药物应用
WO2017180915A2 (en) 2016-04-13 2017-10-19 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
EP4613756A3 (en) 2016-04-25 2025-11-12 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
CN107326046A (zh) * 2016-04-28 2017-11-07 上海邦耀生物科技有限公司 一种提高外源基因同源重组效率的方法
WO2017201476A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
US20190100732A1 (en) * 2016-06-02 2019-04-04 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
CA3026321C (en) * 2016-06-02 2023-10-03 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2017217768A1 (ko) * 2016-06-15 2017-12-21 주식회사 툴젠 온타겟 및 오프타겟의 다중 타겟 시스템을 이용하는, 표적 특이적 유전자 가위 스크리닝 방법 및 이의 용도
DK3474669T3 (da) 2016-06-24 2022-06-27 Univ Colorado Regents Fremgangsmåde til generering af stregkodede kombinatoriske biblioteker
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US10669558B2 (en) * 2016-07-01 2020-06-02 Microsoft Technology Licensing, Llc Storage through iterative DNA editing
CA3030783A1 (en) * 2016-07-13 2018-01-18 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
US20230151341A1 (en) * 2016-07-13 2023-05-18 Qihan Chen Method for specifically editing genomic dna and application thereof
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
EP3491133A4 (en) * 2016-07-26 2020-05-06 The General Hospital Corporation VARIANTS OF CRISPR OF PREVOTELLA AND FRANCISELLA 1 (CPF1)
JP6875500B2 (ja) * 2016-07-28 2021-05-26 インスティテュート フォー ベーシック サイエンスＩｎｓｔｉｔｕｔｅ Ｆｏｒ Ｂａｓｉｃ Ｓｃｉｅｎｃｅ Ｃａｓ９タンパク質およびガイドＲＮＡを含む眼疾患治療用薬学組成物
BR112019001783A2 (pt) 2016-07-29 2019-05-07 Regeneron Pharmaceuticals, Inc. mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto.
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
EP3498834A4 (en) 2016-08-10 2020-06-17 Genahead Bio, Inc. METHOD FOR MODIFYING A TARGET SITE IN THE GENOME OF A EUKARYOTE CELL, AND METHOD FOR DETECTING THE PRESENCE OR ABSENCE OF A NUCLEIC ACID SEQUENCE TO BE DETECTED AT A TARGET SITE
WO2018035495A1 (en) * 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation
AU2017313616B2 (en) * 2016-08-19 2022-12-08 Institute For Basic Science Artificially engineered angiogenesis regulatory system
US10975393B2 (en) 2016-08-24 2021-04-13 Sangamo Therapeutics, Inc. Engineered target specific nucleases
SG11201901531TA (en) 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Regulation of gene expression using engineered nucleases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018048194A1 (ko) * 2016-09-07 2018-03-15 울산대학교 산학협력단 dCas9 단백질 및 표적 핵산 서열에 결합하는 gRNA를 이용한 핵산 검출의 민감도 및 특이도 향상용 조성물 및 방법
WO2018049075A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
CN107880132B (zh) * 2016-09-30 2022-06-17 北京大学 一种融合蛋白及使用其进行同源重组的方法
CN110023494A (zh) 2016-09-30 2019-07-16 加利福尼亚大学董事会 Rna指导的核酸修饰酶及其使用方法
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
CA3039409A1 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
KR20240064734A (ko) 2016-10-14 2024-05-13 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
US20180282722A1 (en) * 2016-11-21 2018-10-04 Massachusetts Institute Of Technology Chimeric DNA:RNA Guide for High Accuracy Cas9 Genome Editing
JP2019535287A (ja) 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Ｃｒｉｓｐｒ／ｃｐｆ１システム及び方法
CN110291198B (zh) 2016-12-08 2024-11-26 因特利亚治疗公司 经修饰的指导rna
US20200149039A1 (en) 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
JP7317706B2 (ja) 2016-12-14 2023-07-31 リガンダル インコーポレイテッド 核酸およびタンパク質ペイロード送達のための方法および組成物
US12286727B2 (en) 2016-12-19 2025-04-29 Editas Medicine, Inc. Assessing nuclease cleavage
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3562942A4 (en) * 2016-12-28 2020-12-09 Ionis Pharmaceuticals, Inc. MODIFIED CRISPR-RNA AND ITS USES
