JP2016504026A - 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング - Google Patents

配列操作のための系、方法および最適化ガイド組成物のエンジニアリング Download PDF

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JP2016504026A
JP2016504026A JP2015547573A JP2015547573A JP2016504026A JP 2016504026 A JP2016504026 A JP 2016504026A JP 2015547573 A JP2015547573 A JP 2015547573A JP 2015547573 A JP2015547573 A JP 2015547573A JP 2016504026 A JP2016504026 A JP 2016504026A
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crispr
sequence
cas
tracr
chirna
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JP2016504026A5 (cg-RX-API-DMAC7.html
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フェン・ジャン
レ・コン
パトリック・シュウ
フェイ・ラン
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Broad Institute Inc
Harvard University
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Broad Institute Inc
Harvard University
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Publication of JP2016504026A publication Critical patent/JP2016504026A/ja
Publication of JP2016504026A5 publication Critical patent/JP2016504026A5/ja
Priority to JP2019039723A priority Critical patent/JP6960950B2/ja
Priority to JP2019039724A priority patent/JP6960951B2/ja
Priority to JP2021167368A priority patent/JP7198328B2/ja
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JP2015547573A 2012-12-12 2013-12-12 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング Pending JP2016504026A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2019039723A JP6960950B2 (ja) 2012-12-12 2019-03-05 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
JP2019039724A JP6960951B2 (ja) 2012-12-12 2019-03-05 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
JP2021167368A JP7198328B2 (ja) 2012-12-12 2021-10-12 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング

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US201261736527P 2012-12-12 2012-12-12
US61/736,527 2012-12-12
US201361748427P 2013-01-02 2013-01-02
US61/748,427 2013-01-02
US201361758468P 2013-01-30 2013-01-30
US61/758,468 2013-01-30
US201361769046P 2013-02-25 2013-02-25
US61/769,046 2013-02-25
US201361791409P 2013-03-15 2013-03-15
US201361802174P 2013-03-15 2013-03-15
US61/802,174 2013-03-15
US61/791,409 2013-03-15
US201361806375P 2013-03-28 2013-03-28
US61/806,375 2013-03-28
US201361814263P 2013-04-20 2013-04-20
US61/814,263 2013-04-20
US201361819803P 2013-05-06 2013-05-06
US61/819,803 2013-05-06
US201361828130P 2013-05-28 2013-05-28
US61/828,130 2013-05-28
US201361836127P 2013-06-17 2013-06-17
US201361835931P 2013-06-17 2013-06-17
US61/835,931 2013-06-17
US61/836,127 2013-06-17
PCT/US2013/074819 WO2014093712A1 (en) 2012-12-12 2013-12-12 Engineering of systems, methods and optimized guide compositions for sequence manipulation

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Cited By (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016512048A (ja) * 2013-03-15 2016-04-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ＣＲＩＳＰＲ／Ｃａｓシステムを使用した植物ゲノム操作
JP2016093196A (ja) * 2012-12-12 2016-05-26 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
US9970024B2 (en) 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering
JP2018075026A (ja) * 2013-03-14 2018-05-17 カリブー・バイオサイエンシーズ・インコーポレイテッド 核酸ターゲティング核酸の組成物および方法
JP2019511240A (ja) * 2016-04-14 2019-04-25 ボコ シリコン バレー， インコーポレイテッド ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集
JP2019524072A (ja) * 2016-06-17 2019-09-05 ザ・ブロード・インスティテュート・インコーポレイテッド Ｖｉ型ｃｒｉｓｐｒオルソログ及び系
US10494621B2 (en) 2015-06-18 2019-12-03 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US10550372B2 (en) 2013-12-12 2020-02-04 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US10577630B2 (en) 2013-06-17 2020-03-03 The Broad Institute, Inc. Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
US10696986B2 (en) 2014-12-12 2020-06-30 The Board Institute, Inc. Protected guide RNAS (PGRNAS)
US10711285B2 (en) 2013-06-17 2020-07-14 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US10781444B2 (en) 2013-06-17 2020-09-22 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10844378B2 (en) 2012-03-20 2020-11-24 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US10851357B2 (en) 2013-12-12 2020-12-01 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US10930367B2 (en) 2012-12-12 2021-02-23 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
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US11155795B2 (en) 2013-12-12 2021-10-26 The Broad Institute, Inc. CRISPR-Cas systems, crystal structure and uses thereof
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
US11407985B2 (en) 2013-12-12 2022-08-09 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing
US11578312B2 (en) 2015-06-18 2023-02-14 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
US12251450B2 (en) 2013-12-12 2025-03-18 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12435320B2 (en) 2014-12-24 2025-10-07 The Broad Institute, Inc. CRISPR having or associated with destabilization domains
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof

Families Citing this family (685)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9719068B2 (en) 2010-05-06 2017-08-01 Children's Hospital Medical Center Methods and systems for converting precursor cells into intestinal tissues through directed differentiation
AR085533A1 (es) 2011-03-23 2013-10-09 Pioneer Hi Bred Int Metodos de produccion de un locus de rasgos transgenicos complejo
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
KR101904508B1 (ko) 2012-04-25 2018-10-04 리제너론 파마슈티칼스 인코포레이티드 큰 표적화 벡터를 사용한 뉴클레아제-중재된 표적화
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
EP2861737B1 (en) 2012-06-19 2019-04-17 Regents Of The University Of Minnesota Gene targeting in plants using dna viruses
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
EP3808844A1 (en) * 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
US10584381B2 (en) 2012-08-14 2020-03-10 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10323279B2 (en) 2012-08-14 2019-06-18 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10752949B2 (en) 2012-08-14 2020-08-25 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10273541B2 (en) 2012-08-14 2019-04-30 10X Genomics, Inc. Methods and systems for processing polynucleotides
US9951386B2 (en) 2014-06-26 2018-04-24 10X Genomics, Inc. Methods and systems for processing polynucleotides
US11591637B2 (en) 2012-08-14 2023-02-28 10X Genomics, Inc. Compositions and methods for sample processing
BR112015003354A8 (pt) 2012-08-14 2018-01-16 10X Genomics Inc métodos e composições de microcápsula
US9701998B2 (en) 2012-12-14 2017-07-11 10X Genomics, Inc. Methods and systems for processing polynucleotides
EP4556564A3 (en) * 2012-10-23 2025-09-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
AU2013352156B2 (en) 2012-11-27 2018-12-06 Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
KR20150105635A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
US20140186919A1 (en) 2012-12-12 2014-07-03 Feng Zhang Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
EP3567116A1 (en) 2012-12-14 2019-11-13 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10533221B2 (en) 2012-12-14 2020-01-14 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10544405B2 (en) 2013-01-16 2020-01-28 Emory University Cas9-nucleic acid complexes and uses related thereto
CA2900481A1 (en) 2013-02-08 2014-08-14 10X Genomics, Inc. Polynucleotide barcode generation
EP3578666A1 (en) 2013-03-12 2019-12-11 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
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
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
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
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CN115261411A (zh) 2013-04-04 2022-11-01 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
KR20200136508A (ko) 2013-04-16 2020-12-07 리제너론 파마슈티칼스 인코포레이티드 랫트 게놈의 표적화된 변형
WO2014197568A2 (en) 2013-06-04 2014-12-11 President And Fellows Of Harvard College Rna-guideded transcriptional regulation
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
WO2014202250A1 (en) 2013-06-19 2014-12-24 Kolibree Toothbrush system with sensors for a dental hygiene monitoring system
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
SI3019619T1 (sl) * 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
MX2016002306A (es) 2013-08-22 2016-07-08 Du Pont Promotor u6 de polimerasa iii de soja y metodos de uso.
