CA2930877A1 - Crispr-cas system materials and methods - Google Patents

Crispr-cas system materials and methods Download PDF

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Publication number
CA2930877A1
CA2930877A1 CA2930877A CA2930877A CA2930877A1 CA 2930877 A1 CA2930877 A1 CA 2930877A1 CA 2930877 A CA2930877 A CA 2930877A CA 2930877 A CA2930877 A CA 2930877A CA 2930877 A1 CA2930877 A1 CA 2930877A1
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rna
dna
sequence
guide rna
cell
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CA2930877A
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French (fr)
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Emmanuelle CHARPENTIER
Krzysztof CHYLINSKI
Ines FONFARA
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CRISPR Therapeutics AG
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CRISPR Therapeutics AG
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    • C12N9/14Hydrolases (3)
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
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    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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CA2930877A 2013-11-18 2014-11-17 Crispr-cas system materials and methods Pending CA2930877A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361905835P 2013-11-18 2013-11-18
US61/905,835 2013-11-18
PCT/EP2014/074813 WO2015071474A2 (en) 2013-11-18 2014-11-17 Crispr-cas system materials and methods

Publications (1)

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CA2930877A1 true CA2930877A1 (en) 2015-05-21

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CA2930877A Pending CA2930877A1 (en) 2013-11-18 2014-11-17 Crispr-cas system materials and methods

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US (1) US20160298096A1 (enExample)
EP (3) EP3071695A2 (enExample)
JP (4) JP2016537028A (enExample)
AU (3) AU2014350051A1 (enExample)
CA (1) CA2930877A1 (enExample)
WO (1) WO2015071474A2 (enExample)

Families Citing this family (220)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9719068B2 (en) 2010-05-06 2017-08-01 Children's Hospital Medical Center Methods and systems for converting precursor cells into intestinal tissues through directed differentiation
BR112013024337A2 (pt) 2011-03-23 2017-09-26 Du Pont locus de traço transgênico complexo em uma planta, planta ou semente, método para produzir em uma planta um locus de traço transgênico complexo e construto de expressão
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
PL4289948T3 (pl) 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
PT2898075E (pt) * 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
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
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of 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
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20240172759A (ko) 2013-06-17 2024-12-10 더 브로드 인스티튜트, 인코퍼레이티드 간의 표적화 및 치료를 위한 CRISPR­Cas 시스템, 벡터 및 조성물의 전달 및 용도
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3597755A1 (en) 2013-06-17 2020-01-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
MX363842B (es) 2013-08-22 2019-04-05 Du Pont Métodos para producir modificaciones genéticas en un genoma vegetal sin incorporar un marcador transgénico seleccionable, y composiciones de éstas.
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン,インコーポレイテッド CRISPR関連方法および支配gRNAのある組成物
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089354A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3653229A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. 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
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
SG11201605046YA (en) 2013-12-20 2016-07-28 Hutchinson Fred Cancer Res Tagged chimeric effector molecules and receptors thereof
US10787654B2 (en) * 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
DE212015000061U1 (de) 2014-02-11 2017-09-03 The Regents Of The University Of Colorado, A Body Corporate CRISPR-ermöglichtes Multiplex Genom Engineering
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015148863A2 (en) * 2014-03-26 2015-10-01 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
SG10201801654RA (en) 2014-05-28 2018-04-27 Childrens Hospital Med Ct Methods and systems for converting precursor cells into gastric tissues through directed differentiation
