CA2906553C - Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci - Google Patents

Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci Download PDF

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Publication number
CA2906553C
CA2906553C CA2906553A CA2906553A CA2906553C CA 2906553 C CA2906553 C CA 2906553C CA 2906553 A CA2906553 A CA 2906553A CA 2906553 A CA2906553 A CA 2906553A CA 2906553 C CA2906553 C CA 2906553C
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protein
cas9
dcas9
expression
sequence
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CA2906553A1 (en
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J. Keith Joung
Morgan Maeder
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General Hospital Corp
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General Hospital Corp
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N9/0004—Oxidoreductases (1.)
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    • C12N9/10—Transferases (2.)
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    • C12N9/14—Hydrolases (3)
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    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
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CA2906553A 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci Active CA2906553C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3161835A CA3161835A1 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361799647P 2013-03-15 2013-03-15
US61/799,647 2013-03-15
US201361838178P 2013-06-21 2013-06-21
US201361838148P 2013-06-21 2013-06-21
US61/838,178 2013-06-21
US61/838,148 2013-06-21
US201361921007P 2013-12-26 2013-12-26
US61/921,007 2013-12-26
PCT/US2014/027335 WO2014152432A2 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

Related Child Applications (1)

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CA3161835A Division CA3161835A1 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

Publications (2)

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CA2906553A1 CA2906553A1 (en) 2014-09-25
CA2906553C true CA2906553C (en) 2022-08-02

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Family Applications (4)

Application Number Title Priority Date Filing Date
CA2906553A Active CA2906553C (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CA2906724A Pending CA2906724A1 (en) 2013-03-15 2014-03-14 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
CA3161835A Pending CA3161835A1 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CA2907198A Active CA2907198C (en) 2013-03-15 2014-03-14 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing

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CA2906724A Pending CA2906724A1 (en) 2013-03-15 2014-03-14 Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
CA3161835A Pending CA3161835A1 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CA2907198A Active CA2907198C (en) 2013-03-15 2014-03-14 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing

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US (17) US10378027B2 (enExample)
EP (10) EP3988667A1 (enExample)
JP (11) JP6657069B2 (enExample)
KR (8) KR102210323B1 (enExample)
CN (6) CN105408497B (enExample)
AU (10) AU2014227653B2 (enExample)
BR (1) BR112015023489B1 (enExample)
CA (4) CA2906553C (enExample)
ES (1) ES2713503T3 (enExample)
IL (2) IL289396B2 (enExample)
WO (4) WO2014144592A2 (enExample)
ZA (1) ZA201506814B (enExample)

Families Citing this family (460)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8053191B2 (en) 2006-08-31 2011-11-08 Westend Asset Clearinghouse Company, Llc Iterative nucleic acid assembly using activation of vector-encoded traits
CN103517990A (zh) 2010-10-07 2014-01-15 通用医疗公司 癌症生物标志物
US10457935B2 (en) 2010-11-12 2019-10-29 Gen9, Inc. Protein arrays and methods of using and making the same
US9295965B2 (en) 2010-11-12 2016-03-29 Gen9, Inc. Methods and devices for nucleic acid synthesis
EP2689019A2 (en) 2011-03-23 2014-01-29 Pioneer Hi-Bred International Inc. Methods for producing a complex transgenic trait locus
US20140274812A1 (en) 2011-07-15 2014-09-18 The General Hospital Corporation Methods of Transcription Activator Like Effector Assembly
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2737957T3 (es) 2011-08-26 2020-01-17 Gen9 Inc Composiciones y métodos para el ensamblaje de alta fidelidad de ácidos nucleicos
EP3604555B1 (en) 2011-10-14 2024-12-25 President and Fellows of Harvard College Sequencing by structure assembly
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
EP2841601B1 (en) 2012-04-24 2019-03-06 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
CA2877823A1 (en) 2012-06-25 2014-01-03 Gen9, Inc. Methods for nucleic acid assembly and high throughput sequencing
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
EP3135765A1 (en) 2012-12-06 2017-03-01 Sigma-Aldrich Co. LLC Crispr-based genome modification and regulation
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
CA2900338C (en) 2013-02-07 2024-11-05 The General Hospital Corporation TRANSCRIPTIONAL ACTIVATORS TALE
SG11201507378UA (en) 2013-03-14 2015-10-29 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US10378027B2 (en) 2013-03-15 2019-08-13 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
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
JP2016522679A (ja) * 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
SG10201710030QA (en) 2013-06-04 2018-01-30 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
ES3029138T3 (en) * 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
JP6665088B2 (ja) 2013-06-17 2020-03-13 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
US20150044772A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
CA3221516A1 (en) 2013-08-22 2015-02-26 E. I. Du Pont De Nemours And Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
ES2844174T3 (es) 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
WO2015066119A1 (en) 2013-10-30 2015-05-07 North Carolina State University Compositions and methods related to a type-ii crispr-cas system in lactobacillus buchneri
CN106459995B (zh) 2013-11-07 2020-02-21 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
EP3835419A1 (en) * 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
RU2016128077A (ru) 2013-12-12 2018-12-06 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
BR112016013547A2 (pt) 2013-12-12 2017-10-03 Broad Inst Inc Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
KR20160102056A (ko) * 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 ｒｎａ
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
EP4063503A1 (en) 2014-02-11 2022-09-28 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
EP3129490A4 (en) 2014-04-08 2017-10-04 North Carolina State University Methods and compositions for rna-directed repression of transcription using crispr-associated genes
KR20170005494A (ko) 2014-05-30 2017-01-13 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법
EP3919621A1 (en) 2014-06-23 2021-12-08 The General Hospital Corporation Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq)
US20150376587A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
EP3760208B1 (en) 2014-06-25 2024-05-29 The General Hospital Corporation Targeting human satellite ii (hsatii)
EP3167071B1 (en) * 2014-07-09 2020-10-07 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
US10179932B2 (en) 2014-07-11 2019-01-15 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
CN106687594A (zh) 2014-07-11 2017-05-17 纳幕尔杜邦公司 用于产生对草甘膦除草剂具有抗性的植物的组合物和方法
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
BR112017003757A2 (pt) 2014-09-12 2017-12-26 Du Pont ?plantas de milho, partes de planta de milho ou sementes de milho?
