CA2879997A1 - Inducible dna binding proteins and genome perturbation tools and applications thereof - Google Patents

Inducible dna binding proteins and genome perturbation tools and applications thereof Download PDF

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Publication number
CA2879997A1
CA2879997A1 CA2879997A CA2879997A CA2879997A1 CA 2879997 A1 CA2879997 A1 CA 2879997A1 CA 2879997 A CA2879997 A CA 2879997A CA 2879997 A CA2879997 A CA 2879997A CA 2879997 A1 CA2879997 A1 CA 2879997A1
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Prior art keywords
domain
tale
histone
dna
crispr
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Abandoned
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CA2879997A
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English (en)
French (fr)
Inventor
Feng Zhang
Mark BRIGHAM
Le Cong
Silvana KONERMANN
Neville Espi Sanjana
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48914461&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA2879997(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Massachusetts Institute of Technology, Broad Institute Inc, Harvard University filed Critical Massachusetts Institute of Technology
Publication of CA2879997A1 publication Critical patent/CA2879997A1/en
Abandoned legal-status Critical Current

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    • 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Virology (AREA)
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  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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  • Veterinary Medicine (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2879997A 2012-07-25 2013-07-21 Inducible dna binding proteins and genome perturbation tools and applications thereof Abandoned CA2879997A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261675778P 2012-07-25 2012-07-25
US61/675,778 2012-07-25
US201261721283P 2012-11-01 2012-11-01
US61/721,283 2012-11-01
US201261736465P 2012-12-12 2012-12-12
US61/736,465 2012-12-12
US201361794458P 2013-03-15 2013-03-15
US61/794,458 2013-03-15
US201361835973P 2013-06-17 2013-06-17
US61/835,973 2013-06-17
PCT/US2013/051418 WO2014018423A2 (en) 2012-07-25 2013-07-21 Inducible dna binding proteins and genome perturbation tools and applications thereof

Publications (1)

Publication Number Publication Date
CA2879997A1 true CA2879997A1 (en) 2014-01-30

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ID=48914461

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CA2879997A Abandoned CA2879997A1 (en) 2012-07-25 2013-07-21 Inducible dna binding proteins and genome perturbation tools and applications thereof

Country Status (12)

Country Link
US (4) US20150291966A1 (https=)
EP (3) EP3494997B1 (https=)
JP (2) JP2015527889A (https=)
KR (2) KR102530118B1 (https=)
CN (2) CN105188767A (https=)
AU (3) AU2013293270B2 (https=)
CA (1) CA2879997A1 (https=)
DK (1) DK3494997T3 (https=)
ES (1) ES2757623T3 (https=)
PL (1) PL3494997T3 (https=)
PT (1) PT3494997T (https=)
WO (1) WO2014018423A2 (https=)

Cited By (7)

* Cited by examiner, † Cited by third party
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CN110373459A (zh) * 2019-06-27 2019-10-25 郑湘榕 基于STIP1基因rs2236647位点用于儿童哮喘病检测的试剂、试剂盒及应用
CN111500569A (zh) * 2014-03-05 2020-08-07 国立大学法人神户大学 特异性转变靶向dna序列的核酸碱基的基因组序列的修饰方法、及其使用的分子复合体
CN111849991A (zh) * 2020-08-05 2020-10-30 武汉纽福斯生物科技有限公司 一种寡核苷酸及其应用
CN113178263A (zh) * 2021-04-30 2021-07-27 上海市公共卫生临床中心 肺结核病变活动性标志物、试剂盒、方法及模型构建方法
CN114814217A (zh) * 2021-01-19 2022-07-29 首都医科大学附属北京世纪坛医院 磷脂酰肌醇聚糖特异性磷脂酶d及其多肽片段在淋巴水肿中的应用
CN115952468A (zh) * 2022-12-20 2023-04-11 中移信息技术有限公司 特征处理方法、装置、设备及计算机存储介质
CN119818681A (zh) * 2025-01-07 2025-04-15 重庆医科大学附属第二医院 Pax 9激动剂在制备预防或治疗头颈部鳞状细胞癌中的应用