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016021973A1 (ko) * 2014-08-06 2016-02-11 주식회사 툴젠 캄필로박터 제주니 crispr/cas 시스템 유래 rgen을 이용한 유전체 교정
AU2015308910B2 (en) 2014-08-27 2017-12-07 Caribou Biosciences, Inc. Methods for increasing Cas9-mediated engineering efficiency
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
CA2956487A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
US10612042B2 (en) 2014-10-24 2020-04-07 Avectas Limited Delivery across cell plasma membranes
US20170306306A1 (en) * 2014-10-24 2017-10-26 Life Technologies Corporation Compositions and Methods for Enhancing Homologous Recombination
CN113699116A (zh) 2014-12-10 2021-11-26 明尼苏达大学董事会 用于治疗疾病的遗传修饰的细胞、组织和器官
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
US10450576B2 (en) 2015-03-27 2019-10-22 E I Du Pont De Nemours And Company Soybean U6 small nuclear RNA gene promoters and their use in constitutive expression of small RNA genes in plants
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
WO2016176652A2 (en) 2015-04-29 2016-11-03 Fred Hutchinson Cancer Research Center Modified stem cells and uses thereof
PL3291679T3 (pl) 2015-05-06 2022-04-25 Snipr Technologies Limited Zmiana populacji drobnoustrojowych i modyfikowanie mikrobioty
AU2016270649B2 (en) 2015-05-29 2022-04-21 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
ES2960226T3 (es) 2015-06-15 2024-03-01 Univ North Carolina State Métodos y composiciones para la administración eficiente de ácidos nucleicos y antimicrobianos basados en ARN
MX392008B (es) * 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
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
US11414657B2 (en) 2015-06-29 2022-08-16 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
IL295616A (en) 2015-07-31 2022-10-01 Us Health Adapted cells and treatment methods
CA2994969A1 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Targeted protein degradation to attenuate adoptive t-cell therapy associated adverse inflammatory responses
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
WO2017070598A1 (en) 2015-10-23 2017-04-27 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
KR101906491B1 (ko) 2015-11-30 2018-12-05 기초과학연구원 F. novicida 유래 Cas9을 포함하는 유전체 교정용 조성물
RU2018117360A (ru) 2015-12-04 2020-01-09 Карибо Биосайенсиз, Инк. Сконструированные нуклеиновые кислоты, нацеленные на нуклеиновую кислоту
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
US11026969B2 (en) 2015-12-23 2021-06-08 Fred Hutchinson Cancer Research Center High affinity T cell receptors and uses thereof
EP3397760A2 (en) 2015-12-30 2018-11-07 Avectas Limited Vector-free delivery of gene editing proteins and compositions to cells and tissues
JP7012645B2 (ja) 2016-01-11 2022-01-28 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー キメラタンパク質および免疫治療の方法
MX2018008344A (es) 2016-01-11 2018-12-06 Univ Leland Stanford Junior Proteinas quimericas y metodos para regular la expresion genica.
US9896696B2 (en) 2016-02-15 2018-02-20 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
WO2017155408A1 (en) 2016-03-11 2017-09-14 Erasmus University Medical Center Rotterdam Improved crispr-cas9 genome editing tool
EP3699281A1 (en) * 2016-03-11 2020-08-26 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
WO2017155717A1 (en) * 2016-03-11 2017-09-14 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
WO2017155715A1 (en) * 2016-03-11 2017-09-14 Pioneer Hi-Bred International, Inc. Novel cas9 systems and methods of use
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
GB201609811D0 (en) 2016-06-05 2016-07-20 Snipr Technologies Ltd Methods, cells, systems, arrays, RNA and kits
US11779657B2 (en) 2016-06-10 2023-10-10 City Of Hope Compositions and methods for mitochondrial genome editing
US10337051B2 (en) 2016-06-16 2019-07-02 The Regents Of The University Of California Methods and compositions for detecting a target RNA
WO2017222773A1 (en) * 2016-06-20 2017-12-28 Pioneer Hi-Bred International, Inc. Novel cas systems and methods of use
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会(法人团体) 用于生成条形码化组合文库的方法
KR102319845B1 (ko) * 2016-07-13 2021-11-01 디에스엠 아이피 어셋츠 비.브이. 조류 숙주 세포에 대한 crispr-cas 시스템
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
WO2018051347A1 (en) 2016-09-14 2018-03-22 Yeda Research And Development Co. Ltd. Crisp-seq, an integrated method for massively parallel single cell rna-seq and crispr pooled screens