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
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
US11352666B2 (en) 2014-11-14 2022-06-07 Institute For Basic Science Method for detecting off-target sites of programmable nucleases in a genome
WO2016076672A1 (ko) * 2014-11-14 2016-05-19 기초과학연구원 유전체에서 유전자 가위의 비표적 위치를 검출하는 방법
CN104531633A (zh) * 2014-11-18 2015-04-22 李云英 Cas9-scForkI融合蛋白及其应用
KR20160059994A (ko) 2014-11-19 2016-05-27 기초과학연구원 두 개의 벡터로부터 발현된 Cas9 단백질을 이용한 유전자 발현 조절 방법
WO2016089433A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
CN107208079B (zh) * 2014-12-05 2021-06-29 应用干细胞有限公司 整合转基因的位点定向crispr/重组酶组合物和方法
EP3229586B1 (en) 2014-12-10 2025-07-30 Regents of the University of Minnesota Genetically modified cells, tissues, and organs for treating disease
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3230451B1 (en) 2014-12-12 2021-04-07 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016100951A2 (en) * 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
US20180179523A1 (en) * 2014-12-18 2018-06-28 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
EP3245232B1 (en) 2015-01-12 2021-04-21 The Regents of The University of California Heterodimeric cas9 and methods of use thereof
US11180792B2 (en) 2015-01-28 2021-11-23 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
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
IL254038B2 (en) * 2015-02-18 2023-03-01 Univ Iowa State Res Found Inc Modification of the binding site of transcriptional suppressor in the nf-yc4 promoter to increase protein content and stress resistance
JP6817215B2 (ja) 2015-03-03 2021-01-20 ザ ジェネラル ホスピタル コーポレイション 変更ＰＡＭ特異性を有する遺伝子操作ＣＲＩＳＰＲ−Ｃａｓ９ヌクレアーゼ
EP3553178A1 (en) 2015-03-27 2019-10-16 E. I. du Pont de Nemours and Company Soybean u6 small nuclear rna gene promoters and their use in constitutive expression of small rna genes in plants
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
EP3286571B1 (en) 2015-04-24 2021-08-18 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
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016196361A1 (en) 2015-05-29 2016-12-08 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
CA3000155A1 (en) 2015-05-29 2016-12-08 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
AU2016274784B2 (en) * 2015-06-10 2021-10-28 Firmenich Sa Cell lines for screening odorant and aroma receptors
EP3307893A4 (en) 2015-06-12 2019-08-21 Lonza Walkersville, Inc. PROCESS FOR NUCLEAR REPROGRAMMING WITH SYNTHETIC TRANSCRIPTION FACTORS
JP7051438B2 (ja) 2015-06-15 2022-04-11 ノース カロライナ ステート ユニバーシティ 核酸およびｒｎａに基づく抗菌剤の効率的な送達のための方法および組成物
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
IL293323B2 (en) 2015-06-18 2024-01-01 Massachusetts Inst Technology Crispr enzyme mutations reducing off-target effects
CA2990699A1 (en) * 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
WO2017004279A2 (en) * 2015-06-29 2017-01-05 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
JP7044373B2 (ja) * 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
WO2017015637A1 (en) * 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
WO2017023974A1 (en) * 2015-08-03 2017-02-09 President And Fellows Of Harvard College Cas9 genome editing and transcriptional regulation
WO2017024047A1 (en) * 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 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
WO2017044776A1 (en) * 2015-09-10 2017-03-16 Texas Tech University System Single-guide rna (sgrna) with improved knockout efficiency
CN108350453A (zh) 2015-09-11 2018-07-31 通用医疗公司 核酸酶dsb的完全查询和测序(find-seq)
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
JP2018529353A (ja) 2015-09-30 2018-10-11 ザ ジェネラル ホスピタル コーポレイション 配列決定法による切断事象の包括的生体外報告（ＣＩＲＣＬＥ−ｓｅｑ）
BR112018007070A2 (pt) * 2015-10-06 2019-06-04 Institute For Basic Science método, composição e kit para a preparação de uma planta a partir de um protoplasto, planta regenerada e composição para clivagem de dna
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
EP3350327B1 (en) 2015-10-23 2018-09-26 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
WO2017079406A1 (en) 2015-11-03 2017-05-11 President And Fellows Of Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
EP3374501B1 (en) 2015-11-11 2023-07-12 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
AU2016357319B2 (en) 2015-11-20 2022-03-10 Sage Science, Inc. Preparative electrophoretic method for targeted purification of genomic DNA fragments
US10612044B2 (en) 2015-11-25 2020-04-07 National University Corporation Gunma University DNA methylation editing kit and DNA methylation editing method
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017106251A1 (en) * 2015-12-14 2017-06-22 President And Fellows Of Harvard College Cas discrimination using tuned guide rna
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
US20190010495A1 (en) * 2015-12-28 2019-01-10 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
IL304088A (en) 2016-01-11 2023-08-01 Univ Leland Stanford Junior Systems containing chimeric proteins and their uses for controlling gene expression
MX2018008345A (es) 2016-01-11 2018-12-06 Univ Leland Stanford Junior Proteínas quiméricas y métodos de inmunoterapia.