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
ES2844174T3 (es) 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
WO2015054507A1 (en) 2013-10-10 2015-04-16 Pronutria, Inc. Nutritive polypeptide production systems, and methods of manufacture and use thereof
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
JP6174811B2 (ja) 2013-12-11 2017-08-02 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． ゲノムの標的改変のための方法及び組成物
CN105899657A (zh) 2013-12-12 2016-08-24 布罗德研究所有限公司 用于改变基因产物表达的crispr-cas系统和方法、结构信息以及诱导型模块化cas酶
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
WO2015123339A1 (en) 2014-02-11 2015-08-20 The Regents Of The University Of Colorado, A Body Corporate Crispr enabled multiplexed genome engineering
US10286084B2 (en) 2014-02-18 2019-05-14 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
WO2015131101A1 (en) 2014-02-27 2015-09-03 Monsanto Technology Llc Compositions and methods for site directed genomic modification
WO2015130968A2 (en) 2014-02-27 2015-09-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
CA2947941C (en) 2014-03-05 2021-02-23 National University Corporation Kobe University Genome sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US9938521B2 (en) 2014-03-10 2018-04-10 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
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US10349639B2 (en) 2014-03-26 2019-07-16 University Of Maryland, College Park Targeted genome editing in zygotes of domestic large animals
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
EP4123024B1 (en) * 2014-04-01 2024-12-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
WO2015153780A1 (en) 2014-04-02 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
KR102596508B1 (ko) 2014-04-10 2023-10-30 10엑스 제노믹스, 인크. 시약을 캡슐화 및 구획화하기 위한 유체 디바이스, 시스템, 및 방법, 및 이의 응용
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
CN106661548B (zh) 2014-05-28 2020-12-11 儿童医院医疗中心 用于经由定向分化将前体细胞转化为胃组织的方法和系统
US10066241B2 (en) 2014-05-30 2018-09-04 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
JP6779866B2 (ja) 2014-06-13 2020-11-04 チルドレンズ メディカル センター コーポレイション ミトコンドリアを単離するための製品および方法
IL286474B2 (en) 2014-06-23 2023-11-01 Massachusetts Gen Hospital Genome-wide random identification of DSBS assessed by sequencing (guide-sequence)
WO2015200555A2 (en) * 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
US12312640B2 (en) 2014-06-26 2025-05-27 10X Genomics, Inc. Analysis of nucleic acid sequences
EP4574974A3 (en) 2014-06-26 2025-10-08 10x Genomics, Inc. Methods of analyzing nucleic acids from individual cells or cell populations
KR20170026383A (ko) 2014-06-26 2017-03-08 10엑스 제노믹스, 인크. 핵산 서열의 분석
AU2015288157A1 (en) 2014-07-11 2017-01-19 E. I. Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
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
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
CN104195177B (zh) * 2014-08-05 2017-06-09 南京大学 一种显著提高鱼类基因组编辑效率的方法
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
EP3183367B1 (en) 2014-08-19 2019-06-26 Pacific Biosciences Of California, Inc. Compositions and methods for enrichment of nucleic acids
DK3186376T3 (da) 2014-08-27 2019-05-06 Caribou Biosciences Inc Fremgangsmåder til forbedring af cas9-medieret manipulationseffektivitet
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
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
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US10040048B1 (en) 2014-09-25 2018-08-07 Synthego Corporation Automated modular system and method for production of biopolymers
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (cg-RX-API-DMAC7.html) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
WO2016069591A2 (en) 2014-10-27 2016-05-06 The Broad Institute Inc. Compositions, methods and use of synthetic lethal screening
US9975122B2 (en) 2014-11-05 2018-05-22 10X Genomics, Inc. Instrument systems for integrated sample processing
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
EP4324473A3 (en) * 2014-11-10 2024-05-29 ModernaTX, Inc. Multiparametric nucleic acid optimization
WO2016086197A1 (en) 2014-11-25 2016-06-02 The Brigham And Women's Hospital, Inc. Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease
KR101833433B1 (ko) * 2014-11-25 2018-02-28 한국생명공학연구원 돼지 t 세포 및 b 세포 면역결핍 세포주 생산 및 그의 제조 방법
EP3224362B1 (en) 2014-11-26 2024-09-25 The Regents of The University of California Therapeutic compositions comprising transcription factors and methods of making and using the same
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
WO2016089866A1 (en) 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
KR102763527B1 (ko) * 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016094679A1 (en) 2014-12-10 2016-06-16 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3786296A1 (en) * 2014-12-18 2021-03-03 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
SG11201705615UA (en) 2015-01-12 2017-08-30 10X Genomics Inc Processes and systems for preparing nucleic acid sequencing libraries and libraries prepared using same
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
EP3250689B1 (en) 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
SI3250691T1 (sl) 2015-01-28 2023-10-30 Caribou Biosciences, Inc. Hibridni dna/rna-polinukleotidi crispr in postopki za uporabo
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EP3262173A2 (en) 2015-02-23 2018-01-03 Crispr Therapeutics AG Materials and methods for treatment of human genetic diseases including hemoglobinopathies
SG11201706766WA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US10697000B2 (en) 2015-02-24 2020-06-30 10X Genomics, Inc. Partition processing methods and systems
EP3262193A2 (en) 2015-02-26 2018-01-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
JP6871169B2 (ja) 2015-03-02 2021-05-12 シナイ ヘルス システム 相同組換え因子
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3274454B1 (en) * 2015-03-25 2021-08-25 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
BR112017017260A2 (pt) 2015-03-27 2018-04-17 Du Pont construções de dna, vetor, célula, plantas, semente, método de expressão de rna e método de modificação de local alvo
CA2981715A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
CA2988854A1 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
US11572543B2 (en) 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
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
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
KR20230110368A (ko) 2015-06-01 2023-07-21 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
CN108368502B (zh) 2015-06-03 2022-03-18 内布拉斯加大学董事委员会 使用单链dna的dna编辑
US20180296537A1 (en) 2015-06-05 2018-10-18 Novartis Ag Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
US20160362667A1 (en) * 2015-06-10 2016-12-15 Caribou Biosciences, Inc. CRISPR-Cas Compositions and Methods
AU2016278959A1 (en) 2015-06-17 2018-01-18 The Uab Research Foundation CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2016205703A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for genomic editing
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
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
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
US9580727B1 (en) 2015-08-07 2017-02-28 Caribou Biosciences, Inc. Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
US10842812B2 (en) 2015-08-12 2020-11-24 The General Hospital Corporation Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
EP4345454A3 (en) * 2015-08-25 2024-07-17 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
EP3341477B1 (en) 2015-08-28 2022-03-23 The General Hospital Corporation Engineered crispr-cas9 nucleases
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
CN108271384B (zh) 2015-09-09 2022-04-15 国立大学法人神户大学 用于特异性转变靶向dna序列的核酸碱基的革兰氏阳性菌的基因组序列的转变方法、及其使用的分子复合体
AU2016319110B2 (en) 2015-09-11 2022-01-27 The General Hospital Corporation Full interrogation of nuclease DSBs and sequencing (FIND-seq)
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
EP3353298B1 (en) * 2015-09-21 2023-09-13 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
US11667911B2 (en) 2015-09-24 2023-06-06 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/CAS-mediated genome editing
WO2017059313A1 (en) 2015-09-30 2017-04-06 The General Hospital Corporation Comprehensive in vitro reporting of cleavage events by sequencing (circle-seq)
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