EP3516044B1 (en) 2016-09-23 2025-04-16 Fred Hutchinson Cancer Center Tcrs specific for minor histocompatibility (h) antigen ha-1 and uses thereof
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
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
WO2018064352A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3534976A4 (en) 2016-11-04 2020-09-16 Children's Hospital Medical Center PATHOLOGICAL MODELS OF HEPATIC ORGANOIDS AND ASSOCIATED METHODS OF MANUFACTURE AND USE
US11332713B2 (en) 2016-11-16 2022-05-17 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
US9816093B1 (en) 2016-12-06 2017-11-14 Caribou Biosciences, Inc. Engineered nucleic acid-targeting nucleic acids
EP3559204B1 (en) 2016-12-22 2022-05-04 Avectas Limited Vector-free intracellular delivery by reversible permeabilisation
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JP7142637B2 (ja) * 2016-12-29 2022-09-27 ヨハン ウォルフガング ゲーテ-ウニベルジテート フランクフルト アム マイン より高次のゲノム編集ライブラリーを生成する方法
US20190071673A1 (en) * 2017-01-18 2019-03-07 Thomas Malcolm CRISPRs WITH IMPROVED SPECIFICITY
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
EP3580212A4 (en) 2017-02-08 2021-03-17 Dana Farber Cancer Institute, Inc. REGULATION OF CHEMERIC ANTIGEN RECEPTORS
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
US11898179B2 (en) 2017-03-09 2024-02-13 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
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7037577B2 (ja) 2017-03-15 2022-03-16 フレッド ハッチンソン キャンサー リサーチ センター 高親和性mage-a1特異的tcr及びその使用
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018191673A1 (en) 2017-04-14 2018-10-18 Children's Hospital Medical Center Multi donor stem cell compositions and methods of making same
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US11692184B2 (en) 2017-05-16 2023-07-04 The Regents Of The University Of California Thermostable RNA-guided endonucleases and methods of use thereof
CA3064000A1 (en) 2017-05-24 2018-11-29 Effector Therapeutics, Inc. Methods and compositions for cellular immunotherapy
KR20250026894A (ko) 2017-06-21 2025-02-25 티모시 에이. 버트람 신장 질환의 치료를 위한 면역특권화 생체활성 신장 세포
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
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019030306A1 (en) 2017-08-08 2019-02-14 Depixus ISOLATION AND IN VITRO ENRICHMENT OF NUCLEIC ACIDS USING SITE-SPECIFIC NUCLEASES
US10316324B2 (en) 2017-08-09 2019-06-11 Benson Hill Biosystems, Inc. Compositions and methods for modifying genomes
JP2020533962A (ja) 2017-08-11 2020-11-26 フレッド ハッチンソン キャンサー リサーチ センター Braf特異的tcrおよびその使用
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3071661A1 (en) 2017-09-06 2019-03-14 Fred Hutchinson Cancer Research Center Strep-tag specific chimeric receptors and uses thereof
EP3678690A1 (en) 2017-09-06 2020-07-15 Fred Hutchinson Cancer Research Center Methods for improving adoptive cell therapy
WO2019055862A1 (en) 2017-09-14 2019-03-21 Fred Hutchinson Cancer Research Center HIGH AFFINITY T CELL RECEPTORS AND USES THEREOF
CA3111479A1 (en) 2017-09-26 2019-04-04 The Board Of Trustees Of The University Of Illinois Crispr/cas system and method for genome editing and modulating transcription
JP2020536529A (ja) 2017-10-10 2020-12-17 チルドレンズ ホスピタル メディカル センター 食道組織および/または臓器組成物およびそれを作製する方法
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US20210069241A1 (en) 2017-10-20 2021-03-11 Fred Hutchinson Cancer Research Center Compositions and methods of immunotherapy targeting tigit and/or cd112r or comprising cd226 overexpression
CN117487776A (zh) 2017-11-01 2024-02-02 加利福尼亚大学董事会 Casz组合物和使用方法
US11970719B2 (en) 2017-11-01 2024-04-30 The Regents Of The University Of California Class 2 CRISPR/Cas compositions and methods of use
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated CRISPR/CAS Systems For Treatment of DMD
AU2018364993B2 (en) * 2017-11-10 2022-10-06 University Of Massachusetts Targeted CRISPR delivery platforms
WO2019109047A1 (en) 2017-12-01 2019-06-06 Fred Hutchinson Cancer Research Center Binding proteins specific for 5t4 and uses thereof
US11578323B2 (en) * 2017-12-14 2023-02-14 Bayer Healthcare Llc RNA-programmable endonuclease systems and their use in genome editing and other applications
US11932858B2 (en) 2017-12-14 2024-03-19 Donald Danforth Plant Science Center Homologous recombination via transcriptional activation