US11845933B2 (en) 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
JP2019513345A (ja) 2016-02-10 2019-05-30 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 核酸の検知
EP3417061B1 (en) 2016-02-18 2022-10-26 The Regents of the University of California Methods and compositions for gene editing in stem cells
WO2017151444A1 (en) * 2016-02-29 2017-09-08 Agilent Technologies, Inc. Methods and compositions for blocking off-target nucleic acids from cleavage by crispr proteins
CA3016899C (en) * 2016-03-15 2023-09-19 Apse, Inc. Methods and compositions for increased double stranded rna production
WO2017161043A1 (en) 2016-03-16 2017-09-21 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
CN106701765A (zh) * 2016-04-11 2017-05-24 广东赤萌医疗科技有限公司 用于hiv感染治疗的多核苷酸及其制备药物应用
EP3443081A4 (en) * 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
AU2017257624B2 (en) 2016-04-25 2023-05-25 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
CN107326046A (zh) * 2016-04-28 2017-11-07 上海邦耀生物科技有限公司 一种提高外源基因同源重组效率的方法
US20170332610A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
WO2017208247A1 (en) * 2016-06-02 2017-12-07 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
LT3604527T (lt) 2016-06-02 2021-06-25 Sigma-Aldrich Co., Llc Programuojamų, dnr surišančių baltymų panaudojimas tiksliniam genomo modifikavimui pagerinti
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN109312386B (zh) * 2016-06-15 2022-10-25 株式会社图尔金 使用中靶靶标和脱靶靶标的多重靶标系统筛选靶特异性核酸酶的方法及其用途
ES2915562T3 (es) 2016-06-24 2022-06-23 Univ Colorado Regents Métodos para generar bibliotecas combinatorias con código de barras
WO2018005445A1 (en) 2016-06-27 2018-01-04 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
EP3478852B1 (en) 2016-07-01 2020-08-12 Microsoft Technology Licensing, LLC Storage through iterative dna editing
EP3484870B1 (en) 2016-07-13 2022-11-16 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
CN109477086A (zh) * 2016-07-13 2019-03-15 陈奇涵 一种基因组dna特异性编辑方法和应用
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
US11168313B2 (en) 2016-07-26 2021-11-09 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
EP3492096A4 (en) * 2016-07-28 2020-04-15 Institute for Basic Science PHARMACEUTICAL COMPOSITION FOR THE TREATMENT OF EYE DISEASES WITH CAS9 PROTEINS AND GUIDE RNA
KR20190041476A (ko) 2016-07-29 2019-04-22 리제너론 파마슈티칼스 인코포레이티드 C-절단된 피브릴린-1의 발현을 유도하는 돌연변이를 포함하는 마우스
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
EP3498834A4 (en) 2016-08-10 2020-06-17 Genahead Bio, Inc. METHOD FOR MODIFYING A DESTINATION IN THE GENOM OF EUKARYOTIC CELLS AND METHOD FOR DETECTING THE PRESENCE OR ABSENCE OF A NUCLEIC ACID SEQUENCE TO BE DETECTED AT THE DESTINATION
WO2018034554A1 (ko) 2016-08-19 2018-02-22 주식회사 툴젠 인위적으로 조작된 신생혈관형성 조절 시스템
CN109863246A (zh) * 2016-08-19 2019-06-07 怀特黑德生物医学研究所 编辑dna甲基化的方法
BR112019003100A2 (pt) 2016-08-24 2019-07-09 Sangamo Therapeutics, Inc. regulação da expressão gênica usando nucleases manipuladas
IL300783B2 (en) 2016-08-24 2026-03-01 Sangamo Therapeutics Inc Engineered nucleases with target-specific zinc fingers
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2569252A (en) 2016-08-31 2019-06-12 Harvard College Methods of combining the detection of biomolecules into a single assay using fluorescent in situ sequencing
WO2018048194A1 (ko) * 2016-09-07 2018-03-15 울산대학교 산학협력단 dCas9 단백질 및 표적 핵산 서열에 결합하는 gRNA를 이용한 핵산 검출의 민감도 및 특이도 향상용 조성물 및 방법
WO2018049075A1 (en) * 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
CA3105873A1 (en) * 2016-09-30 2018-04-05 Poseida Therapeutics, Inc. Modified stem cell memory t cells, methods of making and methods of using same
CN107880132B (zh) * 2016-09-30 2022-06-17 北京大学 一种融合蛋白及使用其进行同源重组的方法
JP2019532644A (ja) 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
CA3039409A1 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
EP3525832A4 (en) 2016-10-14 2020-04-29 The General Hospital Corp. Epigenetically regulated site-specific nucleases
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
GB2573406B (en) 2016-10-18 2021-11-10 Univ Minnesota Tumor infiltrating lymphocytes and methods of therapy
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
US20180282722A1 (en) * 2016-11-21 2018-10-04 Massachusetts Institute Of Technology Chimeric DNA:RNA Guide for High Accuracy Cas9 Genome Editing
AU2017364084B2 (en) 2016-11-22 2024-07-04 Integrated Dna Technologies, Inc. CRISPR/Cpf1 systems and methods
MY206324A (en) 2016-12-08 2024-12-10 Intellia Therapeutics Inc Modified guide rnas
US11293022B2 (en) 2016-12-12 2022-04-05 Integrated Dna Technologies, Inc. Genome editing enhancement
WO2018111944A1 (en) 2016-12-12 2018-06-21 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
US20180179553A1 (en) 2016-12-14 2018-06-28 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload delivery
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
CA3047415A1 (en) * 2016-12-22 2018-06-28 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US20190382751A1 (en) * 2016-12-28 2019-12-19 Ionis Pharmaceuticals, Inc. Modified crispr rna and uses thereof
JP7219972B2 (ja) 2017-01-05 2023-02-09 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー Ｄｎａ二本鎖切断に非依存的な標的化遺伝子編集プラットフォームおよびその用途
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
US20180258418A1 (en) * 2017-01-17 2018-09-13 Institute For Basic Science Method of identifying genome-wide off-target sites of base editors by detecting single strand breaks in genomic dna
SG11201906735RA (en) 2017-01-23 2019-08-27 Regeneron Pharma Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
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
CA3049989A1 (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
US11518991B2 (en) 2017-02-24 2022-12-06 Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts, Universitaetsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated RASAL1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including RASAL1 in a subject
EP3592768B1 (en) 2017-03-08 2024-12-25 The Regents of The University of Michigan Analyte detection
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN108660161B (zh) * 2017-03-31 2023-05-09 中国科学院脑科学与智能技术卓越创新中心 基于CRISPR/Cas9技术的制备无嵌合基因敲除动物的方法
WO2018187779A1 (en) 2017-04-07 2018-10-11 Sage Science, Inc. Systems and methods for detection of genetic structural variation using integrated electrophoretic dna purification
WO2018191603A1 (en) 2017-04-14 2018-10-18 University Of Massachusetts Targeting cell tropism receptors to inhibit cytomegalovirus infection
ES2992912T3 (en) 2017-04-21 2024-12-19 Massachusetts Gen Hospital Variants of cpf1 (cas12a) with altered pam specificity
CN110799205A (zh) 2017-04-21 2020-02-14 通用医疗公司 利用CRISPR-Cpf1的可诱导、可调和多重的人类基因调节
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
AU2018273986A1 (en) 2017-05-25 2019-12-12 The General Hospital Corporation Bipartite base editor (BBE) architectures and type-II-C-Cas9 zinc finger editing
WO2018226560A1 (en) 2017-06-05 2018-12-13 Regeneron Pharmaceuticals, Inc. B4galt1 variants and uses thereof
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
JP7518620B2 (ja) 2017-06-09 2024-07-18 エディタス・メディシン，インコーポレイテッド 操作されたｃａｓ９ヌクレアーゼ
MX2019015143A (es) 2017-06-14 2020-07-27 Wisconsin Alumni Res Found Rna guias modificados, complejos crispr-ribonucleoproteina y metodos de uso.