Families Citing this family (550)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2013010911A (es) 2011-03-23 2015-03-03 Pioneer Hi Bred Int Metodos para producir un locus de rasgo transgenico complejo.
WO2012158945A2 (en) * 2011-05-19 2012-11-22 The Scripps Research Institute Compositions and methods for treating charcot-marie-tooth diseases and related neuronal diseases
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
US10465042B2 (en) 2011-12-02 2019-11-05 Yale University Poly(amine-co-ester) nanoparticles and methods of use thereof
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
IN2014DN09261A (https=) 2012-04-25 2015-07-10 Regeneron Pharma
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
KR102530118B1 (ko) 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
EP4357457B1 (en) * 2012-10-23 2024-10-16 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
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
AU2014214719B2 (en) * 2013-02-07 2020-02-13 The General Hospital Corporation Tale transcriptional activators
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
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
AU2014227653B2 (en) 2013-03-15 2017-04-20 The General Hospital Corporation Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
WO2014186435A2 (en) 2013-05-14 2014-11-20 University Of Georgia Research Foundation, Inc. Compositions and methods for reducing neointima formation
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
KR20250012194A (ko) * 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
US10421957B2 (en) 2013-07-29 2019-09-24 Agilent Technologies, Inc. DNA assembly using an RNA-programmable nickase
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CA3109801C (en) 2013-08-22 2024-01-09 Andrew Cigan Plant genome modification using guide rna/cas endonuclease systems and methods of use
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
IL312865B2 (en) 2013-09-11 2025-06-01 Eagle Biologics Inc Liquid protein formulations containing viscosity-lowering agents
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
MX2016007325A (es) 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
BR112016013207A2 (pt) 2013-12-12 2017-09-26 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para o hbv e distúrbios e doenças virais
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
EP3470089A1 (en) 2013-12-12 2019-04-17 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
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US10354746B2 (en) 2014-01-27 2019-07-16 Georgia Tech Research Corporation Methods and systems for identifying CRISPR/Cas off-target sites
US10287590B2 (en) 2014-02-12 2019-05-14 Dna2.0, Inc. Methods for generating libraries with co-varying regions of polynuleotides for genome modification
WO2015122967A1 (en) 2014-02-13 2015-08-20 Clontech Laboratories, Inc. Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same
US10907219B2 (en) * 2014-02-18 2021-02-02 Unm Rainforest Innovations Compositions and methods for controlling cellular function
US11186843B2 (en) 2014-02-27 2021-11-30 Monsanto Technology Llc Compositions and methods for site directed genomic modification
EP3110971B1 (en) 2014-02-27 2019-05-08 The Broad Institute Inc. T cell balance gene expression and methods of use thereof
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2015138510A1 (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
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
EP3126503A1 (en) * 2014-04-03 2017-02-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
EP3556858A3 (en) * 2014-04-09 2020-01-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating cystic fibrosis
CN103952424B (zh) 2014-04-23 2017-01-11 尹熙俊 生产mstn双侧基因敲除的双肌性状体细胞克隆猪的方法
CN104630267B (zh) * 2014-07-17 2017-10-31 清华大学 利用tale转录抑制子在哺乳动物细胞中模块化构建合成基因线路的试剂盒
WO2015172149A1 (en) 2014-05-09 2015-11-12 Yale University Hyperbranched polyglycerol-coated particles and methods of making and using thereof
US11918695B2 (en) 2014-05-09 2024-03-05 Yale University Topical formulation of hyperbranched polymer-coated particles
EP3152319A4 (en) * 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
SG11201610357UA (en) 2014-06-13 2017-01-27 Illumina Cambridge Ltd Methods and compositions for preparing sequencing libraries
WO2016003814A1 (en) 2014-06-30 2016-01-07 Illumina, Inc. Methods and compositions using one-sided transposition
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
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
ES3047792T3 (en) * 2014-07-14 2025-12-04 Univ California Crispr/cas transcriptional modulation
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016025759A1 (en) 2014-08-14 2016-02-18 Shen Yuelei Dna knock-in system
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
ES2730378T3 (es) 2014-08-27 2019-11-11 Caribou Biosciences Inc Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9
CN108064129A (zh) 2014-09-12 2018-05-22 纳幕尔杜邦公司 玉米和大豆中复合性状基因座的位点特异性整合位点的产生和使用方法
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
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
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CN113016623A (zh) 2014-09-26 2021-06-25 谱赛科美国股份有限公司 用于甜菊的单核苷酸多态性(snp)标志物
EP3200804A4 (en) 2014-10-01 2018-04-18 Eagle Biologics, Inc. Polysaccharide and nucleic acid formulations containing viscosity-lowering agents
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016069591A2 (en) 2014-10-27 2016-05-06 The Broad Institute Inc. Compositions, methods and use of synthetic lethal screening
CA2963820A1 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
EP3224381B1 (en) 2014-11-25 2019-09-04 The Brigham and Women's Hospital, Inc. Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease
CA2969145A1 (en) 2014-11-26 2016-06-02 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
AU2015360502A1 (en) 2014-12-10 2017-06-29 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
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)
US20190054117A1 (en) * 2014-12-19 2019-02-21 Novartis Ag Dimerization switches and uses thereof
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
US20170369855A1 (en) * 2014-12-24 2017-12-28 Dana-Farber Cancer Institute, Inc. Systems and methods for genome modification and regulation
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
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
EP3250691B9 (en) 2015-01-28 2023-08-02 Caribou Biosciences, Inc. Crispr hybrid dna/rna polynucleotides and methods of use
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
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
AR103926A1 (es) 2015-03-16 2017-06-14 Inst Genetics & Dev Biolog Cas Método para realizar modificaciones sitio dirigidas en el genoma de una planta usando materiales no heredables
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
JP6892642B2 (ja) * 2015-04-13 2021-06-23 国立大学法人 東京大学 光依存的に又は薬物存在下でヌクレアーゼ活性若しくはニッカーゼ活性を示す、又は標的遺伝子の発現を抑制若しくは活性化するポリペプチドのセット
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
CN107667173A (zh) 2015-05-06 2018-02-06 斯尼普技术有限公司 改变微生物种群和改善微生物群
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
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
EP3095870A1 (en) 2015-05-19 2016-11-23 Kws Saat Se Methods for the in planta transformation of plants and manufacturing processes and products based and obtainable therefrom
WO2016191684A1 (en) * 2015-05-28 2016-12-01 Finer Mitchell H Genome editing vectors
EA037359B1 (ru) 2015-06-01 2021-03-17 Тэмпл Юниверсити - Оф Зе Коммонвэлс Систем Оф Хайе Эдьюкейшн Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич
EP3307887A1 (en) 2015-06-09 2018-04-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
EP4159856A1 (en) 2015-06-18 2023-04-05 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
US10954513B2 (en) * 2015-06-18 2021-03-23 University Of Utah Research Foundation RNA-guided transcriptional regulation and methods of using the same for the treatment of back pain
EP4545544A3 (en) 2015-06-29 2025-10-08 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
EP4512819A3 (en) 2015-08-12 2025-08-06 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
CA3033909A1 (en) 2015-08-14 2017-02-23 The University Of Sydney Connexin 45 inhibition for therapy
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
EP3341727B1 (en) * 2015-08-25 2022-08-10 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
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
US11693017B2 (en) 2015-09-18 2023-07-04 President And Fellows Of Harvard College Small molecule biosensors