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126626A1 (en) 2017-12-21 2019-06-27 Children's Hospital Medical Center Digitalized human organoids and methods of using same
WO2019140278A1 (en) 2018-01-11 2019-07-18 Fred Hutchinson Cancer Research Center Immunotherapy targeting core binding factor antigens
US20200362366A1 (en) 2018-01-12 2020-11-19 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
WO2019165168A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
WO2019165116A1 (en) 2018-02-26 2019-08-29 Fred Hutchinson Cancer Research Center Compositions and methods for cellular immunotherapy
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
AU2019236204B2 (en) 2018-03-15 2026-02-26 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
JP2021518160A (ja) 2018-03-15 2021-08-02 ケーエスキュー セラピューティクス, インコーポレイテッド 免疫療法の改善のための遺伝子調節組成物及び遺伝子調節方法
US10760075B2 (en) 2018-04-30 2020-09-01 Snipr Biome Aps Treating and preventing microbial infections
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
CA3100050A1 (en) 2018-05-11 2019-11-14 Lupagen, Inc. Systems and methods for closed loop, real-time modifications of patient cells
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12398360B2 (en) 2018-06-01 2025-08-26 Avectas Limited Cell engineering platform
EP4512900A3 (en) * 2018-06-05 2025-04-02 LifeEDIT Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020018964A1 (en) 2018-07-20 2020-01-23 Fred Hutchinson Cancer Research Center Compositions and methods for controlled expression of antigen-specific receptors
EP3826652B1 (en) 2018-07-26 2026-02-25 Children's Hospital Medical Center Hepato-biliary-pancreatic tissues and methods of making same
WO2020028729A1 (en) 2018-08-01 2020-02-06 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
MX2021001938A (es) 2018-08-22 2021-04-19 Fred Hutchinson Cancer Center Inmunoterapia dirigida a antigenos kras o her2.
EP3844266A1 (en) 2018-08-28 2021-07-07 Fred Hutchinson Cancer Research Center Methods and compositions for adoptive t cell therapy incorporating induced notch signaling
EP3849568A4 (en) 2018-09-12 2022-06-15 Children's Hospital Medical Center Organoid compositions for the production of hematopoietic stem cells and derivatives thereof
WO2020068702A1 (en) 2018-09-24 2020-04-02 Fred Hutchinson Cancer Research Center Chimeric receptor proteins and uses thereof
WO2020072248A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system
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
US12264313B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for genome modification and alteration of expression
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
US11851663B2 (en) 2018-10-14 2023-12-26 Snipr Biome Aps Single-vector type I vectors
BR112021007229A2 (pt) 2018-10-16 2021-08-10 Blueallele, Llc métodos para inserção dirigida de dna em genes
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3877404A2 (en) 2018-11-09 2021-09-15 Fred Hutchinson Cancer Research Center T cell receptors specific for mesothelin and their use in immunotherapy
US12305218B2 (en) 2018-11-16 2025-05-20 Depixus Optimization of in vitro isolation of nucleic acids using site-specific nucleases
RU2712497C1 (ru) * 2018-11-26 2020-01-29 Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий Средство разрезания ДНК на основе Cas9 белка из биотехнологически значимой бактерии Clostridium cellulolyticum
CA3123151A1 (en) 2018-12-12 2020-06-18 Depixus Method of nucleic acid enrichment using site-specific nucleases followed by capture
US12365888B2 (en) 2018-12-14 2025-07-22 Pioneer Hi-Bred International, Inc. CRISPR-Cas systems for genome editing
IL284050B2 (en) 2018-12-20 2026-01-01 Ksq Therapeutics Inc Modified pyrazolopyrimidines and modified purines and their use as 1 uso inhibitors
WO2020142754A2 (en) 2019-01-04 2020-07-09 Mammoth Biosciences, Inc. Programmable nuclease improvements and compositions and methods for nucleic acid amplification and detection
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN114026116A (zh) 2019-02-20 2022-02-08 弗雷德哈钦森癌症研究中心 Ras新抗原特异性结合蛋白及其用途
BR112021017703A8 (pt) 2019-03-11 2023-04-18 Hutchinson Fred Cancer Res Receptores de células t wt1 de alta avidez e usos dos mesmos
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3966327A1 (en) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
EP3976066A4 (en) 2019-05-31 2023-06-28 Children's Hospital Medical Center Methods of generating and expanding hematopoietic stem cells