JP7219972B2 (ja) 2017-01-05 2023-02-09 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー Ｄｎａ二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
US20180258418A1 (en) * 2017-01-17 2018-09-13 Institute For Basic Science Method of identifying genome-wide off-target sites of base editors by detecting single strand breaks in genomic dna
KR102712656B1 (ko) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
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
CN110312797A (zh) 2017-02-10 2019-10-08 齐默尔根公司 组装和编辑用于多个宿主的多个dna构建体的模块化通用质粒设计策略
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
CA3055565A1 (en) 2017-03-08 2018-09-13 The Regents Of The University Of Michigan Analyte detection
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN108660161B (zh) * 2017-03-31 2023-05-09 中国科学院脑科学与智能技术卓越创新中心 基于CRISPR/Cas9技术的制备无嵌合基因敲除动物的方法
JP2020515250A (ja) * 2017-04-07 2020-05-28 セージ サイエンス， インコーポレイテッド 統合型電気泳動ｄｎａ精製を使用する遺伝的構造変動を検出するためのシステムおよび方法
EP3612551B1 (en) 2017-04-21 2024-09-04 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
CA3059208A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Inducible, tunable, and multiplex human gene regulation using crispr-cpf1
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2018218188A2 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
CN110997906B (zh) 2017-06-05 2024-05-07 雷杰纳荣制药公司 B4galt1变体及其用途
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
KR102634727B1 (ko) 2017-06-14 2024-02-07 위스콘신 얼럼나이 리서어치 화운데이션 변형된 가이드 rna, crispr-리보뉴클레오프로테인 복합체 및 사용 방법
KR102720855B1 (ko) 2017-06-15 2024-10-25 더 리전트 오브 더 유니버시티 오브 캘리포니아 표적화된 비-바이러스 dna 삽입
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
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019014230A1 (en) 2017-07-11 2019-01-17 Sigma-Aldrich Co. Llc USE OF DOMAINS OF PROTEINS INTERACTING WITH NUCLEOSOMES TO IMPROVE THE TARGETED MODIFICATION OF THE GENOME
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
KR20200033259A (ko) * 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
RU2019143568A (ru) 2017-07-31 2021-09-02 Регенерон Фармасьютикалс, Инк. Способы и композиции для оценки crispr/cas-индуцированной рекомбинации с экзогенной донорной нуклеиновой кислотой in vivo
KR102780441B1 (ko) 2017-07-31 2025-03-17 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
WO2019024081A1 (zh) 2017-08-04 2019-02-07 北京大学 特异性识别甲基化修饰dna碱基的tale rvd及其应用
CN111278983A (zh) 2017-08-08 2020-06-12 北京大学 基因敲除方法
EP3672612A4 (en) 2017-08-23 2021-09-29 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES WITH MODIFIED PAM SPECIFICITY
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
EP3585162B1 (en) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
EP3694993A4 (en) 2017-10-11 2021-10-13 The General Hospital Corporation SITE-SPECIFIC AND PARASITIC GENOMIC DESAMINATION DETECTION METHODS INDUCED BY BASIC EDITING TECHNOLOGIES
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN107602707B (zh) * 2017-10-17 2021-04-23 湖北大学 一种特异性调节枯草芽孢杆菌外源基因表达的dcas9-ω融合蛋白及其应用
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
US20230061455A1 (en) * 2017-11-01 2023-03-02 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy
CN120310773A (zh) * 2017-11-16 2025-07-15 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
KR102098915B1 (ko) * 2017-12-22 2020-04-09 (주)지플러스 생명과학 키메라 게놈 조작 분자 및 방법
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
EP3755798A4 (en) 2018-02-19 2022-04-06 Yale University PHOSPHOPEPTIDE-ENCODING OLIGONUCLEOTIDE LIBRARIES AND METHODS FOR DETECTING PHOSPHORYLATION-DEPENDENT MOLECULAR INTERACTIONS