WO2017053431A2 (en) * 2015-09-21 2017-03-30 Arcturus Therapeutics, Inc. Allele selective gene editing and uses thereof
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
CN105177038B (zh) * 2015-09-29 2018-08-24 中国科学院遗传与发育生物学研究所 一种高效定点编辑植物基因组的CRISPR/Cas9系统
JP7097070B2 (ja) 2015-10-01 2022-07-07 ゴレイニ インコーポレイテッド クロライドチャネルの標的発現及びその使用方法
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US10947559B2 (en) 2015-10-16 2021-03-16 Astrazeneca Ab Inducible modification of a cell genome
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
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したＣａｓ９蛋白質
EP3368687B1 (en) 2015-10-27 2021-09-29 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
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
US11092607B2 (en) * 2015-10-28 2021-08-17 The Board Institute, Inc. Multiplex analysis of single cell constituents
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
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
SG11201803151PA (en) 2015-11-05 2018-05-30 Agency Science Tech & Res Chemical-inducible genome engineering technology
AU2016355178B9 (en) 2015-11-19 2019-05-30 Massachusetts Institute Of Technology Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
AU2016382512A1 (en) 2015-12-30 2018-07-12 Novartis Ag Immune effector cell therapies with enhanced efficacy
ES2875747T3 (es) * 2016-01-11 2021-11-11 Univ Leland Stanford Junior Proteínas quiméricas y métodos de inmunoterapia
WO2017123559A2 (en) * 2016-01-11 2017-07-20 The Board Of Trustees Of The Leland Stanford Junior University Chimeric proteins and methods of regulating gene expression
CA3014795A1 (en) 2016-02-16 2017-08-24 Yale University Compositions and methods for treatment of cystic fibrosis
US11136597B2 (en) 2016-02-16 2021-10-05 Yale University Compositions for enhancing targeted gene editing and methods of use thereof
WO2017147196A1 (en) 2016-02-22 2017-08-31 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
US11530253B2 (en) 2016-02-25 2022-12-20 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by CRISPR/CAS9 technology
CA3016571A1 (en) 2016-03-04 2017-09-08 Indoor Biotechnologies Inc. Fel d 1 knockouts and associated compositions and methods based on crispr-cas9 genomic editing
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
CN116059245A (zh) 2016-03-23 2023-05-05 加利福尼亚大学董事会 治疗线粒体障碍的方法
EP3433363A1 (en) 2016-03-25 2019-01-30 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
WO2017173453A1 (en) 2016-04-01 2017-10-05 The Brigham And Women's Hospital, Inc. Stimuli-responsive nanoparticles for biomedical applications
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
CN117925730A (zh) * 2016-04-21 2024-04-26 国立大学法人神户大学 提高基因组序列修饰技术中的突变导入效率的方法、及其使用的分子复合体
WO2017189870A1 (en) 2016-04-27 2017-11-02 Massachusetts Institute Of Technology Stable nanoscale nucleic acid assemblies and methods thereof
US11514331B2 (en) 2016-04-27 2022-11-29 Massachusetts Institute Of Technology Sequence-controlled polymer random access memory storage
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US10568840B2 (en) 2016-05-06 2020-02-25 The Brigham And Women's Hospital, Inc. Self assembled gels for controlled delivery of encapsulated agents to cartilage
WO2017197128A1 (en) 2016-05-11 2017-11-16 Yale University Poly(amine-co-ester) nanoparticles and methods of use thereof
IL323024A (en) 2016-05-20 2025-10-01 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide RNAs
GB201609811D0 (en) 2016-06-05 2016-07-20 Snipr Technologies Ltd Methods, cells, systems, arrays, RNA and kits
CA3028158A1 (en) 2016-06-17 2017-12-21 The Broad Institute, Inc. Type vi crispr orthologs and systems
US11293021B1 (en) 2016-06-23 2022-04-05 Inscripta, Inc. Automated cell processing methods, modules, instruments, and systems
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
BR112019000057A2 (pt) * 2016-07-05 2019-04-02 The Johns Hopkins University composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina
AU2017292173B2 (en) 2016-07-06 2022-01-13 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
JP7305534B2 (ja) 2016-07-06 2023-07-10 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 疼痛関連障害を処置するための物質及び方法
EP3484870B1 (en) 2016-07-13 2022-11-16 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2018020323A2 (en) * 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
JP7200104B2 (ja) * 2016-08-01 2023-01-06 ヴァイロジン バイオテック カナダ リミテッド 免疫系刺激分子を発現する腫瘍溶解性単純ヘルペスウイルスベクター
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR102547316B1 (ko) 2016-08-03 2023-06-23 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
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
WO2018035388A1 (en) 2016-08-17 2018-02-22 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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
JP7179354B2 (ja) 2016-08-29 2022-11-29 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 皮膚処置のためのイオン種に基づく局所処方物
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
CN110536966A (zh) * 2016-09-08 2019-12-03 能源环境和技术研究中心O.A.,M.P. 用于范可尼贫血患者的基因疗法
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
BR112019006652A2 (pt) 2016-10-13 2019-07-02 Juno Therapeutics Inc métodos e composições para imunoterapia envolvendo moduladores da via metabólica do triptofano
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
JP7181862B2 (ja) 2016-10-18 2022-12-01 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 腫瘍浸潤リンパ球および治療の方法
CA3041517A1 (en) 2016-10-19 2018-04-26 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
WO2018081138A1 (en) 2016-10-24 2018-05-03 Yale University Biodegradable contraceptive implants
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
WO2018144097A1 (en) 2016-11-04 2018-08-09 Akeagen Llc Genetically modified non-human animals and methods for producing heavy chain-only antibodies
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
US11261428B2 (en) 2018-03-15 2022-03-01 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
US20190359661A1 (en) * 2016-11-25 2019-11-28 Nanoscope Technologies, LLC Method and device for pain modulation by optical activation of neurons and other cells
US12404514B2 (en) 2016-12-09 2025-09-02 The Broad Institute, Inc. CRISPR-systems for modifying a trait of interest in a plant
WO2018112470A1 (en) 2016-12-16 2018-06-21 The Brigham And Women's Hospital, Inc. Co-delivery of nucleic acids for simultaneous suppression and expression of target genes
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018126087A1 (en) 2016-12-29 2018-07-05 Applied Stemcell, Inc. Gene editing method using virus
EP3565907B1 (en) 2017-01-06 2022-05-04 Editas Medicine, Inc. Methods of assessing nuclease cleavage
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
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
AU2018234825B2 (en) 2017-03-15 2020-12-17 Massachusetts Institute Of Technology Novel CAS13B orthologues CRISPR enzymes and systems
US20200248203A1 (en) 2017-03-17 2020-08-06 Rescue Hearing Inc Gene Therapy Constructs and Methods for Treatment of Hearing Loss
US12180546B2 (en) 2017-03-17 2024-12-31 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
US12391936B2 (en) 2017-03-17 2025-08-19 Rescue Hearing, Inc. Gene therapy systems and related methods for treatment of hearing loss
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US12161694B2 (en) 2017-03-24 2024-12-10 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
EP3606518A4 (en) 2017-04-01 2021-04-07 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTION AND MODULATION OF IMMUNOTHERAPY RESISTANCE GENE SIGNATURE IN CANCER
WO2018187493A1 (en) 2017-04-04 2018-10-11 Yale University Compositions and methods for in utero delivery
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
WO2018191558A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Modulation of epithelial cell differentiation, maintenance and/or function through t cell action, and markers and methods of use thereof
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
US12331319B2 (en) 2017-04-12 2025-06-17 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
US20210293783A1 (en) 2017-04-18 2021-09-23 The General Hospital Corporation Compositions for detecting secretion and methods of use
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
US11499151B2 (en) 2017-04-28 2022-11-15 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
JP7218303B2 (ja) * 2017-05-09 2023-02-06 ウルトラジェニックス ファーマシューティカル インコーポレイテッド トランスフェクション試薬を生成するための拡大可能な方法