SG11201912179SA (en) 2017-06-15 2020-01-30 Univ California Targeted non-viral dna insertions
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
KR20240046316A (ko) * 2017-07-11 2024-04-08 시그마-알드리치 컴퍼니., 엘엘씨 표적된 게놈 변형을 개선하기 위한 뉴클레오솜 상호작용 단백질 도메인 사용
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
JP2020530990A (ja) 2017-07-31 2020-11-05 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． ｉｎ ｖｉｖｏでの外因性ドナー核酸とのＣＲＩＳＰＲ／Ｃａｓ誘導性組換えの評価
CA3068072A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing crispr/cas-mediated disruption or excision and crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
CN110891420B (zh) 2017-07-31 2022-06-03 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
US11897920B2 (en) 2017-08-04 2024-02-13 Peking University Tale RVD specifically recognizing DNA base modified by methylation and application thereof
WO2019028686A1 (zh) 2017-08-08 2019-02-14 北京大学 基因敲除方法
CA3073448A1 (en) * 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
JP7139419B2 (ja) 2017-09-29 2022-09-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化ｔｔｒ遺伝子座を含む非ヒト動物および使用方法
WO2019075197A1 (en) 2017-10-11 2019-04-18 The General Hospital Corporation METHODS OF DETECTION OF INDIVIDUAL SITE-SPECIFIC PARASITE GENOMIC DEAMINATION BY BASE EDITING TECHNOLOGIES
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
CN107602707B (zh) * 2017-10-17 2021-04-23 湖北大学 一种特异性调节枯草芽孢杆菌外源基因表达的dcas9-ω融合蛋白及其应用
WO2019084552A1 (en) 2017-10-27 2019-05-02 The Regents Of The University Of California TARGETED REPLACEMENT OF ENDOGENIC T CELL RECEPTORS
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
CN111527209B (zh) * 2017-11-01 2024-11-12 爱迪塔斯医药股份有限公司 Crispr-cas9编辑t细胞中的tgfbr2以用于免疫疗法的方法、组合物和组分
WO2019099943A1 (en) 2017-11-16 2019-05-23 Astrazeneca Ab Compositions and methods for improving the efficacy of cas9-based knock-in strategies
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR102098915B1 (ko) * 2017-12-22 2020-04-09 (주)지플러스 생명과학 키메라 게놈 조작 분자 및 방법
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
US11718849B2 (en) 2018-02-19 2023-08-08 Agilent Technologies, Inc. Phosphopeptide-encoding oligonucleotide libraries and methods for detecting phosphorylation-dependent molecular interactions
EP3755792A4 (en) 2018-02-23 2021-12-08 Pioneer Hi-Bred International, Inc. NEW CAS9 ORTHOLOGIST
KR102907245B1 (ko) 2018-03-14 2026-01-05 에디타스 메디신, 인코포레이티드 혈색소병증 치료를 위한 시스템 및 방법
KR20240038811A (ko) 2018-03-19 2024-03-25 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
CN116536272A (zh) 2018-04-06 2023-08-04 儿童医疗中心有限公司 用于体细胞重新编程和调整印记的组合物和方法
EP3781585A4 (en) 2018-04-17 2022-01-26 The General Hospital Corporation SENSITIVE IN VITRO TESTS FOR SUBSTRATE PREFERENCES AND SITE FOR NUCLEIC ACID BINDERS, MODIFIERS AND CLEAVAGE AGENTS
GB2587970B (en) 2018-04-19 2023-02-08 Univ California Compositions and methods for gene editing
WO2019213430A1 (en) * 2018-05-03 2019-11-07 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for nicking target dna sequences
CN108588123A (zh) * 2018-05-07 2018-09-28 南京医科大学 CRISPR/Cas9载体组合在制备基因敲除猪的血液制品中的应用
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3794130A4 (en) 2018-05-16 2022-07-27 Synthego Corporation METHODS AND SYSTEMS FOR DESIGNING AND USING GUIDE RNA
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3575396A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
CN110592141B (zh) * 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
JP2021530212A (ja) 2018-07-13 2021-11-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California レトロトランスポゾンベースの送達媒体及びその使用方法
EP3821020A4 (en) 2018-08-15 2022-05-04 Zymergen Inc. APPLICATIONS OF CRISPRI IN HIGH-PERFORMANCE METABOLIC ENGINEERING
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California Variant type v crispr/cas effector polypeptides and methods of use thereof
EP3841204A4 (en) 2018-08-23 2022-05-18 Sangamo Therapeutics, Inc. CHANGED TARGET SPECIFIC BASE EDITORS
EP3847251A1 (en) 2018-09-07 2021-07-14 Astrazeneca AB Compositions and methods for improved nucleases
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
WO2020072250A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for genome modification and alteration of expression
WO2020072253A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for screening for variant cells
US10711267B2 (en) 2018-10-01 2020-07-14 North Carolina State University Recombinant type I CRISPR-Cas system
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases
EP3877517A4 (en) 2018-11-09 2022-09-07 Inari Agriculture, Inc. RNA-DRIVEN NUCLEASES AND DNA-BINDING PROTEINS
CN113166744B (zh) 2018-12-14 2025-02-07 先锋国际良种公司 用于基因组编辑的新颖crispr-cas系统
US11690362B2 (en) 2018-12-20 2023-07-04 Regeneran Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
EP3911746A1 (en) 2019-01-14 2021-11-24 Institut National de la Santé et de la Recherche Médicale (INSERM) Methods and kits for generating and selecting a variant of a binding protein with increased binding affinity and/or specificity
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3917573A4 (en) 2019-01-28 2022-12-07 The Administrators of The Tulane Educational Fund CRISPR-BASED METHODS AND NOVEL COMPOSITIONS FOR THE TREATMENT OF VASCULAR DISEASES
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation VARIANTS OF ADENINE DNA BASES WITH REDUCED OFF-TARGET RNA EDIT
WO2020163856A1 (en) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Modified mitochondrion and methods of use thereof
EP4219700A1 (en) 2019-03-07 2023-08-02 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
CA3127814C (en) 2019-03-18 2024-05-14 Regeneron Pharmaceuticals, Inc. Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
US11845931B2 (en) 2019-03-18 2023-12-19 Regeneron Pharmaceuticals, Inc. CRISPR/Cas screening platform to identify genetic modifiers of tau seeding or aggregation
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
JP7524214B2 (ja) 2019-04-03 2024-07-29 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 抗体コード配列をセーフハーバー遺伝子座に挿入するための方法および組成物
AU2020253531C1 (en) 2019-04-04 2022-06-16 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
ES2966625T3 (es) 2019-04-04 2024-04-23 Regeneron Pharma Roedores que comprenden un locus del factor de coagulación 12 humanizado