US12084676B2 (en) 2018-02-23 2024-09-10 Pioneer Hi-Bred International, Inc. Cas9 orthologs
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
WO2019195738A1 (en) 2018-04-06 2019-10-10 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
EP3781585A4 (en) 2018-04-17 2022-01-26 The General Hospital Corporation SENSITIVE IN VITRO ASSAYS FOR SUBSTRATE AND SITE PREFERENCES OF NUCLEIC ACID BINDERS, MODIFIERS AND CLEAVATORS
CN112334577B (zh) 2018-04-19 2023-10-17 加利福尼亚大学董事会 用于基因编辑的组合物和方法
WO2019213430A1 (en) * 2018-05-03 2019-11-07 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for nicking target dna sequences
CN108588123A (zh) * 2018-05-07 2018-09-28 南京医科大学 CRISPR/Cas9载体组合在制备基因敲除猪的血液制品中的应用
US12133884B2 (en) 2018-05-11 2024-11-05 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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3575396A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
CN110592141B (zh) * 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US20210285009A1 (en) 2018-07-13 2021-09-16 The Regents Of The University Of California Retrotransposon-based delivery vehicle and methods of use thereof
CN112703250A (zh) 2018-08-15 2021-04-23 齐默尔根公司 CRISPRi在高通量代谢工程中的应用
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
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的な塩基エディター
WO2020049158A1 (en) 2018-09-07 2020-03-12 Astrazeneca Ab Compositions and methods for improved nucleases
US12264313B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for genome modification and alteration of expression
US12203123B2 (en) 2018-10-01 2025-01-21 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells
EP3861120A4 (en) 2018-10-01 2023-08-16 North Carolina State University Recombinant type i crispr-cas system
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases
CA3117730A1 (en) 2018-11-09 2020-05-14 Inari Agriculture, Inc. Rna-guided nucleases and dna binding proteins
AU2019398351A1 (en) 2018-12-14 2021-06-03 Pioneer Hi-Bred International, Inc. Novel CRISPR-Cas systems for genome editing
KR20210105914A (ko) 2018-12-20 2021-08-27 리제너론 파마슈티칼스 인코포레이티드 뉴클레아제-매개 반복부 팽창
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
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
US12460230B2 (en) 2019-02-10 2025-11-04 The J. David Gladstone Institutes Modified mitochondrion and methods of use thereof
CN113811607A (zh) 2019-03-07 2021-12-17 加利福尼亚大学董事会 CRISPR-Cas效应子多肽和其使用方法
IL286359B2 (en) 2019-03-18 2024-06-01 Regeneron Pharma Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
KR20240126075A (ko) 2019-03-18 2024-08-20 리제너론 파마슈티칼스 인코포레이티드 타우 시딩 또는 응집의 유전적 변형제를 식별하기 위한 CRISPR/Cas 스크리닝 플랫폼
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
CN118064502A (zh) 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
ES2923629T3 (es) 2019-04-04 2022-09-29 Regeneron Pharma Métodos para la introducción sin cicatrices de modificaciones dirigidas en vectores de direccionamiento
AU2020253532B2 (en) 2019-04-04 2024-06-20 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
JP7641233B2 (ja) 2019-04-12 2025-03-06 アストラゼネカ・アクチエボラーグ 改善された遺伝子編集のための組成物及び方法
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
CA3137765A1 (en) 2019-06-14 2020-12-17 Regeneron Pharmaceuticals, Inc. Models of tauopathy
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
WO2021050940A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
ES2938896T3 (es) 2019-10-21 2023-04-17 Univ Freiburg Albert Ludwigs Un ensayo in vitro verdaderamente imparcial para perfilar la actividad fuera de objetivo de una o más nucleasas programables específicas de objetivo en células (ABNOBA-SEQ)