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3622092A4 (en) 2017-05-11 2021-06-23 The Broad Institute, Inc. METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF
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
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3102054A1 (en) 2017-06-05 2018-12-13 Fred Hutchinson Cancer Research Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
WO2018227114A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
WO2018232114A1 (en) * 2017-06-14 2018-12-20 Wisconsin Alumni Research Foundation Modified guide rnas, crispr-ribonucleotprotein complexes and methods of use
WO2018232195A1 (en) 2017-06-14 2018-12-20 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
MX2019015505A (es) 2017-06-30 2020-07-28 Inscripta Inc Metodos, modulos, instrumentos y sistemas de procesamiento de celulas automatizados.
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019003193A1 (en) 2017-06-30 2019-01-03 Novartis Ag METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS
CN109207516B (zh) * 2017-07-07 2020-12-01 中国科学院动物研究所 一种药物诱导的基因转录激活方法
CN109206520A (zh) * 2017-07-07 2019-01-15 中国科学院动物研究所 用于基因组编辑的药物诱导型融合蛋白及其编码基因与应用
CN109207518B (zh) * 2017-07-07 2020-12-01 中国科学院动物研究所 用于基因转录激活的药物诱导型CRISPR/Cas9系统
WO2019010422A1 (en) 2017-07-07 2019-01-10 The Broad Institute, Inc. ANTIVIRAL THERAPY BASED ON THE CRISPR SYSTEM
CN109207517B (zh) * 2017-07-07 2020-12-01 中国科学院动物研究所 用于基因组编辑和转录调控的药物诱导型CRISPR/Cas9系统
KR102523217B1 (ko) 2017-07-11 2023-04-20 시그마-알드리치 컴퍼니., 엘엘씨 표적된 게놈 변형을 개선하기 위한 뉴클레오솜 상호작용 단백질 도메인 사용
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
EP3654993A4 (en) 2017-07-17 2021-08-25 The Broad Institute, Inc. CELL ATLAS OF HEALTHY HUMAN COLUMN AND HUMAN COLUMN WITH COLITIS ULCEROSA
US12239646B2 (en) 2017-07-19 2025-03-04 Dana-Farber Cancer Institute, Inc. Cancer diagnostic and treatment
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
BR112019028146A2 (pt) * 2017-07-31 2020-07-07 Sigma-Aldrich Co. Llc rna guia sintético para sistemas ativadores de crispr/cas
WO2019024081A1 (zh) 2017-08-04 2019-02-07 北京大学 特异性识别甲基化修饰dna碱基的tale rvd及其应用
JP7414281B2 (ja) * 2017-08-08 2024-01-16 クイーンズランド ユニバーシティ オブ テクノロジー 初期心不全の診断方法
US11624077B2 (en) 2017-08-08 2023-04-11 Peking University Gene knockout method
CN111278468A (zh) * 2017-08-10 2020-06-12 马萨诸塞大学 用于脂肪营养不良的自体细胞疗法的人脂肪组织祖细胞
US10738327B2 (en) 2017-08-28 2020-08-11 Inscripta, Inc. Electroporation cuvettes for automation
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN109495227B (zh) * 2017-09-11 2020-10-20 维沃移动通信有限公司 一种控制资源集的配置方法、网络设备及终端
TW201920661A (zh) 2017-09-18 2019-06-01 大陸商博雅輯因（北京）生物科技有限公司 一種基因編輯t細胞及其用途
WO2019058376A1 (en) * 2017-09-19 2019-03-28 The State Of Israel, Ministry Of Agriculture & Rural Development, Agricultural Research Organization (Aro) (Volcani Center) BIRDS WITH GENOME EDITION
JP2020535802A (ja) 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
EP3687621A4 (en) 2017-09-30 2021-08-11 Inscripta, Inc. Flow through electroporation instrumentation
EP3695408B1 (en) 2017-10-02 2026-01-21 The Broad Institute, Inc. Medical use of cdk4/6- and checkpoint-inhibitors in phased combination, in treatment of cancer
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US11953479B2 (en) 2017-10-06 2024-04-09 The Research Foundation For The State University Of New York Selective optical aqueous and non-aqueous detection of free sulfites
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
KR101870694B1 (ko) * 2017-10-18 2018-06-25 주식회사 무진메디 Cas9 단백질, SRD5A2 유전자의 발현을 억제하는 가이드 RNA 및 양이온성 폴리머의 복합체가 봉입된 나노 리포좀-마이크로버블 결합체 및 이를 함유하는 탈모 개선 또는 치료용 조성물
MX2020004063A (es) 2017-10-20 2020-10-05 Hutchinson Fred Cancer Res Sistemas y metodos para producir celulas b modificadas geneticamente para expresar anticuerpos seleccionados.
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
CN109722415B (zh) 2017-10-27 2021-01-26 博雅辑因（北京）生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019087113A1 (en) 2017-11-01 2019-05-09 Novartis Ag Synthetic rnas and methods of use
WO2019094928A1 (en) 2017-11-10 2019-05-16 Massachusetts Institute Of Technology Microbial production of pure single stranded nucleic acids
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
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
CN107858430B (zh) * 2017-11-20 2019-01-04 武汉迈特维尔生物科技有限公司 一种用于诊断预示Her-2过表达型乳腺癌骨转移的基因诊断试剂盒
EP3713644B1 (en) 2017-11-20 2024-08-07 University of Georgia Research Foundation, Inc. Compositions and methods for modulating hif-2a to improve muscle generation and repair
IL274926B2 (en) * 2017-12-01 2026-04-01 Encoded Therapeutics Inc Pyrazolopyrimidines with activity against respiratory syncytial virus
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
WO2019118921A1 (en) 2017-12-14 2019-06-20 Flodesign Sonics, Inc. Acoustic transducer drive and controller
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US11730769B2 (en) 2017-12-22 2023-08-22 Children's Medical Center Corporation Compositions and methods for Williams Syndrome (WS) therapy
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
JP7267289B2 (ja) 2018-01-12 2023-05-01 セルジーン コーポレイション セレブロン改変化合物のスクリーニング方法
CA3088788A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
JP7483612B2 (ja) 2018-01-17 2024-05-15 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 ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット
WO2019144061A1 (en) 2018-01-19 2019-07-25 Duke University Genome engineering with crispr-cas systems in eukaryotes
CN109507429A (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位点相互作用蛋白的方法
KR20200124702A (ko) 2018-02-23 2020-11-03 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 신규한 cas9 오르소로그
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2019183572A1 (en) * 2018-03-23 2019-09-26 Refuge Biotechnologies, Inc. Gene regulation via conditional nuclear localization of gene modulating polypeptides
US10760075B2 (en) 2018-04-30 2020-09-01 Snipr Biome Aps Treating and preventing microbial infections
WO2019190874A1 (en) 2018-03-29 2019-10-03 Inscripta, Inc. Automated control of cell growth rates for induction and transformation
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CN108410909B (zh) * 2018-04-12 2021-08-03 吉林大学 一种利用植物激素aba及小分子物质pyr调节细胞通路的方法
US10376889B1 (en) 2018-04-13 2019-08-13 Inscripta, Inc. Automated cell processing instruments comprising reagent cartridges
US12338486B2 (en) 2018-04-19 2025-06-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded DNA
DK3560330T3 (da) 2018-04-24 2022-07-11 Kws Saat Se & Co Kgaa Planter med forbedret fordøjelighed og markørhaplotyper
US10508273B2 (en) 2018-04-24 2019-12-17 Inscripta, Inc. Methods for identifying selective binding pairs
US10858761B2 (en) 2018-04-24 2020-12-08 Inscripta, Inc. Nucleic acid-guided editing of exogenous polynucleotides in heterologous cells
US10526598B2 (en) 2018-04-24 2020-01-07 Inscripta, Inc. Methods for identifying T-cell receptor antigens
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
WO2019210268A2 (en) 2018-04-27 2019-10-31 The Broad Institute, Inc. Sequencing-based proteomics
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
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US20210371932A1 (en) 2018-06-01 2021-12-02 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
CA3102163A1 (en) 2018-06-26 2020-01-02 Massachusetts Institute Of Technology Crispr effector system based amplification methods, systems, and diagnostics
SG11202011240YA (en) 2018-06-26 2020-12-30 Broad Inst Inc Crispr/cas and transposase based amplification compositions, systems and methods
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
AU2019292919A1 (en) 2018-06-30 2021-03-11 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
JP7543246B2 (ja) * 2018-07-13 2024-09-02 ロンザ リミテッド 内因性タンパク質のレベルを低下させることによって生物学的産物の産生を改善する方法
US12559721B2 (en) * 2018-07-16 2026-02-24 Agency For Science, Technology And Research Method for isolating a cardiomyocyte population
CN113348245A (zh) 2018-07-31 2021-09-03 博德研究所 新型crispr酶和系统
US11939593B2 (en) 2018-08-01 2024-03-26 University Of Georgia Research Foundation, Inc. Compositions and methods for improving embryo development
CN113286884A (zh) 2018-08-07 2021-08-20 博德研究所 新型cas12b酶和系统
US10532324B1 (en) 2018-08-14 2020-01-14 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US11142740B2 (en) 2018-08-14 2021-10-12 Inscripta, Inc. Detection of nuclease edited sequences in automated modules and instruments
US10752874B2 (en) 2018-08-14 2020-08-25 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
CN110857440B (zh) * 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