WO2020208185A1 (en) 2019-04-12 2020-10-15 Astrazeneca Ab Compositions and methods for improved gene editing
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
CA3137764A1 (en) 2019-06-07 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
KR20220024053A (ko) 2019-06-14 2022-03-03 리제너론 파마슈티칼스 인코포레이티드 타우병증의 모델
WO2021026294A1 (en) 2019-08-06 2021-02-11 Children's Medical Center Corporation Methods and compositions for reconstituting microglia
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
US20210079394A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
AU2020352552A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
LT3812472T (lt) 2019-10-21 2023-03-10 Albert-Ludwigs-Universität Freiburg Objektyviai nešališkas tyrimas in vitro sąlygomis, skirtas vienos arba daugiau tikslinių programuojamų nukleazių ląstelėje nespecifinės sąveikos aktyvumui profiliuoti (abnoba-seq)
JP7756639B2 (ja) 2019-11-08 2025-10-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略
US11331333B2 (en) 2019-11-08 2022-05-17 Georg-August-Universität Göttingen Stiftung Öffentichen Rechts, Universitätsmadizin Treatment of aberrant fibroblast proliferation
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021108561A1 (en) * 2019-11-25 2021-06-03 La Jolla Institute For Immunology Methods and compositions for modulating heterochromatin dysfunction, genomic instability, and associated conditions
BR112022011214A2 (pt) 2019-12-11 2022-08-23 Intellia Therapeutics Inc Rnas-guia modificados para edição de gene
CN111088357B (zh) * 2019-12-31 2022-09-20 深圳大学 针对escc的肿瘤标志物及其应用
WO2021151073A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
CN115362264A (zh) 2020-01-29 2022-11-18 10X基因组学有限公司 用于分析物检测的组合物和方法
US12129496B2 (en) 2020-02-12 2024-10-29 Massachusetts Eye And Ear Infirmary Haplotype-based treatment of RP1 associated retinal degenerations
CA3167369A1 (en) 2020-03-04 2021-09-10 Tara YOUNG Methods and compositions for sensitization of tumor cells to immune therapy
EP4114952A4 (en) * 2020-03-05 2024-05-08 Board of Regents of the University of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
WO2021183720A1 (en) * 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
WO2021204877A2 (en) * 2020-04-08 2021-10-14 Astrazeneca Ab Compositions and methods for improved site-specific modification
GB2613457B (en) * 2020-04-09 2023-12-06 Verve Therapeutics Inc Base editing of angptl3 and methods of using same for treatment of disease
MX2022013879A (es) * 2020-05-04 2023-02-01 Editas Medicine Inc Selección por inserción génica en genes esenciales.
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
JP7856581B2 (ja) 2020-05-13 2026-05-11 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル β－ヘモグロビン異常症の処置のための塩基編集アプローチ
WO2021246165A1 (ja) * 2020-06-03 2021-12-09 国立大学法人広島大学 Ｏａｓｉｓ遺伝子の脱メチル化のための核酸及びそれを用いた脱メチル化方法
AU2021282659A1 (en) * 2020-06-05 2023-01-05 The Regents Of The University Of California Compositions and methods for epigenome editing
WO2022008935A1 (en) 2020-07-10 2022-01-13 Horizon Discovery Limited Method for producing genetically modified cells
EP4196595B1 (en) 2020-08-11 2025-03-12 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Method for the treatment of wwox associated diseases
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
US20240016855A1 (en) 2020-10-13 2024-01-18 Centre National De La Recherche Scientifique (Cnrs) Targeted-antibacterial-plasmids combining conjugation and crispr/cas systems and uses thereof
CN112430622A (zh) * 2020-10-26 2021-03-02 扬州大学 一种FokI和dCpf1融合蛋白表达载体及其介导的定点基因编辑方法
RU2762831C1 (ru) * 2020-10-26 2021-12-23 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) Молекула рнк-проводника для геномного редактирования протомоторной области гена vrn-a1 однодольных зерновых с применением системы crispr/cas9
EP4256052A1 (en) 2020-12-02 2023-10-11 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
KR20220082186A (ko) 2020-12-10 2022-06-17 한세준 유,무선 충전이 가능한 보조배터리형 uv-led살균기
CA3207144A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Limited Method for producing genetically modified cells
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022180153A1 (en) 2021-02-25 2022-09-01 INSERM (Institut National de la Santé et de la Recherche Médicale) Allele-specific genome editing of the nr2e3 mutation g56r
KR102882704B1 (ko) * 2021-03-03 2025-11-12 중앙대학교 산학협력단 CRISPR/Cas9 시스템을 이용한 유전체 단일염기 편집 방법 및 이의 용도
CN113846019B (zh) * 2021-03-05 2023-08-01 海南师范大学 一种海洋微拟球藻靶向表观基因组遗传调控方法
EP4095243A1 (en) 2021-05-25 2022-11-30 European Molecular Biology Laboratory System for hybridization-based precision genome cleavage and editing, and uses thereof
WO2022248645A1 (en) 2021-05-27 2022-12-01 Astrazeneca Ab Cas9 effector proteins with enhanced stability
EP4352225A1 (en) 2021-06-10 2024-04-17 Intellia Therapeutics, Inc. Modified guide rnas comprising an internal linker for gene editing
US20240309360A1 (en) * 2021-07-14 2024-09-19 The Governing Council Of The University Of Toronto Trans-activators and methods and use thereof
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
CA3229450A1 (en) 2021-08-20 2023-02-23 Wisconsin Alumni Research Foundation Nonviral generation of genome edited chimeric antigen receptor t cells
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
EP4596697A3 (en) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions and methods for nucleic acid modifications
WO2023052366A1 (en) 2021-09-28 2023-04-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
EP4408996A2 (en) 2021-09-30 2024-08-07 Astrazeneca AB Use of inhibitors to increase efficiency of crispr/cas insertions
IL311570A (en) 2021-10-14 2024-05-01 Arsenal Biosciences Inc Immune cells with common expression chain and logic gate systems
AU2022368907A1 (en) 2021-10-20 2024-05-02 University Of Copenhagen Rejuvenation treatment of age-related white matter loss
JP2024540086A (ja) 2021-10-28 2024-10-31 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 Ｃ５をノックアウトするためのｃｒｉｓｐｒ／ｃａｓ関連方法及び組成物
EP4426832A1 (en) 2021-11-03 2024-09-11 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Precise genome editing using retrons
MX2024005242A (es) 2021-11-03 2024-07-02 Intellia Therapeutics Inc Polinucleotidos, composiciones y metodos para la edicion del genoma.