US11331333B2 (en) 2019-11-08 2022-05-17 Georg-August-Universität Göttingen Stiftung Öffentichen Rechts, Universitätsmadizin Treatment of aberrant fibroblast proliferation
EP4054651A1 (en) 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021108561A1 (en) * 2019-11-25 2021-06-03 La Jolla Institute For Immunology Methods and compositions for modulating heterochromatin dysfunction, genomic instability, and associated conditions
CN115176001A (zh) 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
CN111088357B (zh) * 2019-12-31 2022-09-20 深圳大学 针对escc的肿瘤标志物及其应用
EP4093864A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
US12264341B2 (en) 2020-01-24 2025-04-01 The General Hospital Corporation CRISPR-Cas enzymes with enhanced on-target activity
JP2023519101A (ja) 2020-02-12 2023-05-10 マサチューセッツ アイ アンド イヤー インファーマリー Ｒｐ１関連網膜変性症のハプロタイプに基づく処置
WO2021178556A1 (en) 2020-03-04 2021-09-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
WO2021178924A1 (en) * 2020-03-05 2021-09-10 Board Of Regents Of The University Of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
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
BR112022020407A2 (pt) * 2020-04-09 2023-05-02 Verve Therapeutics Inc Edição base do pcsk9 e métodos de uso do mesmo para tratamento de doenças
PH12022553003A1 (en) * 2020-05-04 2023-04-24 Bluerock Therapeutics Lp Selection by essential-gene knock-in
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
EP4150081A1 (en) 2020-05-13 2023-03-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
WO2021246165A1 (ja) * 2020-06-03 2021-12-09 国立大学法人広島大学 Ｏａｓｉｓ遺伝子の脱メチル化のための核酸及びそれを用いた脱メチル化方法
US20230212323A1 (en) * 2020-06-05 2023-07-06 The Regents Of The University Of California Compositions and methods for epigenome editing
WO2022008935A1 (en) 2020-07-10 2022-01-13 Horizon Discovery Limited Method for producing genetically modified cells
AU2021324538A1 (en) 2020-08-11 2023-03-09 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Method for the treatment of wwox associated diseases
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
CN116391040A (zh) 2020-10-13 2023-07-04 法国国家科学研究中心(Cnrs) 结合接合和Crispr/Cas系统的靶向抗菌质粒及其用途
RU2762831C1 (ru) * 2020-10-26 2021-12-23 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) Молекула рнк-проводника для геномного редактирования протомоторной области гена vrn-a1 однодольных зерновых с применением системы crispr/cas9
CN112430622A (zh) * 2020-10-26 2021-03-02 扬州大学 一种FokI和dCpf1融合蛋白表达载体及其介导的定点基因编辑方法
US20240002839A1 (en) 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
KR20220082186A (ko) 2020-12-10 2022-06-17 한세준 유,무선 충전이 가능한 보조배터리형 uv-led살균기
EP4274893B1 (en) 2021-01-05 2025-01-01 Revvity Discovery Limited Method for producing genetically modified cells
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
EP4297799A1 (en) 2021-02-25 2024-01-03 Institut National de la Santé et de la Recherche Médicale (INSERM) Allele-specific genome editing of the nr2e3 mutation g56r
KR102882704B1 (ko) * 2021-03-03 2025-11-12 중앙대학교 산학협력단 CRISPR/Cas9 시스템을 이용한 유전체 단일염기 편집 방법 및 이의 용도
CN113846019B (zh) * 2021-03-05 2023-08-01 海南师范大学 一种海洋微拟球藻靶向表观基因组遗传调控方法
EP4095243A1 (en) 2021-05-25 2022-11-30 European Molecular Biology Laboratory System for hybridization-based precision genome cleavage and editing, and uses thereof
WO2022248645A1 (en) 2021-05-27 2022-12-01 Astrazeneca Ab Cas9 effector proteins with enhanced stability
MX2023014470A (es) 2021-06-10 2024-03-05 Intellia Therapeutics Inc O arn guía modificados que comprenden un enlazador interno para modificación del genoma.