CN109153980B (zh) 2015-10-22 2023-04-14 布罗德研究所有限公司 Vi-b型crispr酶和系统
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
DK3350327T3 (en) 2015-10-23 2019-01-21 Caribou Biosciences Inc CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075265A1 (en) 2015-10-28 2017-05-04 The Broad Institute, Inc. Multiplex analysis of single cell constituents
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
EP3371306B8 (en) 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2017083852A1 (en) 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies
CA3005633C (en) * 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
WO2017087708A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
US11371094B2 (en) 2015-11-19 2022-06-28 10X Genomics, Inc. Systems and methods for nucleic acid processing using degenerate nucleotides
EP3377657B1 (en) * 2015-11-19 2020-11-11 10X Genomics, Inc. Transformable tagging methods
CN108495932B (zh) * 2015-11-27 2022-08-09 国立大学法人神户大学 用于特异性转换靶向dna序列的核酸碱基的单子叶植物的基因组序列的转换方法、及其使用的分子复合体
EP4644567A2 (en) 2015-11-30 2025-11-05 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017095946A1 (en) 2015-11-30 2017-06-08 Flagship Pioneering, Inc. Methods and compositions relating to chondrisomes
EP4144861B1 (en) 2015-12-04 2024-09-11 10X Genomics, Inc. Methods and compositions for nucleic acid analysis
CA3004757C (en) 2015-12-04 2020-07-21 Caribou Biosciences, Inc. Engineered nucleic-acid targeting nucleic acids
JP6821598B2 (ja) 2015-12-07 2021-01-27 ザイマージェン インコーポレイテッド Ｃｏｒｙｎｅｂａｃｔｅｒｉｕｍ ｇｌｕｔａｍｉｃｕｍ由来のプロモーター
US11208649B2 (en) 2015-12-07 2021-12-28 Zymergen Inc. HTP genomic engineering platform
US9988624B2 (en) 2015-12-07 2018-06-05 Zymergen Inc. Microbial strain improvement by a HTP genomic engineering platform
WO2017106767A1 (en) * 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
US11118194B2 (en) 2015-12-18 2021-09-14 The Regents Of The University Of California Modified site-directed modifying polypeptides and methods of use thereof
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP4219689A3 (en) 2015-12-30 2023-12-20 Novartis AG Immune effector cell therapies with enhanced efficacy
KR102808969B1 (ko) 2016-01-11 2025-05-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 키메라 단백질 및 면역요법 방법
SG11201805792PA (en) 2016-01-11 2018-08-30 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
JP7185527B2 (ja) 2016-01-15 2022-12-07 ザ チルドレンズ メディカル センター コーポレーション ミトコンドリアおよび組み合わされたミトコンドリア剤の治療的使用
US11845933B2 (en) * 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
WO2017139309A1 (en) 2016-02-12 2017-08-17 Ceres, Inc. Methods and materials for high throughput testing of mutagenized allele combinations
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
WO2017147196A1 (en) 2016-02-22 2017-08-31 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
SG11201807025SA (en) * 2016-02-26 2018-09-27 Lanzatech New Zealand Ltd Crispr/cas systems for c-1 fixing bacteria
EP3423083A4 (en) 2016-03-04 2019-10-02 Indoor Biotechnologies Inc. FEL-D-1 KNOCKOUTS AND ASSOCIATED COMPOSITIONS AND METHODS BASED ON CRISPR-CAS9 GENERALIZATION
EP3429567B1 (en) 2016-03-16 2024-01-10 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
DK3433266T3 (da) 2016-03-23 2025-08-25 Univ California Fremgangsmåder til behandling af mitokondrieforstyrrelser
US12011488B2 (en) 2016-03-23 2024-06-18 The Regents Of The University Of California Methods of treating mitochondrial disorders
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
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
KR102424476B1 (ko) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
JP7259182B2 (ja) 2016-04-25 2023-04-18 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 ｉｎ ｓｉｔｕ分子検出のためのハイブリダイゼーション連鎖反応法
WO2017197343A2 (en) 2016-05-12 2017-11-16 10X Genomics, Inc. Microfluidic on-chip filters
WO2017197338A1 (en) 2016-05-13 2017-11-16 10X Genomics, Inc. Microfluidic systems and methods of use
US10767175B2 (en) * 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US10017760B2 (en) 2016-06-24 2018-07-10 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
WO2018005589A1 (en) 2016-06-28 2018-01-04 Cellectis Altering expression of gene products in plants through targeted insertion of nucleic acid sequences
EP3478833A4 (en) 2016-06-30 2019-10-02 Zymergen, Inc. METHOD FOR PRODUCING A BACTERIAL HEMOGLOBIN LIBRARY AND ITS USE
US10544411B2 (en) 2016-06-30 2020-01-28 Zymergen Inc. Methods for generating a glucose permease library and uses thereof
CN109863143B (zh) 2016-07-13 2021-10-15 威泰克斯制药公司 提高基因组编辑效率的方法、组合物和试剂盒
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
AU2017308473B2 (en) 2016-08-12 2021-08-19 Toolgen Incorporated Manipulated immunoregulatory element and immunity altered thereby
US11810649B2 (en) 2016-08-17 2023-11-07 The Broad Institute, Inc. Methods for identifying novel gene editing elements
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11630103B2 (en) 2016-08-17 2023-04-18 The Broad Institute, Inc. Product and methods useful for modulating and evaluating immune responses
EP3500671B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method of selecting target sequences for the design of guide rnas
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
KR20230155013A (ko) 2016-08-20 2023-11-09 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
WO2018057946A2 (en) 2016-09-23 2018-03-29 Board Of Trustees Of Southern Illinois University Tuning crispr/cas9 activity with chemically modified nucleotide substitutions
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
KR20230170126A (ko) 2016-09-30 2023-12-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Rna-가이드된 핵산 변형 효소 및 이의 사용 방법
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
CN110462034A (zh) 2016-10-07 2019-11-15 综合Dna技术公司 化脓链球菌cas9突变基因和由其编码的多肽
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
NZ753051A (en) 2016-11-04 2023-03-31 Children’S Hospital Medical Center Liver organoid compositions and methods of making and using same
EP3535416A4 (en) 2016-11-04 2020-05-13 Flagship Pioneering Innovations V. Inc. NEW PLANT CELLS, PLANTS AND SEEDS
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
KR20250021632A (ko) 2016-11-22 2025-02-13 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Crispr/cpf1 시스템 및 방법
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
WO2018107028A1 (en) 2016-12-08 2018-06-14 Intellia Therapeutics, Inc. Modified guide rnas
WO2018107103A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr-systems for modifying a trait of interest in a plant
US11293022B2 (en) 2016-12-12 2022-04-05 Integrated Dna Technologies, Inc. Genome editing enhancement
KR20240172763A (ko) 2016-12-14 2024-12-10 리간달 인코포레이티드 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
US10815525B2 (en) 2016-12-22 2020-10-27 10X Genomics, Inc. Methods and systems for processing polynucleotides
US10550429B2 (en) 2016-12-22 2020-02-04 10X Genomics, Inc. Methods and systems for processing polynucleotides
WO2018119225A1 (en) 2016-12-22 2018-06-28 Monsanto Technology Llc Genome editing-based crop engineering and production of brachytic plants
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
SG11201906795SA (en) 2017-01-28 2019-08-27 Inari Agriculture Inc Novel plant cells, plants, and seeds
EP4310183B1 (en) 2017-01-30 2025-07-09 10X Genomics, Inc. Methods and systems for droplet-based single cell barcoding
US10995333B2 (en) 2017-02-06 2021-05-04 10X Genomics, Inc. Systems and methods for nucleic acid preparation
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
WO2018148511A1 (en) 2017-02-10 2018-08-16 Zymergen Inc. A modular universal plasmid design strategy for the assembly and editing of multiple dna constructs for multiple hosts
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11739308B2 (en) 2017-03-15 2023-08-29 The Broad Institute, Inc. Cas13b orthologues CRISPR enzymes and systems
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
WO2018183921A1 (en) 2017-04-01 2018-10-04 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
EP3610009A1 (en) 2017-04-12 2020-02-19 The Broad Institute, Inc. Novel type vi crispr orthologs and systems
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
JP7248586B2 (ja) 2017-04-14 2023-03-29 チルドレンズ ホスピタル メディカル センター 複数ドナー幹細胞組成物およびそれを作製する方法
EP3612629A1 (en) 2017-04-18 2020-02-26 The Broad Institute, Inc. Compositions for detecting secretion and methods of use
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