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
US12582104B2 (en) 2021-12-08 2026-03-24 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
CA3247927A1 (en) 2022-01-14 2023-07-20 Tune Therapeutics, Inc. Compositions, systems and methods for programming T-lymphocyte phenotypes by targeted gene repression
US20250134999A1 (en) 2022-01-14 2025-05-01 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
WO2023141487A1 (en) * 2022-01-20 2023-07-27 Inari Agriculture Technology, Inc. Improved soybean explant preparation and transformation
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023144104A1 (en) 2022-01-25 2023-08-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of βeta-thalassemia
CA3242731A1 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
EP4479534A1 (en) 2022-02-14 2024-12-25 Institut National de la Santé et de la Recherche Médicale (INSERM) Treatment of liver cancers by disrupting the beta-catenin/tcf-4 binding site located upstream of meg3 in the dlk1/dio3 locus
EP4514981A2 (en) 2022-04-29 2025-03-05 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
EP4522726A1 (en) 2022-05-09 2025-03-19 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
EP4522647A1 (en) 2022-05-10 2025-03-19 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia
JP2025521154A (ja) 2022-05-31 2025-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
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
WO2024018056A1 (en) 2022-07-22 2024-01-25 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from βeta-thalassemia
KR20250054842A (ko) 2022-07-29 2025-04-23 리제너론 파마슈티칼스 인코포레이티드 뇌 및 근육으로의 트랜스페린 수용체(tfr)-매개 전달을 위한 조성물 및 방법
CN120435557A (zh) 2022-08-19 2025-08-05 图恩疗法股份有限公司 通过靶向基因阻遏调节乙型肝炎病毒的组合物、系统和方法
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
WO2024064642A2 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
CA3265590A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. MODIFIED ANTIBODY-RESISTANT RECEPTORS TO IMPROVE CELL-BASED THERAPIES
EP4605525A1 (en) 2022-10-21 2025-08-27 Keygene N.V. Rna transfection in plant cells with modified rna
CN120693347A (zh) 2022-11-04 2025-09-23 瑞泽恩制药公司 钙电压门控通道辅助亚基γ1(CACNG1)结合蛋白和CACNG1介导的向骨骼肌的递送
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
CN115820603B (zh) * 2022-11-15 2024-07-05 吉林大学 一种基于dCasRx-NSUN6单基因特异性M5C修饰编辑方法
WO2024121354A1 (en) 2022-12-08 2024-06-13 Keygene N.V. Duplex sequencing with covalently closed dna ends
TW202440931A (zh) * 2022-12-23 2024-10-16 大陸商益杰立科（上海）生物科技有限公司 融合物及其用途
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
JP2026506554A (ja) 2023-02-06 2026-02-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 多重塩基編集による遺伝子改変造血幹細胞の濃縮
CN116376975B (zh) * 2023-02-27 2024-05-14 中国科学院脑科学与智能技术卓越创新中心 激活异染色质基因的方法及应用
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
CN118684781A (zh) * 2023-03-21 2024-09-24 深圳赫兹生命科学技术有限公司 GnRH-VLP重组去势疫苗及其制备方法
EP4689155A1 (en) 2023-03-29 2026-02-11 Astrazeneca AB Use of inhibitors to increase efficiency of crispr/cas insertions
EP4689157A1 (en) 2023-04-04 2026-02-11 Keygene N.V. Linkers for duplex sequencing
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
KR20260032927A (ko) 2023-06-30 2026-03-10 리제너론 파마슈티칼스 인코포레이티드 상동성 지향 복구를 증가시키는 방법 및 조성물
WO2025017030A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
WO2025017033A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법
TW202513801A (zh) 2023-07-28 2025-04-01 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入
KR20260046119A (ko) 2023-07-28 2026-04-06 리제너론 파마슈티칼스 인코포레이티드 단방향 유전자 삽입 동안 도입유전자 발현을 증진시키는 bGH-SV40L 직렬 PolyA의 용도
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
TW202521564A (zh) 2023-08-14 2025-06-01 美商英特利亞醫療公司 用於基於細胞之療法的ｃｄ７０ ｃａｒ－ｔ組合物及方法
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
AU2024357861A1 (en) 2023-10-12 2026-04-23 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025202473A1 (en) 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2025261475A1 (zh) * 2024-06-21 2025-12-26 益杰立科(上海)生物科技有限公司 靶向血管内皮生长因子的工程化基因转录阻抑工具及用途
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026077659A1 (en) 2024-09-17 2026-04-16 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through epigenome editing
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence
WO2026080515A2 (en) 2024-10-08 2026-04-16 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cells and cell lines and methods of use thereof
WO2026083309A1 (en) 2024-10-17 2026-04-23 Astrazeneca Ab Use of inhibitors or modulators of artemis in gene editing
CN121554544B (zh) * 2026-01-23 2026-04-28 成都医学院 一种NFT蛋白及其制备方法和应用，以及一种NusA或NusA融合蛋白的纯化方法

Family Cites Families (166)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo Improvements in or relating to binding proteins for recognition of dna
US20030017149A1 (en) 1996-10-10 2003-01-23 Hoeffler James P. Single chain monoclonal antibody fusion reagents that regulate transcription in vivo
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
HK1049515B (en) 1999-12-06 2006-01-13 Sangamo Biosciences Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
WO2001083819A2 (en) 2000-04-28 2001-11-08 Sangamo Biosciences, Inc. Methods for designing exogenous regulatory molecules
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
AU2003219847A1 (en) 2002-02-21 2003-09-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
AU2003274404A1 (en) 2002-06-11 2003-12-22 The Scripps Research Institute Artificial transcription factors
WO2004099366A2 (en) 2002-10-23 2004-11-18 The General Hospital Corporation Context sensitive parallel optimization of zinc finger dna binding domains
US7021555B2 (en) 2004-01-06 2006-04-04 Zoo Med Laboratories, Inc. Spraying/misting for plants and animals
US7919277B2 (en) 2004-04-28 2011-04-05 Danisco A/S Detection and typing of bacterial strains
SI2351772T1 (sl) 2005-02-18 2016-11-30 Glaxosmithkline Biologicals Sa Proteini in nukleinske kisline iz Escherichia coli povezane z meningitisom/sepso
WO2007014181A2 (en) * 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
NZ597299A (en) * 2005-08-26 2013-03-28 Dupont Nutrition Biosci Aps Use of a cas gene in combination with CRISPR repeats for modulating resistance in a cell
EP1963499A4 (en) 2005-11-28 2009-04-08 Scripps Research Inst ZINCFINGER BINDING DOMAIN FOR TNN
US9399801B2 (en) 2006-05-19 2016-07-26 Dupont Nutrition Biosciences Aps Tagged microorganisms and methods of tagging
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
EP2034848B1 (en) 2006-06-16 2016-10-19 DuPont Nutrition Biosciences ApS Streptococcus thermophilus bacterium
US9201063B2 (en) 2006-11-16 2015-12-01 General Electric Company Sequential analysis of biological samples
WO2008093152A1 (en) * 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
PT2126130E (pt) 2007-03-02 2015-08-03 Dupont Nutrition Biosci Aps Culturas com resistência melhorada a fagos