AU2020283048A1 (en) 2019-05-31 2021-12-23 Children's Hospital Medical Center Shaped organoid compositions and methods of making same
JP7640527B2 (ja) * 2019-08-12 2025-03-05 ライフエディット セラピューティクス,インコーポレイティド Rna誘導ヌクレアーゼ及びその活性断片及び変異体ならびに使用方法
US20220409661A1 (en) 2019-08-20 2022-12-29 Fred Hutchinson Cancer Research Center T-cell immunotherapy specific for wt-1
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP7452884B2 (ja) * 2019-10-23 2024-03-19 国立研究開発法人農業・食品産業技術総合研究機構 Dnaが編集された植物細胞を製造する方法、及びそれに用いるためのキット
US11060141B1 (en) 2019-12-23 2021-07-13 Stilla Technologies Multiplex drop-off digital polymerase chain reaction methods
WO2021191678A1 (en) 2020-03-23 2021-09-30 Avectas Limited Engineering of dendritic cells for generation of vaccines against sars-cov-2
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
JP2023542528A (ja) 2020-09-24 2023-10-10 フレッド ハッチンソン キャンサー センター Sox2抗原を標的とする免疫療法
WO2022066973A1 (en) 2020-09-24 2022-03-31 Fred Hutchinson Cancer Research Center Immunotherapy targeting pbk or oip5 antigens
TW202222841A (zh) 2020-10-06 2022-06-16 福瑞德哈金森腫瘤研究中心 用於治療表現mage-a1之疾病的組成物及方法
KR20230098186A (ko) 2020-10-30 2023-07-03 케이에스큐 세러퓨틱스 인코포레이티드 치환된 피라졸로피리미딘의 고체 상태 형태 및 이의 용도
US20250032540A1 (en) 2020-12-14 2025-01-30 Fred Hutchinson Cancer Center Compositions and methods for cellular immunotherapy
EP4263578A1 (en) 2020-12-18 2023-10-25 Basf Se Bioconversion of ferulic acid to vanillin
KR20230121065A (ko) 2020-12-18 2023-08-17 바스프 에스이 바닐린 생산을 위한 바닐산 형성이 억제된 아미콜라톱시스 균주
US20240110144A1 (en) 2021-02-01 2024-04-04 Avectas Limited Delivery platform
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
TW202317602A (zh) 2021-07-15 2023-05-01 福瑞德哈金森腫瘤中心 嵌合多肽
WO2023215725A1 (en) 2022-05-02 2023-11-09 Fred Hutchinson Cancer Center Compositions and methods for cellular immunotherapy
CN114934031B (zh) * 2022-05-25 2023-08-01 广州瑞风生物科技有限公司 新型Cas效应蛋白、基因编辑系统及用途
GB202209518D0 (en) 2022-06-29 2022-08-10 Snipr Biome Aps Treating & preventing E coli infections
CN121013904A (zh) 2023-03-30 2025-11-25 儿童医院医学中心 临床级类器官
CN118792281B (zh) * 2023-04-11 2025-11-25 清华大学 一种小型CRISPR-Cas系统及其应用
EP4713453A1 (en) 2023-05-15 2026-03-25 nChroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
EP4713433A2 (en) 2023-05-18 2026-03-25 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
WO2024263961A2 (en) 2023-06-23 2024-12-26 Children's Hospital Medical Center Methods of matrix-free suspension culture
WO2025072803A1 (en) 2023-09-29 2025-04-03 Children's Hospital Medical Center Ntrk2 signaling-mediated alveolar capillary injury and repair
WO2025078978A1 (en) 2023-10-09 2025-04-17 Avectas Limited Transfection of cells via reversible permeabilization
WO2025081123A1 (en) 2023-10-12 2025-04-17 Fred Hutchinson Cancer Center Methods and compositions for improving t cell immunotherapy
WO2025101820A1 (en) 2023-11-08 2025-05-15 Fred Hutchinson Cancer Center Compositions and methods for cellular immunotherapy
WO2025149983A2 (en) 2024-01-12 2025-07-17 Avectas Limited Delivery platform with integrated non-viral transfection and cell processing
WO2025149984A2 (en) 2024-01-12 2025-07-17 Avectas Limited Delivery platform with flow-through system (fts)
WO2025212920A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Multi-zonal liver organoids
WO2025217202A1 (en) 2024-04-08 2025-10-16 Children's Hospital Medical Center Bile duct organoid
WO2025245169A1 (en) 2024-05-21 2025-11-27 Fred Hutchinson Cancer Center Immunotherapy cells equipped with a collagen-targeting payload

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US5451513A (en) 1990-05-01 1995-09-19 The State University of New Jersey Rutgers Method for stably transforming plastids of multicellular plants
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5222982A (en) 1991-02-11 1993-06-29 Ommaya Ayub K Spinal fluid driven artificial organ
EP0571525A1 (en) 1991-02-11 1993-12-01 OMMAYA, Ayub K. Spinal fluid driven artificial organ
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
AU663725B2 (en) 1991-08-20 1995-10-19 United States Of America, Represented By The Secretary, Department Of Health And Human Services, The Adenovirus mediated transfer of genes to the gastrointestinal tract
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
FR2688514A1 (fr) 1992-03-16 1993-09-17 Centre Nat Rech Scient Adenovirus recombinants defectifs exprimant des cytokines et medicaments antitumoraux les contenant.