JP7235391B2 (ja) * 2017-05-08 2023-03-08 ツールゲン インコーポレイテッド 人工的に操作された免疫細胞
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209324A2 (en) 2017-05-11 2018-11-15 The Broad Institute, Inc. Methods and compositions of use of cd8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
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
CA3063739A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3063449A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
CN109526228B (zh) 2017-05-26 2022-11-25 10X基因组学有限公司 转座酶可接近性染色质的单细胞分析
US20180340169A1 (en) 2017-05-26 2018-11-29 10X Genomics, Inc. Single cell analysis of transposase accessible chromatin
EP3409104A1 (en) 2017-05-31 2018-12-05 Vilmorin et Cie Tomato plant resistant to tomato yellow leaf curl virus, powdery mildew, and nematodes
EP3409106A1 (en) 2017-06-01 2018-12-05 Vilmorin et Cie Tolerance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus (tbrfv)
WO2020249996A1 (en) 2019-06-14 2020-12-17 Vilmorin & Cie Resistance in plants of solanum lycopersicum to the tobamovirus tomato brown rugose fruit virus
WO2018226602A1 (en) 2017-06-05 2018-12-13 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
EP3635113A4 (en) 2017-06-05 2021-03-17 Fred Hutchinson Cancer Research Center SAFE GENOMIC HAVERS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND NANOPARTICLES MODIFIED TO ENABLE TARGETED GENETIC THERAPIES
WO2018226900A2 (en) 2017-06-06 2018-12-13 Zymergen Inc. A htp genomic engineering platform for improving fungal strains
EP3485013B1 (en) 2017-06-06 2021-03-24 Zymergen, Inc. A htp genomic engineering platform for improving escherichia coli
US10428319B2 (en) 2017-06-09 2019-10-01 Editas Medicine, Inc. Engineered Cas9 nucleases
JP2020524993A (ja) 2017-06-13 2020-08-27 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド クロンを含む組成物及びその使用
US11897953B2 (en) 2017-06-14 2024-02-13 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
US12049643B2 (en) 2017-07-14 2024-07-30 The Broad Institute, Inc. Methods and compositions for modulating cytotoxic lymphocyte activity
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
EP3640333A4 (en) * 2017-07-14 2020-12-30 Cure Genetics Co., Ltd GENE EDITING SYSTEM AND GENE EDITING METHOD
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
US12239646B2 (en) 2017-07-19 2025-03-04 Dana-Farber Cancer Institute, Inc. Cancer diagnostic and treatment
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
JP7581046B2 (ja) 2017-08-23 2024-11-12 ザ ジェネラル ホスピタル コーポレイション 変更されたＰＡＭ特異性を有する遺伝子操作されたＣＲＩＳＰＲ－Ｃａｓ９ヌクレアーゼ
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
US20190076814A1 (en) 2017-09-11 2019-03-14 Synthego Corporation Biopolymer synthesis system and method
US11534471B2 (en) 2017-09-21 2022-12-27 Dana-Farber Cancer Institute, Inc. Isolation, preservation, compositions and uses of extracts from justicia plants
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
CA3073449A1 (en) 2017-09-25 2019-03-28 Agrospheres, Inc. Compositions and methods for scalable production and delivery of biologicals
CN118581083A (zh) * 2017-09-28 2024-09-03 株式会社图尔金 用于基因表达调节的人工基因组操纵
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
WO2019070755A1 (en) 2017-10-02 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
EP3694603A4 (en) 2017-10-10 2021-07-14 Children's Hospital Medical Center ESOPHAGUS TISSUE AND / OR ORGANOID COMPOSITIONS AND METHOD FOR MANUFACTURING THEREOF
US11725228B2 (en) 2017-10-11 2023-08-15 The General Hospital Corporation Methods for detecting site-specific and spurious genomic deamination induced by base editing technologies
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
BR112020007823A2 (pt) 2017-10-20 2020-10-27 Fred Hutchinson Cancer Research Center sistemas e métodos para produzir células b geneticamente modificadas para expressar anticorpos selecionados
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
WO2019084664A1 (en) * 2017-11-02 2019-05-09 The Governors Of The University Of Alberta Chemically-modified guide rnas to improve crispr-cas protein specificity
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
US12221720B2 (en) 2017-11-13 2025-02-11 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
WO2019099751A1 (en) 2017-11-15 2019-05-23 10X Genomics, Inc. Functionalized gel beads
CN111448313A (zh) * 2017-11-16 2020-07-24 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
MX2020004967A (es) 2017-11-17 2020-08-27 Iovance Biotherapeutics Inc Expansion de linfocitos infiltrantes del tumor (til) de aspirados con aguja fina y biopsias por puncion.
US10829815B2 (en) 2017-11-17 2020-11-10 10X Genomics, Inc. Methods and systems for associating physical and genetic properties of biological particles
JP2021503885A (ja) 2017-11-22 2021-02-15 アイオバンス バイオセラピューティクス，インコーポレイテッド 末梢血からの末梢血リンパ球（ｐｂｌ）の拡大培養
MX2020005849A (es) 2017-12-05 2020-11-06 Vertex Pharma Células madre y progenitoras hematopoyéticas humanas cd34+ modificadas con crispr-cas9 y sus usos.
US11332736B2 (en) 2017-12-07 2022-05-17 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US11939575B2 (en) 2017-12-19 2024-03-26 City Of Hope Modified tracrRNAs gRNAs, and uses thereof
US12379372B2 (en) 2017-12-21 2025-08-05 Children's Hospital Medical Center Digitalized human organoids and methods of using same
RU2652899C1 (ru) * 2017-12-28 2018-05-03 Федеральное бюджетное учреждение науки "Центральный научно-исследовательский институт эпидемиологии" Федеральной службы по надзору в сфере защиты прав потребителей и благополучия человека (ФБУН ЦНИИ Эпидемиологии Роспотребнадзора) РНК-проводники для подавления репликации вируса гепатита B и для элиминации вируса гепатита B из клетки-хозяина
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
AU2019207409B2 (en) 2018-01-12 2023-02-23 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
EP3740483B1 (en) 2018-01-17 2023-01-11 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
US11926835B1 (en) 2018-01-29 2024-03-12 Inari Agriculture Technology, Inc. Methods for efficient tomato genome editing
KR20250099758A (ko) 2018-01-31 2025-07-02 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019150196A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CN111699254A (zh) 2018-02-08 2020-09-22 齐默尔根公司 使用crispr在棒状杆菌属中进行基因组编辑
CN112005115A (zh) 2018-02-12 2020-11-27 10X基因组学有限公司 表征来自单个细胞或细胞群体的多种分析物的方法
CN112020554B (zh) 2018-02-23 2024-10-22 先锋国际良种公司 新颖cas9直系同源物
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
PL235163B1 (pl) * 2018-04-05 2020-06-01 Inst Hodowli I Aklimatyzacji Roslin Panstwowy Inst Badawczy Sekwencja nukleotydowa syntetycznego genu Cas9, kaseta kierująca sgRNA do edytowania genomu roślinnego i wydajny system do ukierunkowanej mutagenezy wybranego regionu genomu roślinnego
SG11202009889VA (en) 2018-04-06 2020-11-27 10X Genomics Inc Systems and methods for quality control in single cell processing
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
WO2019204378A1 (en) 2018-04-17 2019-10-24 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
US12338486B2 (en) 2018-04-19 2025-06-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded DNA
EP3781705A4 (en) 2018-04-19 2022-01-26 The Regents of the University of California COMPOSITIONS AND METHODS OF GENE EDIT
SI3560330T1 (sl) 2018-04-24 2022-08-31 KWS SAAT SE & Co. KGaA Rastline z izboljšano prebavljivostjo in markerskimi haplotipi
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
MX2020011339A (es) 2018-04-27 2021-02-09 Genedit Inc Polímero catiónico y uso para suministro de biomolecúla.
WO2019210131A1 (en) 2018-04-27 2019-10-31 Iovance Biotherapeutics, Inc. Closed process for expansion and gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
US12281301B2 (en) 2018-04-27 2025-04-22 The Broad Institute, Inc. Sequencing-based proteomics
EP3788144B1 (en) 2018-05-01 2025-09-24 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
US20210386829A1 (en) 2018-05-04 2021-12-16 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
US12480140B2 (en) 2018-05-06 2025-11-25 Emendobio Inc. Differential knockout of an allele of a heterozygous ELANE gene
WO2019217758A1 (en) 2018-05-10 2019-11-14 10X Genomics, Inc. Methods and systems for molecular library generation
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108707628B (zh) * 2018-05-28 2021-11-23 上海海洋大学 斑马鱼notch2基因突变体的制备方法