US11965154B2 (en) 2018-08-30 2024-04-23 Inscripta, Inc. Detection of nuclease edited sequences in automated modules and instruments
JP7570107B2 (ja) 2018-08-31 2024-10-21 イェール ユニバーシティー ドナーオリゴヌクレオチドベースの遺伝子編集を亢進するための組成物および方法
WO2020045651A1 (ja) * 2018-08-31 2020-03-05 国立研究開発法人科学技術振興機構 光活性化可能なＴｅｔ発現制御システム
SG11202101984PA (en) 2018-08-31 2021-03-30 Univ Yale Compositions and methods for enhancing triplex and nuclease-based gene editing
US20210317479A1 (en) 2018-09-06 2021-10-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP3847251A1 (en) 2018-09-07 2021-07-14 Astrazeneca AB Compositions and methods for improved nucleases
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
JP7344300B2 (ja) 2018-09-18 2023-09-13 ブイエヌブイ ニューコ インク． Ａｒｃベースのカプシドおよびその使用
CN109408766B (zh) * 2018-09-26 2023-05-16 国网山西省电力公司电力科学研究院 一种梯形图频率计算的方法
CN109557315B (zh) * 2018-09-28 2020-04-17 山东大学 一种光控微管示踪剂及其应用
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
AU2019358519A1 (en) * 2018-10-11 2021-05-27 Fondazione Telethon Ets Selection by means of artificial transactivators
US20220411783A1 (en) 2018-10-12 2022-12-29 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US11851663B2 (en) 2018-10-14 2023-12-26 Snipr Biome Aps Single-vector type I vectors
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
AU2019368215B2 (en) 2018-10-22 2023-05-18 Inscripta, Inc. Engineered enzymes
US11214781B2 (en) 2018-10-22 2022-01-04 Inscripta, Inc. Engineered enzyme
CN109406469B (zh) * 2018-10-24 2021-04-09 中国医科大学 基于蛋白结合诱导dna双链变构的检测色氨酸的方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020092057A1 (en) 2018-10-30 2020-05-07 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
JP7487939B2 (ja) * 2018-11-01 2024-05-21 国立大学法人 東京大学 ２分割されたＣｐｆ１タンパク質
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
WO2020097411A1 (en) * 2018-11-08 2020-05-14 Massachusetts Institute Of Technology Encryption and steganography of synthetic gene circuits
WO2020097370A2 (en) 2018-11-08 2020-05-14 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
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
WO2020112195A1 (en) 2018-11-30 2020-06-04 Yale University Compositions, technologies and methods of using plerixafor to enhance gene editing
WO2020117715A1 (en) 2018-12-03 2020-06-11 Board Of Regents, The University Of Texas System Oligo-benzamide analogs and their use in cancer treatment
US12365888B2 (en) 2018-12-14 2025-07-22 Pioneer Hi-Bred International, Inc. CRISPR-Cas systems for genome editing
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
EP3911369A4 (en) * 2019-01-18 2022-11-16 Orthobio Therapeutics, Inc. EDITING GENES TO IMPROVE JOINT FUNCTION
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3914708A1 (en) 2019-01-24 2021-12-01 Massachusetts Institute Of Technology Nucleic acid nanostructure platform for antigen presentation and vaccine formulations formed therefrom
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US12522857B2 (en) 2019-03-04 2026-01-13 King Abdullah University Of Science And Technology Compositions and methods of targeted nucleic acid enrichment by loop adapter protection and exonuclease digestion
IL286011B2 (en) 2019-03-05 2024-06-01 The State Of Israel Ministry Of Agriculture & Rural Development Agricultural Res Organization Aro Vo Genome-edited birds
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CN113631713A (zh) 2019-03-25 2021-11-09 因思科瑞普特公司 酵母中的同时多重基因组编辑
US11001831B2 (en) 2019-03-25 2021-05-11 Inscripta, Inc. Simultaneous multiplex genome editing in yeast
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
CN111778277B (zh) * 2019-04-04 2023-02-28 中国科学院动物研究所 克氏综合征动物模型和其应用
US12564637B2 (en) 2019-04-11 2026-03-03 The University Of Hong Kong Nucleic acid mazzocchio and methods of making and use thereof
WO2020214619A1 (en) * 2019-04-15 2020-10-22 Emendobio Inc. Crispr compositions and methods for promoting gene editing of gata2
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220003512A (ko) * 2019-04-18 2022-01-10 미터 헬스, 인코포레이티드 호흡성 부정맥을 치료하기 위한 방법 및 조성물
EA202192931A1 (ru) 2019-04-30 2022-02-22 Эдиджен Инк. Способ прогнозирования эффективности лечения гемоглобинопатии
US20220243287A1 (en) 2019-05-13 2022-08-04 KWS SAAT SE & Co. KGaA Drought tolerance in corn
CN114072514A (zh) * 2019-05-14 2022-02-18 杜克大学 用于治疗atp酶介导的疾病的组合物和方法
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
WO2020243651A1 (en) 2019-05-29 2020-12-03 Encoded Therapeutics, Inc. Compositions and methods for selective gene regulation
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
AU2020288623A1 (en) 2019-06-06 2022-01-06 Inscripta, Inc. Curing for recursive nucleic acid-guided cell editing
CN110244048A (zh) * 2019-06-19 2019-09-17 中国人民解放军总医院第八医学中心 Serping1蛋白作为标志物在开发诊断活动性结核病的试剂中的应用
US10907125B2 (en) 2019-06-20 2021-02-02 Inscripta, Inc. Flow through electroporation modules and instrumentation
US10920189B2 (en) 2019-06-21 2021-02-16 Inscripta, Inc. Genome-wide rationally-designed mutations leading to enhanced lysine production in E. coli
US10927385B2 (en) 2019-06-25 2021-02-23 Inscripta, Inc. Increased nucleic-acid guided cell editing in yeast
US11905532B2 (en) 2019-06-25 2024-02-20 Massachusetts Institute Of Technology Compositions and methods for molecular memory storage and retrieval
JP2022539248A (ja) 2019-07-02 2022-09-07 フレッド ハッチンソン キャンサー リサーチ センター 組換えａｄ３５ベクター及び関連遺伝子治療改善
WO2021011649A2 (en) * 2019-07-15 2021-01-21 National University Of Singapore Methods and compositions for gene specific demethylation and activation
KR102183208B1 (ko) * 2019-07-25 2020-11-25 한국과학기술연구원 Ngn2(neurogenin-2)를 이용하여 척수 손상 모델에서 반응성 성상세포를 신경세포로 리프로그래밍하는 방법
CN110551755A (zh) * 2019-07-26 2019-12-10 天津大学 一种光控蛋白降解系统及构建方法和光调控蛋白降解的方法
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
CN112646009B (zh) * 2019-08-27 2022-02-08 上海交通大学 灵芝免疫调节蛋白突变体及应用
WO2021041922A1 (en) 2019-08-30 2021-03-04 The Broad Institute, Inc. Crispr-associated mu transposase systems
AU2020336992A1 (en) 2019-08-30 2022-04-14 Yale University Compositions and methods for delivery of nucleic acids to cells
CN114269941A (zh) 2019-09-04 2022-04-01 博雅辑因（北京）生物科技有限公司 基于脱靶评估评价基因编辑治疗的方法
WO2021050974A1 (en) 2019-09-12 2021-03-18 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12264367B2 (en) 2019-09-19 2025-04-01 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
CN115175996A (zh) 2019-09-20 2022-10-11 博德研究所 新颖vi型crispr酶和系统
JP2022169813A (ja) * 2019-09-30 2022-11-10 国立研究開発法人産業技術総合研究所 Ｃａｓ９ヌクレアーゼのＤＮＡ切断活性を制御する方法
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
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
US20230357788A1 (en) 2019-10-17 2023-11-09 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
WO2021081135A2 (en) * 2019-10-21 2021-04-29 The Regents Of The University Of California Compositions and methods for editing of the cdkl5 gene
US11203762B2 (en) 2019-11-19 2021-12-21 Inscripta, Inc. Methods for increasing observed editing in bacteria
CA3163565A1 (en) * 2019-12-02 2021-06-10 Council Of Scientific & Industrial Research Method and kit for detection of polynucleotide
KR102776469B1 (ko) 2019-12-10 2025-03-05 인스크립타 인코포레이티드 신규 mad 뉴클레아제
US10704033B1 (en) 2019-12-13 2020-07-07 Inscripta, Inc. Nucleic acid-guided nucleases
EP4069851A4 (en) 2019-12-18 2023-11-22 Inscripta, Inc. Cascade/dcas3 complementation assays for in vivo detection of nucleic acid-guided nuclease edited cells
US20230059884A1 (en) 2019-12-30 2023-02-23 Edigene Biotechnology Inc. Universal car-t targeting t-cell lymphoma cell and preparation method therefor and use thereof
CN114867850A (zh) 2019-12-30 2022-08-05 博雅缉因（北京）生物科技有限公司 一种纯化ucart细胞的方法与应用
US11859172B2 (en) 2020-01-02 2024-01-02 The Trustees Of Columbia University In The City Of New York Programmable and portable CRISPR-Cas transcriptional activation in bacteria
US10689669B1 (en) 2020-01-11 2020-06-23 Inscripta, Inc. Automated multi-module cell processing methods, instruments, and systems
AU2021213705A1 (en) 2020-01-27 2022-06-16 Inscripta, Inc. Electroporation modules and instrumentation
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
JP2023526172A (ja) * 2020-04-13 2023-06-21 ネイカー， インスティテュート バスコ デ インベスティガシオン イ デサロージョ アグラーリオ， エス．アー． マイコバクテリウム種に感染した対象を診断するｉｎ ｖｉｔｒｏ法
CN111376422B (zh) * 2020-04-18 2021-08-27 山东宇能环境工程有限公司 一种保温毡的自动成型生产线
US20210332388A1 (en) 2020-04-24 2021-10-28 Inscripta, Inc. Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells
CN111534590A (zh) * 2020-04-30 2020-08-14 福建西陇生物技术有限公司 一种结直肠癌多基因甲基化联合检测试剂盒及其应用
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