WO2008118394A1 (en) 2007-03-23 2008-10-02 New York University Methods of affecting nitrogen assimilation in plants
JP5258874B2 (ja) 2007-04-10 2013-08-07 キアゲン ゲゼルシャフト ミット ベシュレンクテル ハフツング Ｒｎａ干渉タグ
WO2008151032A2 (en) 2007-05-31 2008-12-11 Washington University In St. Louis Arrays and methods comprising m. smithii gene products
WO2009041832A2 (en) 2007-09-25 2009-04-02 Pastoral Greenhouse Gas Research Ltd Vaccines and vaccine components for inhibition of microbial cells
FR2925918A1 (fr) 2007-12-28 2009-07-03 Pasteur Institut Typage et sous-typage moleculaire de salmonella par identification des sequences nucleotidiques variables des loci crispr
FR2930264B1 (fr) 2008-04-18 2013-02-22 Gervais Danone Sa Nouvelle souche de lactobacillus paracasei subsp. paracasei dotee de proprietes antimicrobiennes et immunomodulatrices.
JP2010017179A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010017178A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
JP2010068800A (ja) 2008-08-19 2010-04-02 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010048566A (ja) 2008-08-19 2010-03-04 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010037001A2 (en) * 2008-09-26 2010-04-01 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins
CA2741351C (en) 2008-10-21 2018-01-23 Animal Health Trust Diagnostic test for eqbe gene of streptococcus equi
WO2010046493A2 (en) 2008-10-23 2010-04-29 Université de Lausanne Gene transfer vectors comprising at least one isolated dna molecule having insulator and or boundary properties and methods to identify the same
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
WO2010054154A2 (en) 2008-11-07 2010-05-14 Danisco A/S Bifidobacteria crispr sequences
CN102984955B (zh) 2008-11-11 2015-01-07 营养健康有限公司 长双歧杆菌
GB2466177A (en) 2008-12-03 2010-06-16 Arab Science & Technology Found Bacteriophage selection and breeding
EP2367938B1 (en) 2008-12-12 2014-06-11 DuPont Nutrition Biosciences ApS Genetic cluster of strains of streptococcus thermophilus having unique rheological properties for dairy fermentation
KR20100093626A (ko) 2009-02-17 2010-08-26 서강대학교산학협력단 슈도모나스 애루지노사에 대한 파아지 치료
WO2010113037A1 (en) 2009-04-03 2010-10-07 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
WO2010129019A2 (en) 2009-04-27 2010-11-11 Pacific Biosciences Of California, Inc. Real-time sequencing methods and systems
WO2010144151A2 (en) 2009-06-12 2010-12-16 Pacific Biosciences Of California, Inc. Single-molecule real-time analysis of protein synthesis
WO2011017293A2 (en) 2009-08-03 2011-02-10 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
AU2010299857A1 (en) 2009-09-25 2012-04-19 Basf Plant Science Company Gmbh Plants having enhanced yield-related traits and a method for making the same
US9677125B2 (en) 2009-10-21 2017-06-13 General Electric Company Detection of plurality of targets in biological samples
US20110269119A1 (en) 2009-10-30 2011-11-03 Synthetic Genomics, Inc. Encoding text into nucleic acid sequences
WO2011097036A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2011101696A1 (en) * 2010-02-18 2011-08-25 Cellectis Improved meganuclease recombination system
US20120027786A1 (en) 2010-02-23 2012-02-02 Massachusetts Institute Of Technology Genetically programmable pathogen sense and destroy
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
MX379482B (es) 2010-03-12 2025-03-10 Brookhaven Science Associates/Brookhaven Nat Laboratory Enterobacter sp.638 y metodos de uso de la misma.
EA024121B9 (ru) 2010-05-10 2017-01-30 Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния Композиции эндорибонуклеаз и способы их использования
US20110201118A1 (en) 2010-06-14 2011-08-18 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
WO2012047726A1 (en) 2010-09-29 2012-04-12 The Broad Institute, Inc. Methods for chromatin immuno-precipitations
DK2630156T3 (en) 2010-10-20 2018-12-17 Dupont Nutrition Biosci Aps CRISPR-CAS SEQUENCES OF LACTOCOCCUS
EP2633040B1 (en) 2010-10-27 2019-07-10 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
WO2012093833A2 (en) 2011-01-03 2012-07-12 Toolgen Incorporation Genome engineering via designed tal effector nucleases
US20120214160A1 (en) 2011-01-14 2012-08-23 Life Technologies Corporation Methods, compositions, and kits for detecting rare cells
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
ES2767884T5 (es) 2011-07-04 2023-05-09 Dsm Ip Assets Bv Cultivo mixto anti-listeria y método para la producción de queso
US20140274812A1 (en) * 2011-07-15 2014-09-18 The General Hospital Corporation Methods of Transcription Activator Like Effector Assembly
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
DK2836226T3 (en) 2012-02-24 2017-09-18 Hutchinson Fred Cancer Res COMPOSITIONS AND PROCEDURES FOR TREATING HEMOGLOBINOPATHY
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
JP6352250B2 (ja) 2012-05-02 2018-07-04 ダウ アグロサイエンシィズ エルエルシー リンゴ酸デヒドロゲナーゼの標的改変
JP6559063B2 (ja) 2012-05-07 2019-08-14 サンガモ セラピューティクス， インコーポレイテッド 導入遺伝子のヌクレアーゼ媒介標的化組み込みのための方法および組成物
US11120889B2 (en) 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
LT3401400T (lt) * 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
US9102936B2 (en) 2012-06-11 2015-08-11 Agilent Technologies, Inc. Method of adaptor-dimer subtraction using a CRISPR CAS6 protein
EP2674501A1 (en) 2012-06-14 2013-12-18 Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail Method for detecting and identifying enterohemorrhagic Escherichia coli
US10025647B2 (en) * 2012-06-30 2018-07-17 Intel Corporation Memory poisoning with hints
EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
JP2015527889A (ja) 2012-07-25 2015-09-24 ザ ブロード インスティテュート， インコーポレイテッド 誘導可能なｄｎａ結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用
HK1217732A1 (zh) 2012-09-07 2017-01-20 美国陶氏益农公司 Fad3性能基因座及相應的能夠誘導靶向斷裂的靶位點特異性結合蛋白
AU2013329308B2 (en) 2012-10-09 2018-11-01 Liposcience, Inc. NMR quantification of branched chain amino acids
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
WO2014071235A1 (en) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
EP3135765A1 (en) 2012-12-06 2017-03-01 Sigma-Aldrich Co. LLC Crispr-based genome modification and regulation
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
IL239317B (en) * 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
JP2016504026A (ja) 2012-12-12 2016-02-12 ザ・ブロード・インスティテュート・インコーポレイテッド 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
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
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
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
AU2013359146B2 (en) 2012-12-13 2017-12-07 Corteva Agriscience Llc DNA detection methods for site specific nuclease activity
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
MX2015008107A (es) * 2012-12-19 2015-11-06 Dow Agrosciences Llc Transformacion de soja mejorada para una produccion aficaz de eventos transgenicos de alto rendimiento.