WO2019232542A2 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US11866719B1 (en) 2018-06-04 2024-01-09 Inari Agriculture Technology, Inc. Heterologous integration of regulatory elements to alter gene expression in wheat cells and wheat plants
US12286643B2 (en) 2018-06-07 2025-04-29 The Brigham And Women's Hospital, Inc. Methods for generating hematopoietic stem cells
EP3806888B1 (en) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
BR112020025349A2 (pt) 2018-06-15 2021-03-09 KWS SAAT SE & Co. KGaA Métodos para melhorar a engenharia e regeneração do genoma em planta
WO2019238911A1 (en) 2018-06-15 2019-12-19 KWS SAAT SE & Co. KGaA Methods for improving genome engineering and regeneration in plant ii
CN112543812A (zh) 2018-06-26 2021-03-23 麻省理工学院 基于crispr效应系统的扩增方法、系统和诊断
KR20210024009A (ko) 2018-06-26 2021-03-04 더 브로드 인스티튜트, 인코퍼레이티드 Crispr/cas 및 트랜스포사제 기반 증폭 조성물, 시스템 및 방법
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US11629179B2 (en) 2018-06-29 2023-04-18 Stichting Het Nederlands Kanker Instituut—Antoni van Leeuwenhoek Ziekenhuis TWEAK-receptor agonists for use in combination with immunotherapy of a cancer
CN112543810A (zh) 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
CA3105658A1 (en) 2018-07-13 2020-01-16 The Regents Of The University Of California Retrotransposon-based delivery vehicle and methods of use thereof
SG11202100533VA (en) 2018-07-26 2021-02-25 Childrens Hospital Med Ct Hepato-biliary-pancreatic tissues and methods of making same
US20200032335A1 (en) 2018-07-27 2020-01-30 10X Genomics, Inc. Systems and methods for metabolome analysis
KR20210053898A (ko) 2018-07-31 2021-05-12 더 브로드 인스티튜트, 인코퍼레이티드 신규 crispr 효소 및 시스템
EP3830301B1 (en) 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
EP3833761A1 (en) 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems
EP3607819A1 (en) 2018-08-10 2020-02-12 Vilmorin et Cie Resistance to xanthomonas campestris pv. campestris (xcc) in cauliflower
WO2020086144A2 (en) 2018-08-15 2020-04-30 Zymergen Inc. APPLICATIONS OF CRISPRi IN HIGH THROUGHPUT METABOLIC ENGINEERING
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020041387A1 (en) 2018-08-20 2020-02-27 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
WO2020041456A1 (en) 2018-08-22 2020-02-27 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
EP3841203A4 (en) * 2018-08-23 2022-11-02 The Broad Institute Inc. CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND THEIR USES
CN112867792A (zh) 2018-08-23 2021-05-28 桑格摩生物治疗股份有限公司 工程化靶特异性碱基编辑器
US11479762B1 (en) 2018-08-31 2022-10-25 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP3847650A1 (en) 2018-09-06 2021-07-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
US12378572B2 (en) 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
EP4268831A3 (en) 2018-09-12 2024-05-22 Fred Hutchinson Cancer Center Reducing cd33 expression to selectively protect therapeutic cells
SG11202102431YA (en) 2018-09-12 2021-04-29 Childrens Hospital Med Ct Organoid compositions for the production of hematopoietic stem cells and derivatives thereof
EP3852813A4 (en) 2018-09-18 2022-06-22 VNV Newco Inc. ARC-BASED CAPSIDS AND THEIR USES
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
WO2020077236A1 (en) 2018-10-12 2020-04-16 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
US12116458B2 (en) 2018-10-24 2024-10-15 Genedit Inc. Cationic polymer with alkyl side chains and use for biomolecule delivery
WO2020086742A1 (en) 2018-10-24 2020-04-30 Obsidian Therapeutics, Inc. Er tunable protein regulation
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
US11066663B2 (en) 2018-10-31 2021-07-20 Zymergen Inc. Multiplexed deterministic assembly of DNA libraries
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
AU2019375416A1 (en) 2018-11-05 2021-05-27 Iovance Biotherapeutics, Inc. Selection of improved tumor reactive T-cells
ES3021682T3 (en) 2018-11-05 2025-05-27 Iovance Biotherapeutics Inc Tumour infiltrating lymphocytes for use in treating nsclc patients refractory to anti-pd-1 antibodies
JP2022506586A (ja) 2018-11-05 2022-01-17 アイオバンス バイオセラピューティクス，インコーポレイテッド 腫瘍浸潤リンパ球の産生のためのプロセスおよび免疫療法におけるその使用
MX2021004775A (es) 2018-11-05 2021-06-08 Iovance Biotherapeutics Inc Expansion de linfocitos infiltrantes de tumores (tils) usando inhibidores de la via de proteina cinasa b (akt).
SG11202104560XA (en) 2018-11-08 2021-05-28 Triton Algae Innovations Inc Methods for overproducing protoporphyrin ix in algae and compositions therefrom
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
AU2019374883A1 (en) 2018-11-09 2021-05-20 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
US11459607B1 (en) 2018-12-10 2022-10-04 10X Genomics, Inc. Systems and methods for processing-nucleic acid molecules from a single cell using sequential co-partitioning and composite barcodes
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
CA3117228A1 (en) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US20220193131A1 (en) 2018-12-19 2022-06-23 Iovance Biotherapeutics, Inc. Methods of Expanding Tumor Infiltrating Lymphocytes Using Engineered Cytokine Receptor Pairs and Uses Thereof
WO2020142754A2 (en) 2019-01-04 2020-07-09 Mammoth Biosciences, Inc. Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US11845983B1 (en) 2019-01-09 2023-12-19 10X Genomics, Inc. Methods and systems for multiplexing of droplet based assays
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220119871A1 (en) 2019-01-28 2022-04-21 The Broad Institute, Inc. In-situ spatial transcriptomics
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
US20220098621A1 (en) 2019-02-05 2022-03-31 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
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
US11851683B1 (en) 2019-02-12 2023-12-26 10X Genomics, Inc. Methods and systems for selective analysis of cellular samples
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
BR112021016875A2 (pt) 2019-03-01 2022-01-04 Iovance Biotherapeutics Inc Processo para expansão de linfócitos de sangue periférico
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
CA3132840A1 (en) 2019-03-08 2020-09-17 Obsidian Therapeutics, Inc. Human carbonic anhydrase 2 compositions and methods for tunable regulation
JP2022524043A (ja) 2019-03-08 2022-04-27 ザイマージェン インコーポレイテッド 微生物の反復ゲノム編集
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
CA3136061A1 (en) 2019-04-05 2020-10-08 Osaka University Method for producing knock-in cell
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022530224A (ja) 2019-04-23 2022-06-28 ジーンエディット インコーポレイテッド アルキル側鎖を有するカチオン性ポリマー
US11162079B2 (en) 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells
CN114096684B (zh) 2019-05-13 2024-11-19 科沃施种子欧洲股份两合公司 玉米的耐旱性
US20220249559A1 (en) 2019-05-13 2022-08-11 Iovance Biotherapeutics, Inc. Methods and compositions for selecting tumor infiltrating lymphocytes and uses of the same in immunotherapy
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
AU2020282798A1 (en) 2019-05-28 2021-12-16 Genedit Inc. Polymer comprising multiple functionalized sidechains for biomolecule delivery
CN114008203A (zh) 2019-05-29 2022-02-01 孟山都技术公司 使用基因组编辑产生显性等位基因的方法和组合物
WO2020243661A1 (en) 2019-05-31 2020-12-03 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CA3141807A1 (en) 2019-05-31 2020-12-03 Children's Hospital Medical Center Methods of generating and expanding hematopoietic stem cells
WO2020254850A1 (en) 2019-06-21 2020-12-24 Vilmorin & Cie Improvement of quality and permanence of green color of peppers at maturity and over-maturity
AU2020301236A1 (en) 2019-06-25 2021-11-25 Inari Agriculture Technology, Inc. Improved homology dependent repair genome editing
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
WO2021019272A1 (en) 2019-07-31 2021-02-04 Vilmorin & Cie Tolerance to tolcndv in cucumber
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021028359A1 (en) 2019-08-09 2021-02-18 Sangamo Therapeutics France Controlled expression of chimeric antigen receptors in t cells
EP4488287A3 (en) 2019-08-21 2025-03-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
US11116798B2 (en) 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
KR20220052370A (ko) 2019-09-05 2022-04-27 크리스퍼 테라퓨틱스 아게 보편적 공여자 세포
US20220348937A1 (en) 2019-09-06 2022-11-03 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
US12235262B1 (en) 2019-09-09 2025-02-25 10X Genomics, Inc. Methods and systems for single cell protein analysis
AU2020348879A1 (en) 2019-09-20 2022-04-14 Massachusetts Institute Of Technology Novel type VI CRISPR enzymes and systems