US11787841B2 (en) 2020-05-19 2023-10-17 Inscripta, Inc. Rationally-designed mutations to the thrA gene for enhanced lysine production in E. coli
PE20230080A1 (es) 2020-05-29 2023-01-11 Kws Saat Se And Co Kgaa Induccion de haploides en plantas
CN116194121A (zh) * 2020-06-09 2023-05-30 吉尼松公司 用于治疗扩张型心肌病的cilp-1抑制剂
CN111850043A (zh) * 2020-06-28 2020-10-30 武汉纽福斯生物科技有限公司 Ecel1重组腺相关病毒载体及应用
FR3111917B1 (fr) * 2020-06-30 2025-04-11 Oreal Signature moléculaire d’un état alopécique commun, associée aux jonctions cellulaires
CN111778333B (zh) * 2020-07-03 2022-11-18 东莞市滨海湾中心医院 测定edar表达水平的试剂的应用和试剂盒
WO2022007803A1 (zh) 2020-07-06 2022-01-13 博雅辑因（北京）生物科技有限公司 一种改善的rna编辑方法
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
JP2023538303A (ja) 2020-08-13 2023-09-07 イェール ユニバーシティー 所望の表現型を有するｃａｒ ｔ細胞の操作および選択のための組成物および方法
US12591976B2 (en) * 2020-08-17 2026-03-31 Thrive Bioscience, Inc. Cell segmentation image processing methods
CA3193424A1 (en) 2020-08-31 2022-03-03 Yale University Compositions and methods for delivery of nucleic acids to cells
WO2022060749A1 (en) 2020-09-15 2022-03-24 Inscripta, Inc. Crispr editing to embed nucleic acid landing pads into genomes of live cells
CN112138146B (zh) * 2020-09-25 2022-08-09 安徽医科大学 Manf蛋白的应用
EP4225904A4 (en) 2020-10-09 2025-10-08 Univ Massachusetts TARGETING NRIP1 TO ALLEVIATE METABOLIC DISEASE
US11512297B2 (en) 2020-11-09 2022-11-29 Inscripta, Inc. Affinity tag for recombination protein recruitment
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)
CA3199037A1 (en) * 2020-11-20 2022-05-27 Rocky CHEUNG Inducible cell death systems
CN112618963A (zh) * 2020-12-12 2021-04-09 安徽省旌一农业旅游发展有限公司 一种用于光疗箱的新生儿保护装置
US11777863B2 (en) * 2020-12-21 2023-10-03 Landis+ Gyr Innovations Optimized route for time-critical traffic in mesh network
CN112516288B (zh) * 2020-12-22 2023-04-18 西藏阿那达生物医药科技有限责任公司 N-糖基化修饰之灵芝免疫调节蛋白的应用
AU2021415461A1 (en) 2021-01-04 2023-08-17 Inscripta, Inc. Mad nucleases
US20240376451A1 (en) 2021-01-07 2024-11-14 Inscripta, Inc. Mad nucleases
JP2024502630A (ja) 2021-01-12 2024-01-22 マーチ セラピューティクス， インコーポレイテッド コンテキスト依存性二本鎖ｄｎａ特異的デアミナーゼ及びその使用
US11884924B2 (en) 2021-02-16 2024-01-30 Inscripta, Inc. Dual strand nucleic acid-guided nickase editing
CN113444730A (zh) * 2021-03-17 2021-09-28 昆明市延安医院 一种原发性肝细胞klotho基因转导干细胞筛选构建方法
CN113181218A (zh) * 2021-03-22 2021-07-30 中国福利会国际和平妇幼保健院 人羊膜上皮细胞在制备修复子宫瘢痕细胞制剂中的应用
MX2023011875A (es) * 2021-04-12 2024-01-05 Univ California Tratamiento genico para miocardiopatía arritmogénica del ventriculo derecho.
CN113124052B (zh) * 2021-04-16 2022-08-23 中国航空发动机研究院 电磁轴承-转子系统不平衡振动控制方法及电子设备
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer
CN113416713A (zh) * 2021-05-11 2021-09-21 中国农业科学院哈尔滨兽医研究所（中国动物卫生与流行病学中心哈尔滨分中心） 一种重组腺病毒的构建及其应用
WO2022261115A1 (en) 2021-06-07 2022-12-15 Yale University Peptide nucleic acids for spatiotemporal control of crispr-cas binding
CN113519460B (zh) * 2021-06-30 2023-02-17 华南农业大学 一种诱导型子宫上皮特异性基因工程鼠的构建及应用
AR126622A1 (es) 2021-07-30 2023-10-25 Kws Saat Se & Co Kgaa Plantas con digestibilidad mejorada y haplotipos marcadores
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells
JP7125727B1 (ja) 2021-09-07 2022-08-25 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法
KR20240099180A (ko) 2021-09-20 2024-06-28 레비비코르 인코포레이션 이종이식을 위한 10개의 유전자 변형을 포함하는 다중전이유전자 돼지
WO2023052508A2 (en) 2021-09-30 2023-04-06 Astrazeneca Ab Use of inhibitors to increase efficiency of crispr/cas insertions
US20250241999A1 (en) 2021-10-15 2025-07-31 Georgia State University Research Foundation, Inc. Delivery of therapeutic recombinant uricase using nanoparticles
WO2023070043A1 (en) 2021-10-20 2023-04-27 Yale University Compositions and methods for targeted editing and evolution of repetitive genetic elements
EP4437092A1 (en) 2021-11-26 2024-10-02 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
WO2023192872A1 (en) 2022-03-28 2023-10-05 Massachusetts Institute Of Technology Rna scaffolded wireframe origami and methods thereof
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
CN114632156B (zh) * 2022-05-17 2022-08-16 中国人民解放军军事科学院军事医学研究院 Tim-3在预防、治疗或减轻疼痛中的应用
CN115060901A (zh) * 2022-06-21 2022-09-16 中国医学科学院基础医学研究所 苹果酸酶2在制备矽肺病或肺纤维化相关疾病诊断试剂或治疗药物中的应用
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
GB202209518D0 (en) 2022-06-29 2022-08-10 Snipr Biome Aps Treating & preventing E coli infections
CN115350176B (zh) * 2022-07-14 2023-10-13 深圳大学 一种胃癌肿瘤细胞和胃癌肿瘤干细胞治疗药物及其应用
JP2025525569A (ja) 2022-07-18 2025-08-05 レナゲード セラピューティクス マネージメント インコーポレイテッド 遺伝子編集成分、システム、及び使用方法
CN114990110B (zh) * 2022-07-19 2023-07-14 江西农业大学 一种野外蝴蝶监测无损伤取样方法
JP2025523965A (ja) 2022-07-22 2025-07-25 ザ・ジョンズ・ホプキンス・ユニバーシティー デンドリマーが可能にする標的化細胞内ｃｒｉｓｐｒ／ｃａｓシステム送達および遺伝子編集
CA3262690A1 (en) * 2022-07-25 2024-02-01 Institut National de la Santé et de la Recherche Médicale USE OF APELINE FOR THE TREATMENT OF LYMPHOEDEMA
CN115177730B (zh) * 2022-08-05 2024-02-27 华中科技大学同济医学院附属协和医院 Ptpn22及其表达抑制剂的新用途
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US20250386788A1 (en) 2022-08-26 2025-12-25 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
WO2024047587A1 (en) 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
WO2024064824A2 (en) 2022-09-21 2024-03-28 Yale University Compositions and methods for identification of membrane targets for enhancement of nk cell therapy
WO2024081736A2 (en) 2022-10-11 2024-04-18 Yale University Compositions and methods of using cell-penetrating antibodies
CN115747222B (zh) * 2022-10-26 2025-01-03 华中农业大学 prdm15基因在制备环境敏感型突变体鱼中的应用
CN115487301B (zh) * 2022-11-08 2023-07-07 四川大学华西医院 Il-13抑制剂在制备延缓或治疗视网膜色素变性的药物中的用途
AU2023406926A1 (en) 2022-12-01 2025-06-26 Yale University Stimuli-responsive traceless engineering platform for intracellular payload delivery
WO2024123789A1 (en) 2022-12-07 2024-06-13 Sanofi Predicting indel frequencies
CN116004825B (zh) * 2022-12-08 2024-12-31 上海交通大学医学院附属上海儿童医学中心 Reg3a在制备辅助诊断或治疗肝母细胞瘤的试剂或药物中的应用
CN115792220B (zh) * 2022-12-12 2025-06-27 武汉大学中南医院 血管生成素受体Tie-2用作烟雾病诊断生物标志物的应用
WO2024136952A1 (en) * 2022-12-20 2024-06-27 Florida Atlantic University Board Of Trustees Phosphagen system modulators and uses 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
EP4680626A1 (en) * 2023-03-13 2026-01-21 Biogen MA Inc. Engineered cells and cell lines for aav production and methods of making and using the same
CN121127605A (zh) 2023-03-29 2025-12-12 阿斯利康(瑞典)有限公司 使用抑制剂来提高CRISPR/Cas插入的效率
CN116519950B (zh) * 2023-05-10 2023-11-07 首都医科大学附属北京天坛医院 一种预测卒中后抑郁的生物标志物及其应用
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
WO2024253957A1 (en) 2023-06-07 2024-12-12 Northwestern University Targeting autoimmunity-associated t cells through regulation of transcription factors
TW202503061A (zh) 2023-06-16 2025-01-16 大陸商益杰立科（上海）生物科技有限公司 靶向ｂ型肝炎病毒基因的表觀編輯工具
CN116908454A (zh) * 2023-07-14 2023-10-20 复旦大学附属肿瘤医院 Gdp-m在预测hrd水平和增敏dna损伤性药物中的应用
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
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
CN117797249A (zh) * 2023-11-14 2024-04-02 苏州祥沁医疗科技有限公司 基于mRNA的抗衰老抗老年痴呆预防及治疗疫苗
CN117257958B (zh) * 2023-11-21 2024-02-09 四川大学华西医院 Trps1抑制剂的新用途及用于治疗和/或预防雄激素性秃发的药物
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025158400A1 (en) 2024-01-24 2025-07-31 Yale University Compositions and methods of natural killer cell hyperboosts for enhancement of nk cell therapy
WO2025196502A1 (en) 2024-03-20 2025-09-25 North Carolina Agricultural & Technical State University Choline kinase inhibitors as a therapeutic treatment for obesity
US20250360228A1 (en) 2024-04-24 2025-11-27 Sarepta Therapeutics, Inc. Treatment of genetic neurological conditions with genomic editing
WO2025224715A1 (en) 2024-04-26 2025-10-30 King Abdullah Univeristy Of Science And Technology Methods for improving precise genome modification and reducing unwanted mutations by crispr-cas editing
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025259610A1 (en) 2024-06-10 2025-12-18 Northwestern University Genetic modulators of kras protein expression and their uses
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome
WO2026006832A1 (en) 2024-06-28 2026-01-02 University Of Connecticut Gene modulation for treating cancer
WO2026047626A1 (en) 2024-08-30 2026-03-05 Inceptor Bio, Llc Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy
WO2026060398A2 (en) 2024-09-16 2026-03-19 Northwestern University B cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies
WO2026060399A2 (en) 2024-09-16 2026-03-19 Northwestern University T cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies
CN119185312B (zh) * 2024-10-11 2026-04-21 浙江大学 盐酸胺苯吖啶在制备防治纤维化疾病的药物中的应用