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023023635A1 (en) 2021-08-20 2023-02-23 Wisconsin Alumni Research Foundation Nonviral generation of genome edited chimeric antigen receptor t cells
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
EP4409000A1 (en) 2021-09-28 2024-08-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
US20240392272A1 (en) 2021-09-28 2024-11-28 Acrigen Biosciences Compositions and methods for nucleic acid modifications
WO2023052508A2 (en) 2021-09-30 2023-04-06 Astrazeneca Ab Use of inhibitors to increase efficiency of crispr/cas insertions
CA3232968A1 (en) 2021-10-14 2023-04-20 Jasper Williams Immune cells having co-expressed shrnas and logic gate systems
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
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
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
CA3237303A1 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
JP2024543966A (ja) 2021-12-01 2024-11-26 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 赤血球特異的ＢＣＬ１１Ａエンハンサーの＋５５ｋｂ領域を編集することにより胎児ヘモグロビン含量を増加させるための方法。
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel 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
EP4463548A1 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
US20250113795A1 (en) * 2022-01-20 2025-04-10 Inari Agriculture Technology, Inc. Improved soybean explant preparation and transformation
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4469574A1 (en) 2022-01-25 2024-12-04 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-thalassemia
TW202332767A (zh) 2022-02-02 2023-08-16 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
WO2023152351A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Treatment of liver cancers by disrupting the beta-catenin/tcf-4 binding site located upstream of meg3 in the dlk1/dio3 locus
EP4514981A2 (en) 2022-04-29 2025-03-05 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
CA3256953A1 (en) 2022-05-09 2023-11-16 Regeneron Pharma VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
US20250241956A1 (en) 2022-05-10 2025-07-31 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia
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
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics Inc TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
MA71557A (fr) 2022-07-18 2025-05-30 Renagade Therapeutics Management Inc. Composants d'édition génique, systèmes et procédés d'utilisation
EP4558633A1 (en) 2022-07-22 2025-05-28 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from beta-thalassemia
JP2025528052A (ja) 2022-07-29 2025-08-26 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 脳及び筋肉へのトランスフェリン受容体（ｔｆｒ）媒介送達のための組成物及び方法
CA3264214A1 (en) 2022-08-19 2024-02-22 Tune Therapeutics Inc Compositions, systems and methods of regulating the hepatitis B virus by targeted gene suppression
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
CN120239746A (zh) 2022-09-19 2025-07-01 图恩疗法股份有限公司 用于调节t细胞功能的组合物、系统和方法
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
EP4605525A1 (en) 2022-10-21 2025-08-27 Keygene N.V. Rna transfection in plant cells with modified rna
CN120693347A (zh) 2022-11-04 2025-09-23 瑞泽恩制药公司 钙电压门控通道辅助亚基γ1(CACNG1)结合蛋白和CACNG1介导的向骨骼肌的递送
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
CN115820603B (zh) * 2022-11-15 2024-07-05 吉林大学 一种基于dCasRx-NSUN6单基因特异性M5C修饰编辑方法
WO2024121354A1 (en) 2022-12-08 2024-06-13 Keygene N.V. Duplex sequencing with covalently closed dna ends
EP4640835A1 (en) * 2022-12-23 2025-10-29 Epigenic Therapeutics Pte. Ltd. Fusion and use thereof
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
EP4662313A1 (en) 2023-02-06 2025-12-17 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through multiplex base editing
CN116376975B (zh) * 2023-02-27 2024-05-14 中国科学院脑科学与智能技术卓越创新中心 激活异染色质基因的方法及应用
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
CN118684781A (zh) * 2023-03-21 2024-09-24 深圳赫兹生命科学技术有限公司 GnRH-VLP重组去势疫苗及其制备方法
WO2024201368A1 (en) 2023-03-29 2024-10-03 Astrazeneca Ab Use of inhibitors to increase efficiency of crispr/cas insertions
WO2024209000A1 (en) 2023-04-04 2024-10-10 Keygene N.V. Linkers for duplex sequencing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025006963A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
WO2025017030A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
WO2025017033A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