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
JP2022548399A (ja) 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020366566A1 (en) 2019-10-17 2022-04-21 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
EP4048295A1 (en) 2019-10-25 2022-08-31 Iovance Biotherapeutics, Inc. Gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
GB201916020D0 (en) 2019-11-04 2019-12-18 Univ Of Essex Enterprise Limited Crispr-mediated identification of biotinylated proteins and chromatin regions
EP4057802A1 (en) 2019-11-14 2022-09-21 Vilmorin & Cie Resistance to acidovorax valerianellae in corn salad
JP7749561B2 (ja) 2019-12-11 2025-10-06 アイオバンス バイオセラピューティクス，インコーポレイテッド 腫瘍浸潤リンパ球（ｔｉｌ）の産生のためのプロセス及びそれを使用する方法
CA3164591A1 (en) 2020-01-13 2021-07-22 William Dowdle Modification of blood type antigens
KR20220130158A (ko) 2020-01-23 2022-09-26 더 칠드런스 메디칼 센터 코포레이션 인간 만능 줄기 세포로부터의 무-간질 t 세포 분화
EP4093863A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation CRISPR-CAS ENZYMES WITH ENHANCED ON-TARGET ACTIVITY
WO2021151073A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
EP4110930A1 (en) 2020-02-28 2023-01-04 KWS SAAT SE & Co. KGaA Method for rapid genome modification in recalcitrant plants
CA3169128A1 (en) 2020-02-28 2021-09-02 Ling MENG Immature inflorescence meristem editing
JP2023517326A (ja) 2020-03-11 2023-04-25 オメガ セラピューティクス， インコーポレイテッド フォークヘッドボックスｐ３（ｆｏｘｐ３）遺伝子発現をモジュレートするための組成物および方法
US12057197B2 (en) 2020-04-03 2024-08-06 Creyon Bio, Inc. Oligonucleotide-based machine learning
WO2021216622A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
US20230165909A1 (en) 2020-04-21 2023-06-01 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
WO2021217082A1 (en) 2020-04-23 2021-10-28 Genedit Inc. Polymer with cationic and hydrophobic side chains
JP2023523855A (ja) 2020-05-04 2023-06-07 アイオバンス バイオセラピューティクス，インコーポレイテッド 腫瘍浸潤リンパ球の製造方法及び免疫療法におけるその使用
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
US20230279418A1 (en) 2020-05-29 2023-09-07 KWS SAAT SE & Co. KGaA Plant haploid induction
WO2021245435A1 (en) 2020-06-03 2021-12-09 Vilmorin & Cie Melon plants resistant to scab disease, aphids and powdery mildew
JP2023530234A (ja) 2020-06-05 2023-07-14 ザ・ブロード・インスティテュート・インコーポレイテッド 新生物を治療するための組成物および方法
US20230276763A1 (en) 2020-06-05 2023-09-07 Vilmorin & Cie Resistance in plants of solanum lycopersicum to the tobrfv
US20230348920A1 (en) 2020-06-29 2023-11-02 KWS SAAT SE & Co. KGaA Boosting homology directed repair in plants
EP4192952A1 (en) 2020-08-10 2023-06-14 Novartis AG Treatments for retinal degenerative diseases
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
EP4221492A1 (en) 2020-10-02 2023-08-09 Vilmorin & Cie Melon with extended shelf life
US20230372397A1 (en) 2020-10-06 2023-11-23 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
WO2022076606A1 (en) 2020-10-06 2022-04-14 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
WO2022090224A1 (en) 2020-10-27 2022-05-05 KWS SAAT SE & Co. KGaA Use of enhanced pol theta activity for eukaryotic genome engineering
WO2022097663A1 (ja) 2020-11-06 2022-05-12 エディットフォース株式会社 ＦｏｋＩヌクレアーゼドメインの変異体
WO2022101286A1 (en) 2020-11-11 2022-05-19 Leibniz-Institut Für Pflanzenbiochemie Fusion protein for editing endogenous dna of a eukaryotic cell
EP4001429A1 (en) 2020-11-16 2022-05-25 Antibodies-Online GmbH Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq)
MA60481B1 (fr) 2020-12-03 2025-04-30 Vilmorin & Cie Plants de tomate résistants au tobrfv, au tmv, au tomv et au tommv, et gènes de résistance correspondants
WO2022133149A1 (en) 2020-12-17 2022-06-23 Iovance Biotherapeutics, Inc. Treatment of cancers with tumor infiltrating lymphocytes
US20240299539A1 (en) 2020-12-17 2024-09-12 Iovance Biotherapeutics, Inc. Treatment with tumor infiltrating lymphocyte therapies in combination with ctla-4 and pd-1 inhibitors
WO2022140605A2 (en) * 2020-12-22 2022-06-30 Ensoma, Inc. Adenoviral serotype 35 helper vectors
IL303892A (en) 2020-12-23 2023-08-01 Flagship Pioneering Innovations V Inc In vitro assembly of RNA-encapsulating enalavirus capsids
AU2021414617A1 (en) 2020-12-31 2023-08-10 Crispr Therapeutics Ag Universal donor cells
EP4284919A1 (en) 2021-01-29 2023-12-06 Iovance Biotherapeutics, Inc. Methods of making modified tumor infiltrating lymphocytes and their use in adoptive cell therapy
TW202304480A (zh) 2021-03-19 2023-02-01 美商艾歐凡斯生物治療公司 腫瘤浸潤淋巴球（ｔｉｌ）中之與ｃｄ３９／ｃｄ６９選擇及基因剔除相關之ｔｉｌ擴增之方法
WO2022213118A1 (en) 2021-03-31 2022-10-06 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
WO2022241408A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating tissue distribution of intracellular therapeutics
WO2022208489A1 (en) 2021-04-02 2022-10-06 Vilmorin & Cie Semi-determinate or determinate growth habit trait in cucurbita
JP2024515189A (ja) 2021-04-19 2024-04-05 アイオバンス バイオセラピューティクス，インコーポレイテッド 細胞免疫療法におけるキメラ共刺激受容体、ケモカイン受容体、及びそれらの使用
CA3217862A1 (en) 2021-05-05 2022-11-10 Radius Pharmaceuticals, Inc. Animal model having homologous recombination of mouse pth1 receptor
CA3217463A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for modulating mrna splicing
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
JP2024519029A (ja) 2021-05-17 2024-05-08 アイオバンス バイオセラピューティクス，インコーポレイテッド Ｐｄ－１遺伝子編集された腫瘍浸潤リンパ球及び免疫療法におけるその使用
KR20240009973A (ko) 2021-05-17 2024-01-23 노그라 파마 리미티드 Il-34 안티센스 작용제 및 이의 사용 방법
GB202107057D0 (en) 2021-05-18 2021-06-30 Univ York Glycosylation method
WO2022243437A1 (en) 2021-05-19 2022-11-24 KWS SAAT SE & Co. KGaA Sample preparation with oppositely oriented guide polynucleotides
CA3222023A1 (en) 2021-06-01 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof
US20250179481A1 (en) 2021-06-01 2025-06-05 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
JP2024525141A (ja) 2021-06-11 2024-07-10 ジーンエディット インコーポレイテッド ポリアミン基およびポリアルキレンオキシド基を有する側鎖を含む生分解性ポリマー
CA3223311A1 (en) 2021-06-18 2022-12-22 Andrea BARGHETTI Compositions and methods for targeting, editing or modifying human genes
GEAP202416440A (en) 2021-06-23 2024-04-25 Entrada Therapeutics Inc Antisense compounds and methods for targeting cug repeats
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
US20240365776A1 (en) 2021-07-22 2024-11-07 Iovance Biotherapeutics, Inc Method for cryopreservation of solid tumor fragments
WO2023009716A1 (en) 2021-07-28 2023-02-02 Iovance Biotherapeutics, Inc. Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with kras inhibitors
US20250179591A1 (en) 2021-07-30 2025-06-05 KWS SAAT SE & Co. KGaA Plants with improved digestibility and marker haplotypes
IL310564A (en) 2021-08-06 2024-03-01 Vilmorin & Cie Resistance to Leveillula Taurica in pepper plant
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
EP4596697A3 (en) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions and methods for nucleic acid modifications
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
EP4166670A1 (en) 2021-10-18 2023-04-19 KWS SAAT SE & Co. KGaA Plant-tag-based weeding control
JP2024541911A (ja) 2021-10-27 2024-11-13 アイオバンス バイオセラピューティクス，インコーポレイテッド 患者特異的免疫療法のための細胞の製造を調整するためのシステム及び方法
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
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
CA3237410A1 (en) 2021-11-10 2023-05-19 Friedrich Graf Finck VON FINCKENSTEIN Methods of expansion treatment utilizing cd8 tumor infiltrating lymphocytes
WO2023093862A1 (en) 2021-11-26 2023-06-01 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
GB202118058D0 (en) 2021-12-14 2022-01-26 Univ Warwick Methods to increase yields in crops
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
US20250059239A1 (en) 2021-12-17 2025-02-20 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
EP4453196A1 (en) 2021-12-21 2024-10-30 Alia Therapeutics Srl Type ii cas proteins and applications thereof
IL313765A (en) 2021-12-22 2024-08-01 Tome Biosciences Inc Joint provision of a gene editor structure and a donor template
WO2023122800A1 (en) 2021-12-23 2023-06-29 University Of Massachusetts Therapeutic treatment for fragile x-associated disorder
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CA3243419A1 (en) 2022-01-28 2023-08-03 Iovance Biotherapeutics, Inc. CYTOKINE-ASSOCIATED TUMOR-INFILTRATING LYMPHOCYTE COMPOSITIONS AND PROCESSES
WO2023150518A1 (en) 2022-02-01 2023-08-10 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023167882A1 (en) 2022-03-01 2023-09-07 Artisan Development Labs, Inc. Composition and methods for transgene insertion