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AT395893B (de) 1990-01-10 1993-03-25 Mayreder Kraus & Co Ing Befestigungsvorrichtung zum verbinden von bauteilen
NL9100084A (nl) 1991-01-17 1992-08-17 Verstraeten Funderingstech Bv Veiligheidsbassin.
CA2223103A1 (en) 1995-06-06 1996-12-12 Isis Pharmaceuticals Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5944710A (en) 1996-06-24 1999-08-31 Genetronics, Inc. Electroporation-mediated intravascular delivery
EP0979101B1 (en) 1996-07-03 2010-10-27 Merial, Inc. Recombinant canine adenovirus 2 (cav2) containing exogenous dna
US5869326A (en) 1996-09-09 1999-02-09 Genetronics, Inc. Electroporation employing user-configured pulsing scheme
US5990091A (en) 1997-03-12 1999-11-23 Virogenetics Corporation Vectors having enhanced expression, and methods of making and uses thereof
GB9710049D0 (en) 1997-05-19 1997-07-09 Nycomed Imaging As Method
US6716823B1 (en) 1997-08-13 2004-04-06 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom, and uses thereof
EP1015035B1 (en) 1997-08-13 2009-01-28 The Uab Research Foundation Vaccination by topical application of genetic vectors
US6706693B1 (en) 1997-08-13 2004-03-16 The Uab Research Foundation Vaccination by topical application of genetic vectors
US6348450B1 (en) 1997-08-13 2002-02-19 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom and uses thereof
AU4004699A (en) 1998-05-15 1999-12-06 Quark Biotech, Inc. Mechanical stress induced genes, expression products therefrom, and uses thereof
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
WO2002048195A2 (en) 2000-10-23 2002-06-20 Engeneos, Inc. Engineered stimulus-responsive switches
ATE452972T1 (de) 2001-05-01 2010-01-15 Ca Nat Research Council Induzierbares expressionssystem in eukaryotischen zellen
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
KR101666228B1 (ko) * 2007-09-28 2016-10-13 인트렉손 코포레이션 생물치료학적 분자를 발현시키기 위한 치료학적 유전자-스위치 작제물 및 생물반응기, 및 이의 용도
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
KR102262704B1 (ko) * 2009-08-11 2021-06-09 상가모 테라퓨틱스, 인코포레이티드 표적화 변형에 대한 동형접합성 유기체
HUE041436T2 (hu) * 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
EP2534163B1 (en) * 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG185481A1 (en) 2010-05-10 2012-12-28 Univ California Endoribonuclease compositions and methods of use thereof
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
DK2714936T3 (en) * 2011-06-01 2019-03-25 Prec Biosciences Inc METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES
WO2013020078A1 (en) * 2011-08-04 2013-02-07 University Of Medicine And Dentistry Of New Jersey Sequence-specific mrna interferase and uses thereof
CN103874894B (zh) 2011-09-02 2017-03-08 开利公司 提供热回收的制冷系统和制冷方法
US8450107B1 (en) * 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
US20130137173A1 (en) 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
US9637739B2 (en) * 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
KR102530118B1 (ko) * 2012-07-25 2023-05-08 더 브로드 인스티튜트, 인코퍼레이티드 유도 ｄｎａ 결합 단백질 및 게놈 교란 도구 및 이의 적용
EP4357457B1 (en) 2012-10-23 2024-10-16 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
EP3363902B1 (en) * 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
WO2014093694A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CA2894684A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
CN105121648B (zh) * 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
DK2931898T3 (en) * 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2013359262C1 (en) * 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
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
AU2014361834B2 (en) * 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
MX2016007325A (es) * 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
EP3889260A1 (en) * 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
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
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
WO2020053940A1 (ja) 2018-09-10 2020-03-19 株式会社Ｎｔｔドコモ ユーザ端末
US11696305B2 (en) 2020-06-18 2023-07-04 Qualcomm Incorporated Scheduling uplink transmissions using relay devices