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
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
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
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
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
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
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo

Family Cites Families (166)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to binding proteins for recognition of dna
US20030017149A1 (en) 1996-10-10 2003-01-23 Hoeffler James P. Single chain monoclonal antibody fusion reagents that regulate transcription in vivo
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
AU2001257331A1 (en) 2000-04-28 2001-11-12 Sangamo Biosciences, Inc. Methods for designing exogenous regulatory molecules
AU5391401A (en) 2000-04-28 2001-11-12 Sangamo Biosciences Inc Targeted modification of chromatin structure
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
AU2003219847A1 (en) 2002-02-21 2003-09-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
WO2003104414A2 (en) * 2002-06-11 2003-12-18 The Scripps Research Institute Artificial transcription factors
WO2004099366A2 (en) 2002-10-23 2004-11-18 The General Hospital Corporation Context sensitive parallel optimization of zinc finger dna binding domains
US7021555B2 (en) 2004-01-06 2006-04-04 Zoo Med Laboratories, Inc. Spraying/misting for plants and animals
US7919277B2 (en) 2004-04-28 2011-04-05 Danisco A/S Detection and typing of bacterial strains
ES2595363T3 (es) 2005-02-18 2016-12-29 J. Craig Venter Institute, Inc. Sepsis asociada a las proteínas y los ácidos nucleicos de meningitis / Escherichia coli
US20100055793A1 (en) * 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
EP2325332B1 (en) 2005-08-26 2012-10-31 DuPont Nutrition Biosciences ApS Use of CRISPR associated genes (CAS)
JP2009520463A (ja) 2005-11-28 2009-05-28 ザ スクリプス リサーチ インスティテュート Ｔｎｎのための亜鉛フィンガー結合ドメイン
US9816140B2 (en) 2006-05-19 2017-11-14 Dupont Nutrition Biosciences Aps Tagged microorganisms and methods of tagging
EP2213731B1 (en) 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
AU2007258872A1 (en) 2006-06-16 2007-12-21 Danisco A/S Bacterium
US9201063B2 (en) 2006-11-16 2015-12-01 General Electric Company Sequential analysis of biological samples
WO2008093152A1 (en) * 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
NZ579002A (en) 2007-03-02 2012-03-30 Danisco Cultures with improved phage resistance
US8153863B2 (en) 2007-03-23 2012-04-10 New York University Transgenic plants expressing GLK1 and CCA1 having increased nitrogen assimilation capacity
WO2008122314A1 (de) 2007-04-10 2008-10-16 Qiagen Gmbh Rna-interferenz tags
WO2008151032A2 (en) 2007-05-31 2008-12-11 Washington University In St. Louis Arrays and methods comprising m. smithii gene products
ES2625144T3 (es) 2007-09-25 2017-07-18 Pastoral Greenhouse Gas Research Ltd Polinucleótidos y polipéptidos del fargo MRU y usos de éstos
FR2925918A1 (fr) 2007-12-28 2009-07-03 Pasteur Institut Typage et sous-typage moleculaire de salmonella par identification des sequences nucleotidiques variables des loci crispr
FR2930264B1 (fr) 2008-04-18 2013-02-22 Gervais Danone Sa Nouvelle souche de lactobacillus paracasei subsp. paracasei dotee de proprietes antimicrobiennes et immunomodulatrices.
JP2010017178A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010017179A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
JP2010048566A (ja) 2008-08-19 2010-03-04 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010068800A (ja) 2008-08-19 2010-04-02 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US9115386B2 (en) 2008-09-26 2015-08-25 Children's Medical Center Corporation Selective oxidation of 5-methylcytosine by TET-family proteins
NZ592994A (en) 2008-10-21 2012-12-21 Animal Health Trust Diagnostic test for Streptococcus equi comprising assessing the presence or absence of the S. equi eqbE gene
EP2344641A2 (en) 2008-10-23 2011-07-20 Université de Lausanne Gene transfer vectors comprising at least one isolated dna molecule having insulator and or boundary properties and methods to identify the same
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
MX337838B (es) * 2008-11-07 2016-03-22 Dupont Nutrition Biosci Aps Secuencias de repetidos palindromicos cortos regularmente intercalados agrupados de bifidobacterias.