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
EP4256950A1 (en) 2022-04-06 2023-10-11 Vilmorin et Cie Tolerance to cgmmv in cucumber
WO2023196877A1 (en) 2022-04-06 2023-10-12 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
EP4507704A1 (en) 2022-04-15 2025-02-19 Iovance Biotherapeutics, Inc. Til expansion processes using specific cytokine combinations and/or akti treatment
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023219933A1 (en) 2022-05-09 2023-11-16 Entrada Therapeutics, Inc. Compositions and methods for delivery of nucleic acid therapeutics
CA3251533A1 (en) 2022-05-10 2023-11-16 Iovance Biotherapeutics, Inc. TREATMENT OF CANCER PATIENTS WITH TUMOR-INFILTRATING LYMPHOCYTE THERAPIES IN COMBINATION WITH AN IL-15R AGONIST
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
US20250308637A1 (en) 2022-05-20 2025-10-02 Celyntra Therapeutics Sa Systems and methods for assessing risk of genome editing events
KR20250017231A (ko) 2022-05-25 2025-02-04 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 면역 반응을 조절하기 위한 조성물 및 방법
CN120035678A (zh) 2022-05-25 2025-05-23 旗舰创业创新第七有限责任公司 用于调节循环因子的组合物和方法
WO2023230566A2 (en) 2022-05-25 2023-11-30 Flagship Pioneering Innovations Vii, Llc Compositions and methods for modulating cytokines
EP4533088A2 (en) 2022-05-25 2025-04-09 Flagship Pioneering Innovations VII, LLC Compositions and methods for modulating genetic drivers
CN119947754A (zh) 2022-05-25 2025-05-06 旗舰创业创新第七有限责任公司 用于调节肿瘤抑制因子和癌基因的组合物和方法
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
JP7152094B1 (ja) * 2022-06-30 2022-10-12 リージョナルフィッシュ株式会社 ｔｒａｃｒＲＮＡユニット、及びゲノム編集方法
WO2024005864A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024005863A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP4299739A1 (en) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
EP4299733A1 (en) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
CN120344660A (zh) 2022-07-18 2025-07-18 雷纳嘉德医疗管理公司 基因编辑组分、系统和使用方法
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4577660A1 (en) 2022-08-26 2025-07-02 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
KR20240034661A (ko) * 2022-09-06 2024-03-14 주식회사 툴젠 캄필로박터 제주니 유래 Cas9의 가이드 RNA 구조변화를 통한 유전자교정 향상 시스템
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
EP4590688A1 (en) 2022-09-21 2025-07-30 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
WO2024098024A1 (en) 2022-11-04 2024-05-10 Iovance Biotherapeutics, Inc. Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof
EP4612277A1 (en) 2022-11-04 2025-09-10 Iovance Biotherapeutics, Inc. Methods for tumor infiltrating lymphocyte (til) expansion related to cd39/cd103 selection
JP2025537750A (ja) 2022-11-10 2025-11-20 セイル バイオメディシンズ インコーポレイテッド 脂質ナノ粒子または脂質再構成天然メッセンジャーパックを含むｒｎａ組成物
WO2024105162A1 (en) 2022-11-16 2024-05-23 Alia Therapeutics Srl Type ii cas proteins and applications thereof
EP4623072A2 (en) 2022-11-21 2025-10-01 Iovance Biotherapeutics, Inc. Two-dimensional processes for the expansion of tumor infiltrating lymphocytes and therapies therefrom
WO2024118836A1 (en) 2022-11-30 2024-06-06 Iovance Biotherapeutics, Inc. Processes for production of tumor infiltrating lymphocytes with shortened rep step
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024137533A1 (en) 2022-12-19 2024-06-27 Graphite Bio, Inc. Improved peptide inhibitors of p53 binding protein 53bp1
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
IL321652A (en) 2022-12-29 2025-08-01 Vilmorin & Cie Tomato plants resistant to TSWV-resistant varieties and genes suitable for resistance
CN115982034B (zh) * 2022-12-30 2023-11-28 云舟生物科技(广州)股份有限公司 载体构建系统虚拟终端的测试方法、存储介质及电子设备
EP4649147A2 (en) 2023-01-11 2025-11-19 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
AU2024220323A1 (en) 2023-02-15 2025-08-14 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
WO2024175308A1 (en) 2023-02-22 2024-08-29 KWS SAAT SE & Co. KGaA Plants with improved performance
US12225874B2 (en) 2023-03-27 2025-02-18 Redsea Science And Technology Inc. Tomato plant designated ‘X22-31’
CN121013904A (zh) 2023-03-30 2025-11-25 儿童医院医学中心 临床级类器官
WO2024211287A1 (en) 2023-04-03 2024-10-10 Seagen Inc. Production cell lines with targeted integration sites
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
TW202449159A (zh) 2023-04-19 2024-12-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於產生文庫之組合物及方法
WO2024218295A1 (en) 2023-04-21 2024-10-24 Vib Vzw Allelic combinations in crops for yield increase
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
WO2024223900A1 (en) 2023-04-28 2024-10-31 Vilmorin & Cie Genetic determinants conferring improved tobrfv resistance
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
WO2024238977A2 (en) 2023-05-18 2024-11-21 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
AU2024274792A1 (en) 2023-05-23 2025-10-23 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2024253957A1 (en) 2023-06-07 2024-12-12 Northwestern University Targeting autoimmunity-associated t cells through regulation of transcription factors
WO2024263961A2 (en) 2023-06-23 2024-12-26 Children's Hospital Medical Center Methods of matrix-free suspension culture
WO2025006419A1 (en) 2023-06-26 2025-01-02 Flagship Pioneering Innovations Vii, Llc Engineered plasmodia and related methods
WO2025003305A1 (en) 2023-06-28 2025-01-02 Vilmorin & Cie Pepper resistance to meloidogyne enterolobii
WO2025021893A1 (en) 2023-07-24 2025-01-30 Vilmorin & Cie Resistance to cysdv in melons
WO2025027134A1 (en) 2023-08-01 2025-02-06 KWS SAAT SE & Co. KGaA Method of reducing off-flavours in alternative proteins
AR133438A1 (es) 2023-08-01 2025-10-01 Kws Saat Se & Co Kgaa Método para mejorar las propiedades de gelificación de proteínas alternativas
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025054202A1 (en) 2023-09-05 2025-03-13 Sana Biotechnology, Inc. Method of screening a sample comprising a transgene with a unique barcode
WO2025054540A1 (en) 2023-09-08 2025-03-13 Iovance Biotherapeutics, Inc. Methods of gene-editing using programmable nucleases
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
GB202314578D0 (en) 2023-09-22 2023-11-08 Univ Manchester Methods of producing homoplasmic modified plants or parts thereof
WO2025072803A1 (en) 2023-09-29 2025-04-03 Children's Hospital Medical Center Ntrk2 signaling-mediated alveolar capillary injury and repair
WO2025076141A1 (en) 2023-10-03 2025-04-10 Inari Agriculture Technology, Inc. Viral delivery of grna to the scion
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025101484A1 (en) 2023-11-06 2025-05-15 Iovance Biotherapeutics, Inc. Treatment of endometrial cancers with tumor infiltrating lymphocyte therapies
WO2025132866A1 (en) 2023-12-21 2025-06-26 KWS SAAT SE & Co. KGaA Maize plants with improved disease resistance
WO2025151838A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025153582A1 (en) 2024-01-17 2025-07-24 KWS SAAT SE & Co. KGaA Maize plants with improved disease resistance
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025168705A1 (en) 2024-02-08 2025-08-14 Vib Vzw Means and methods for the production of saponins with endosomal escape-enhancing properties
WO2025171210A1 (en) 2024-02-09 2025-08-14 Arbor Biotechnologies, Inc. Compositions and methods for gene editing via homology-mediated end joining
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
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
WO2025186284A1 (en) 2024-03-05 2025-09-12 Vilmorin & Cie Increased yield in cucumber
WO2025196502A1 (en) 2024-03-20 2025-09-25 North Carolina Agricultural & Technical State University Choline kinase inhibitors as a therapeutic treatment for obesity
WO2025212920A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Multi-zonal liver organoids
WO2025217202A1 (en) 2024-04-08 2025-10-16 Children's Hospital Medical Center Bile duct organoid
US20250332260A1 (en) 2024-04-10 2025-10-30 Garuda Therapeutics, Inc. Immune compatible cells for allogeneic cell therapies to cover global, ethnic, or disease-specific populations
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
WO2025240947A1 (en) 2024-05-17 2025-11-20 University Of Massachusetts Therapeutic treatment for fragile x-associated disorder
WO2025259610A1 (en) 2024-06-10 2025-12-18 Northwestern University Genetic modulators of kras protein expression and their uses
US20250382627A1 (en) 2024-06-13 2025-12-18 J.R. Simplot Company Engineering remontant flowering in rosaceae