Cited By (7)

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Publication number Priority date Publication date Assignee Title
CN111500569A (zh) * 2014-03-05 2020-08-07 国立大学法人神户大学 特异性转变靶向dna序列的核酸碱基的基因组序列的修饰方法、及其使用的分子复合体
CN110373459A (zh) * 2019-06-27 2019-10-25 郑湘榕 基于STIP1基因rs2236647位点用于儿童哮喘病检测的试剂、试剂盒及应用
CN111849991A (zh) * 2020-08-05 2020-10-30 武汉纽福斯生物科技有限公司 一种寡核苷酸及其应用
CN114814217A (zh) * 2021-01-19 2022-07-29 首都医科大学附属北京世纪坛医院 磷脂酰肌醇聚糖特异性磷脂酶d及其多肽片段在淋巴水肿中的应用
CN113178263A (zh) * 2021-04-30 2021-07-27 上海市公共卫生临床中心 肺结核病变活动性标志物、试剂盒、方法及模型构建方法
CN115952468A (zh) * 2022-12-20 2023-04-11 中移信息技术有限公司 特征处理方法、装置、设备及计算机存储介质
CN119818681A (zh) * 2025-01-07 2025-04-15 重庆医科大学附属第二医院 Pax 9激动剂在制备预防或治疗头颈部鳞状细胞癌中的应用

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