US9771624B2 (en) 2008-11-11 2017-09-26 The Procter & Gamble Company Bifidobacterium longum
GB2466177A (en) 2008-12-03 2010-06-16 Arab Science & Technology Found Bacteriophage selection and breeding
WO2010066907A1 (en) 2008-12-12 2010-06-17 Danisco A/S Genetic cluster of strains of streptococcus thermophilus having unique rheological properties for dairy fermentation
KR20100093626A (ko) 2009-02-17 2010-08-26 서강대학교산학협력단 슈도모나스 애루지노사에 대한 파아지 치료
US8828718B2 (en) 2009-04-03 2014-09-09 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
AU2010245304B2 (en) 2009-04-27 2015-06-04 Pacific Biosciences Of California, Inc. Real-time sequencing methods and systems
US8609421B2 (en) 2009-06-12 2013-12-17 Pacific Biosciences Of California, Inc. Single-molecule real-time analysis of protein synthesis
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
AR080340A1 (es) 2009-09-25 2012-04-04 Crop Functional Genomics Ct Plantas que tienen mejores rasgos relacionados con el rendimiento y un metodo para producirlas mediante la modulacion de la expresion de sgt-1, clcpkg o un polipeptido tipo hd-hidrolasa
US9677125B2 (en) 2009-10-21 2017-06-13 General Electric Company Detection of plurality of targets in biological samples
US20110269119A1 (en) 2009-10-30 2011-11-03 Synthetic Genomics, Inc. Encoding text into nucleic acid sequences
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
WO2011101696A1 (en) * 2010-02-18 2011-08-25 Cellectis Improved meganuclease recombination system
US20120027786A1 (en) 2010-02-23 2012-02-02 Massachusetts Institute Of Technology Genetically programmable pathogen sense and destroy
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
NZ602497A (en) 2010-03-12 2014-03-28 Brookhaven Science Ass Llc Enterobacter sp. 638 and methods of use thereof
EA024121B9 (ru) 2010-05-10 2017-01-30 Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния Композиции эндорибонуклеаз и способы их использования
AU2011265733B2 (en) 2010-06-14 2014-04-17 Iowa State University Research Foundation, Inc. Nuclease activity of TAL effector and Foki fusion protein
WO2012047726A1 (en) 2010-09-29 2012-04-12 The Broad Institute, Inc. Methods for chromatin immuno-precipitations
EP2630156B1 (en) 2010-10-20 2018-08-22 DuPont Nutrition Biosciences ApS Lactococcus crispr-cas sequences
JP2013544082A (ja) 2010-10-27 2013-12-12 セレクティス 二本鎖破断−誘導変異誘発の効率を増大させる方法
WO2012093833A2 (en) * 2011-01-03 2012-07-12 Toolgen Incorporation Genome engineering via designed tal effector nucleases
EP2663654A1 (en) 2011-01-14 2013-11-20 Life Technologies Corporation Methods, compositions, and kits for detecting rare cells
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
EP2543255B2 (en) 2011-07-04 2022-12-28 DSM IP Assets B.V. Anti-listerial mixed culture and method for producing cheese
EP3461896B1 (en) 2011-07-15 2023-11-29 The General Hospital Corporation Methods of transcription activator like effector assembly
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
BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
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
UA115875C2 (uk) 2012-05-02 2018-01-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Рослина помідора зі зменшеною активністю малатдегідрогенази та спосіб її отримання
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
US11120889B2 (en) 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
FI3597749T3 (fi) * 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
EP2674501A1 (en) 2012-06-14 2013-12-18 Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail Method for detecting and identifying enterohemorrhagic Escherichia coli
US10025647B2 (en) * 2012-06-30 2018-07-17 Intel Corporation Memory poisoning with hints
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP2877213B1 (en) 2012-07-25 2020-12-02 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
CA2884162C (en) 2012-09-07 2020-12-29 Dow Agrosciences Llc Fad3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
CA2887475C (en) 2012-10-09 2021-12-14 Liposcience, Inc. Nmr quantification of branched chain amino acids
EP2906602B1 (en) * 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
WO2014071235A1 (en) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
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
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
EP4234696A3 (en) 2012-12-12 2023-09-06 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