CA2897932A1 (en) 2013-01-14 2014-07-17 Recombinetics, Inc. Hornless livestock
CN103233028B (zh) 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
US20140212869A1 (en) 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
CA2900338C (en) 2013-02-07 2024-11-05 The General Hospital Corporation TRANSCRIPTIONAL ACTIVATORS TALE
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
EP2958996B1 (en) 2013-02-25 2019-10-16 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
JP2016507244A (ja) 2013-02-27 2016-03-10 ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト（ゲーエムベーハー）Ｈｅｌｍｈｏｌｔｚ Ｚｅｎｔｒｕｍ ＭｕｅｎｃｈｅｎＤｅｕｔｓｃｈｅｓ Ｆｏｒｓｃｈｕｎｇｓｚｅｎｔｒｕｍ ｆｕｅｒ Ｇｅｓｕｎｄｈｅｉｔ ｕｎｄ Ｕｍｗｅｌｔ （ＧｍｂＨ） Ｃａｓ９ヌクレアーゼによる卵母細胞における遺伝子編集
BR112015021788B1 (pt) 2013-03-08 2023-02-28 Oxford Nanopore Technologies Plc Métodos para mover uma ou mais helicases imobilizadas, para controlar o movimento de um polinucleotídeo alvo, para caracterizar um polinucleotídeo alvo e para controlar o carregamento de uma ou mais helicases em um polinucleotídeo alvo, uso de um poro de transmembrana e de um potencial aplicado e de um ou mais espaçadores, complexo, e, kit
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
SG11201507378UA (en) 2013-03-14 2015-10-29 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
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
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US10378027B2 (en) 2013-03-15 2019-08-13 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
RU2723130C2 (ru) 2013-04-05 2020-06-08 ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи Способы и композиции для встраивания экзогенной последовательности в геном растений
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
DK2986729T3 (en) 2013-04-16 2018-10-29 Regeneron Pharma TARGETED MODIFICATION OF ROOT THROUGH
CN103224947B (zh) 2013-04-28 2015-06-10 陕西师范大学 一种基因打靶系统
CA2910427C (en) 2013-05-10 2024-02-20 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
WO2014194190A1 (en) 2013-05-30 2014-12-04 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
JP6482546B2 (ja) 2013-07-19 2019-03-13 ラリクス・バイオサイエンス・リミテッド・ライアビリティ・カンパニーＬａｒｉｘ Ｂｉｏｓｃｉｅｎｃｅ， Ｌｌｃ 二重対立遺伝子ノックアウトを生成するための方法および組成物
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
CA2922428A1 (en) 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
WO2015033293A1 (en) 2013-09-04 2015-03-12 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
WO2015088643A1 (en) 2013-12-11 2015-06-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
BR112016013213A2 (pt) 2013-12-12 2017-12-05 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
KR20160102056A (ko) 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 ｒｎａ
EP3105327A4 (en) 2014-02-12 2017-10-18 Thomas Jefferson University Compositions and methods of using microrna inhibitors
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
JP6737714B2 (ja) 2014-03-14 2020-08-12 ユニヴァーシティ オブ ワシントン ゲノムインスレーターエレメントおよびその使用
EP3126498A4 (en) 2014-03-20 2017-08-23 Université Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
EP3126503A1 (en) * 2014-04-03 2017-02-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
MA41349A (fr) 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
SI3250691T1 (sl) 2015-01-28 2023-10-30 Caribou Biosciences, Inc. Hibridni dna/rna-polinukleotidi crispr in postopki za uporabo
ES2880473T5 (es) 2015-01-30 2024-05-09 Univ California Suministro de proteínas en células hematopoyéticas primarias
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
JP6817215B2 (ja) 2015-03-03 2021-01-20 ザ ジェネラル ホスピタル コーポレイション 変更ＰＡＭ特異性を有する遺伝子操作ＣＲＩＳＰＲ−Ｃａｓ９ヌクレアーゼ
US20180148711A1 (en) 2015-05-28 2018-05-31 Coda Biotherapeutics, Inc. Genome editing vectors
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
EP3525832A4 (en) 2016-10-14 2020-04-29 The General Hospital Corp. Epigenetically regulated site-specific nucleases
US11730828B2 (en) 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
CN110799205A (zh) 2017-04-21 2020-02-14 通用医疗公司 利用CRISPR-Cpf1的可诱导、可调和多重的人类基因调节
EP3793584A4 (en) 2018-05-17 2022-10-26 The General Hospital Corporation CCCTC BINDING FACTOR VARIATIONS
US20230036273A1 (en) 2019-11-27 2023-02-02 The General Hospital Corporation System and method for activating gene expression
WO2021243289A1 (en) 2020-05-29 2021-12-02 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)

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AU2014227653B2 (en) 2017-04-20
US10415059B2 (en) 2019-09-17
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US20170327805A1 (en) 2017-11-16
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IL289396A (en) 2022-02-01
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WO2014144761A2 (en) 2014-09-18
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US20210130850A1 (en) 2021-05-06
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