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004519245A (ja) * 2001-03-16 2004-07-02 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
WO2011064736A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
WO2011146121A1 (en) * 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
JP2012510812A (ja) * 2008-12-04 2012-05-17 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
WO2012149470A1 (en) * 2011-04-27 2012-11-01 Amyris, Inc. Methods for genomic modification
WO2014089290A1 (en) * 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
DE122007000007I1 (de) 1986-04-09 2007-05-16 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US7150982B2 (en) * 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7868149B2 (en) 1999-07-20 2011-01-11 Monsanto Technology Llc Plant genome sequence and uses thereof
US6603061B1 (en) 1999-07-29 2003-08-05 Monsanto Company Agrobacterium-mediated plant transformation method
US20090100536A1 (en) 2001-12-04 2009-04-16 Monsanto Company Transgenic plants with enhanced agronomic traits
EP1994182B1 (en) * 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
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
US8546553B2 (en) 2008-07-25 2013-10-01 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
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
EP3078753B1 (en) 2010-05-10 2018-04-18 The Regents of The University of California Methods using endoribonuclease compositions
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
GB2537000C (en) * 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
EP4556564A3 (en) * 2012-10-23 2025-09-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
PL2771468T3 (pl) * 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US9970024B2 (en) 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004519245A (ja) * 2001-03-16 2004-07-02 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
JP2012510812A (ja) * 2008-12-04 2012-05-17 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
WO2011064736A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
WO2011146121A1 (en) * 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
WO2012149470A1 (en) * 2011-04-27 2012-11-01 Amyris, Inc. Methods for genomic modification
WO2014089290A1 (en) * 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
BIOCHIMICA ET BIOPHYSICA ACTA, 2006, VOL.1758, P.355-363, JPN6017007681, ISSN: 0003913022 *
BIOLOGY DIRECT, 2011, VOL.6, 38, JPN6017046067, ISSN: 0003913021 *
ELIFE, JAN 2013, VOL.2, E00471, JPN6017016126, ISSN: 0003913028 *
FEBS LETT., 2012, 586(15), P.2076-2083, EPUB 2012 FEB 28, JPN6018033752, ISSN: 0003913025 *
MOL. CELL. BIOL., 1987, 7(6), P.2087-2096, JPN6018033753, ISSN: 0003913026 *
NUCLEIC ACIDS RESEARCH, 2011, VOL.39, P.9275-9282, JPN6017046064, ISSN: 0003913020 *
PNAS, 2011, VOL.108, P.E351-E358, JPN6017007683, ISSN: 0003913024 *
SCIENCE, AUG 2012, VOL.337, P.816-821, SUPPLEMENTARY MATERIALS, JPN6016021641, ISSN: 0003913019 *
SCIENCE, JAN 2013, VOL.339, P.823-826, JPN6017016124, ISSN: 0003913027 *
THE JOURNAL OF CELL BIOLOGY, 1988, VOL.107, P.1279-1287, JPN6017007682, ISSN: 0003913023 *

Cited By (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10844378B2 (en) 2012-03-20 2020-11-24 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US11555187B2 (en) 2012-03-20 2023-01-17 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
JP2016093196A (ja) * 2012-12-12 2016-05-26 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
JP2016165307A (ja) * 2012-12-12 2016-09-15 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof
US12252707B2 (en) 2012-12-12 2025-03-18 The Broad Institute, Inc. Delivery, Engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US10930367B2 (en) 2012-12-12 2021-02-23 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
US12018272B2 (en) 2012-12-17 2024-06-25 President And Fellows Of Harvard College RNA-guided human genome engineering
US11359211B2 (en) 2012-12-17 2022-06-14 President And Fellows Of Harvard College RNA-guided human genome engineering
JP2023168564A (ja) * 2012-12-17 2023-11-24 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 Ｒｎａ誘導性ヒトゲノム改変
US11512325B2 (en) 2012-12-17 2022-11-29 President And Fellows Of Harvard College RNA-guided human genome engineering
US11535863B2 (en) 2012-12-17 2022-12-27 President And Fellows Of Harvard College RNA-guided human genome engineering
US11365429B2 (en) 2012-12-17 2022-06-21 President And Fellows Of Harvard College RNA-guided human genome engineering
US10717990B2 (en) 2012-12-17 2020-07-21 President And Fellows Of Harvard College RNA-guided human genome engineering
US10273501B2 (en) 2012-12-17 2019-04-30 President And Fellows Of Harvard College RNA-guided human genome engineering
US10435708B2 (en) 2012-12-17 2019-10-08 President And Fellows Of Harvard College RNA-guided human genome engineering
US11236359B2 (en) 2012-12-17 2022-02-01 President And Fellows Of Harvard College RNA-guided human genome engineering
US9970024B2 (en) 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering
JP7749631B2 (ja) 2012-12-17 2025-10-06 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 Ｒｎａ誘導性ヒトゲノム改変
JP2018075026A (ja) * 2013-03-14 2018-05-17 カリブー・バイオサイエンシーズ・インコーポレイテッド 核酸ターゲティング核酸の組成物および方法
JP2016512048A (ja) * 2013-03-15 2016-04-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ＣＲＩＳＰＲ／Ｃａｓシステムを使用した植物ゲノム操作
US12441995B2 (en) 2013-06-17 2025-10-14 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US10781444B2 (en) 2013-06-17 2020-09-22 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US10711285B2 (en) 2013-06-17 2020-07-14 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US12018275B2 (en) 2013-06-17 2024-06-25 The Broad Institute, Inc. Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy
US10577630B2 (en) 2013-06-17 2020-03-03 The Broad Institute, Inc. Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
US10851357B2 (en) 2013-12-12 2020-12-01 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11407985B2 (en) 2013-12-12 2022-08-09 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12410435B2 (en) 2013-12-12 2025-09-09 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11591581B2 (en) 2013-12-12 2023-02-28 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11597919B2 (en) 2013-12-12 2023-03-07 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US12258595B2 (en) 2013-12-12 2025-03-25 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US12251450B2 (en) 2013-12-12 2025-03-18 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
US11155795B2 (en) 2013-12-12 2021-10-26 The Broad Institute, Inc. CRISPR-Cas systems, crystal structure and uses thereof
US10550372B2 (en) 2013-12-12 2020-02-04 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US11624078B2 (en) 2014-12-12 2023-04-11 The Broad Institute, Inc. Protected guide RNAS (pgRNAS)
US10696986B2 (en) 2014-12-12 2020-06-30 The Board Institute, Inc. Protected guide RNAS (PGRNAS)
US12435320B2 (en) 2014-12-24 2025-10-07 The Broad Institute, Inc. CRISPR having or associated with destabilization domains
US11578312B2 (en) 2015-06-18 2023-02-14 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
US10494621B2 (en) 2015-06-18 2019-12-03 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US10876100B2 (en) 2015-06-18 2020-12-29 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
US12123032B2 (en) 2015-06-18 2024-10-22 The Broad Institute, Inc. CRISPR enzyme mutations reducing off-target effects
US12168789B2 (en) 2015-06-18 2024-12-17 The Broad Institute, Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
JP7197363B2 (ja) 2016-04-14 2022-12-27 ボコ シリコン バレー， インコーポレイテッド ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集
JP2019511240A (ja) * 2016-04-14 2019-04-25 ボコ シリコン バレー， インコーポレイテッド ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集
JP7267013B2 (ja) 2016-06-17 2023-05-01 ザ・ブロード・インスティテュート・インコーポレイテッド Ｖｉ型ｃｒｉｓｐｒオルソログ及び系
JP2023052051A (ja) * 2016-06-17 2023-04-11 ザ・ブロード・インスティテュート・インコーポレイテッド Ｖｉ型ｃｒｉｓｐｒオルソログ及び系
JP2023134453A (ja) * 2016-06-17 2023-09-27 ザ・ブロード・インスティテュート・インコーポレイテッド Ｖｉ型ｃｒｉｓｐｒオルソログ及び系
US11788083B2 (en) 2016-06-17 2023-10-17 The Broad Institute, Inc. Type VI CRISPR orthologs and systems
JP2019524072A (ja) * 2016-06-17 2019-09-05 ザ・ブロード・インスティテュート・インコーポレイテッド Ｖｉ型ｃｒｉｓｐｒオルソログ及び系
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
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

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