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
BR102013032129B1 (pt) 2012-12-13 2022-06-07 Dow Agrosciences Llc Método para identificar a presença de um polinucleotídeo de dna doador exógeno inserido dentro de um único locus genômico eucariótico alvo
EP4282970A3 (en) * 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
AR094098A1 (es) * 2012-12-19 2015-07-08 Dow Agrosciences Llc Transformacion de soja mejorada para una produccion eficaz de eventos transgenicos de alto rendimiento
WO2014110552A1 (en) 2013-01-14 2014-07-17 Recombinetics, Inc. Hornless livestock
US20140212869A1 (en) 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
CN103233028B (zh) 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
WO2014124226A1 (en) 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US10676749B2 (en) 2013-02-07 2020-06-09 The General Hospital Corporation Tale transcriptional activators
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
JP2016507244A (ja) 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト（ゲーエムベーハー）Ｈｅｌｍｈｏｌｔｚ Ｚｅｎｔｒｕｍ ＭｕｅｎｃｈｅｎＤｅｕｔｓｃｈｅｓ Ｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ｆｕｅｒ Ｇｅｓｕｎｄｈｅｉｔ ｕｎｄ Ｕｍｗｅｌｔ （ＧｍｂＨ） Ｃａｓ９ヌクレアーゼによる卵母細胞における遺伝子編集
AU2014224432B2 (en) * 2013-03-08 2019-10-24 Oxford Nanopore Technologies Limited Enzyme stalling method
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
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
WO2014165612A2 (en) 2013-04-05 2014-10-09 Dow Agrosciences Llc Methods and compositions for integration of an exogenous sequence within the genome of plants
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
CN103224947B (zh) 2013-04-28 2015-06-10 陕西师范大学 一种基因打靶系统
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
US20150067922A1 (en) 2013-05-30 2015-03-05 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
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
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
WO2015010114A1 (en) 2013-07-19 2015-01-22 Larix Bioscience, Llc Methods and compositions for producing double allele knock outs
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
AU2014312123A1 (en) 2013-08-29 2016-03-17 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
WO2015033293A1 (en) 2013-09-04 2015-03-12 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
EP3079726B1 (en) 2013-12-12 2018-12-05 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
CA2935032C (en) 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
EP3105327A4 (en) 2014-02-12 2017-10-18 Thomas Jefferson University Compositions and methods of using microrna inhibitors
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US10590433B2 (en) 2014-03-14 2020-03-17 University Of Washington Genomic insulator elements and uses thereof
EP3126498A4 (en) 2014-03-20 2017-08-23 Université Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
WO2015153940A1 (en) 2014-04-03 2015-10-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
MA41349A (fr) 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
RU2713328C2 (ru) 2015-01-28 2020-02-04 Пайонир Хай-Бред Интернэшнл, Инк. Гибридные днк/рнк-полинуклеотиды crispr и способы применения
ES2880473T5 (es) 2015-01-30 2024-05-09 Univ California Suministro de proteínas en células hematopoyéticas primarias
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
KR102598856B1 (ko) 2015-03-03 2023-11-07 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
HK1253403A1 (zh) 2015-05-28 2019-06-14 Coda Biotherapeutics 基因組編輯載體
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
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
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
KR20240064734A (ko) 2016-10-14 2024-05-13 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
AU2018218280B2 (en) 2017-02-07 2024-10-17 The Regents Of The University Of California Gene therapy for haploinsufficiency
CA3059208A1 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Inducible, tunable, and multiplex human gene regulation using crispr-cpf1
EP3793584A4 (en) 2018-05-17 2022-10-26 The General Hospital Corporation CCCTC BINDING FACTOR VARIATIONS
JP2023503618A (ja) 2019-11-27 2023-01-31 ザ ジェネラル ホスピタル コーポレイション 遺伝子発現を活性化するためのシステムおよび方法
EP4176434A4 (en) 2020-05-29 2024-09-11 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JINEK ET AL., "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.", SCIENCE, (2012), vol. 337, no. 6096, pages 816 - 21 *

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