US7153684B1 (en) 1992-10-08 2006-12-26 Vanderbilt University Pluripotential embryonic stem cells and methods of making same
JPH08503855A (ja) 1992-12-03 1996-04-30 ジェンザイム・コーポレイション 嚢胞性線維症に対する遺伝子治療
ES2249761T3 (es) 1993-06-24 2006-04-01 Advec Inc. Vectores de adenovirus para terapia genica.
SK283703B6 (sk) 1993-10-25 2003-12-02 Canji, Inc. Rekombinantný adenovírusový vektor a jeho použitie
US5576198A (en) 1993-12-14 1996-11-19 Calgene, Inc. Controlled expression of transgenic constructs in plant plastids
US5545818A (en) 1994-03-11 1996-08-13 Calgene Inc. Expression of Bacillus thuringiensis cry proteins in plant plastids
US5545817A (en) 1994-03-11 1996-08-13 Calgene, Inc. Enhanced expression in a plant plastid
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
CA2307807C (en) 1997-10-23 2008-09-02 Andrea G. Bodnar Methods and materials for the growth of primate-derived primordial stem cells in feeder-free culture
US6667176B1 (en) 2000-01-11 2003-12-23 Geron Corporation cDNA libraries reflecting gene expression during growth and differentiation of human pluripotent stem cells
US7410798B2 (en) 2001-01-10 2008-08-12 Geron Corporation Culture system for rapid expansion of human embryonic stem cells
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
US7229961B2 (en) 1999-08-24 2007-06-12 Cellgate, Inc. Compositions and methods for enhancing drug delivery across and into ocular tissues
EP2269654A3 (en) 1999-08-24 2011-04-13 Cellgate Inc. Enhancing drug delivery across and into epithelial tissues using oligo arginine moieties
EP1083231A1 (en) 1999-09-09 2001-03-14 Introgene B.V. Smooth muscle cell promoter and uses thereof
US7256286B2 (en) 1999-11-30 2007-08-14 The Board Of Trustees Of The Leland Stanford Junior University Bryostatin analogues, synthetic methods and uses
AU2002306500C1 (en) 2001-02-16 2006-09-28 Cellgate, Inc. Transporters comprising spaced arginine moieties
US7169874B2 (en) 2001-11-02 2007-01-30 Bausch & Lomb Incorporated High refractive index polymeric siloxysilane compositions
CN101356270B (zh) 2005-12-13 2014-02-12 国立大学法人京都大学 核重新编程因子
US20090227032A1 (en) 2005-12-13 2009-09-10 Kyoto University Nuclear reprogramming factor and induced pluripotent stem cells
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
PT2019683T (pt) 2006-04-25 2017-03-17 Univ California Administração de fatores de crescimento para o tratamento de distúrbios do snc
EP2019839B1 (en) * 2006-05-25 2011-12-07 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
WO2008060360A2 (en) 2006-09-28 2008-05-22 Surmodics, Inc. Implantable medical device with apertures for delivery of bioactive agents
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
US9683232B2 (en) 2007-12-10 2017-06-20 Kyoto University Efficient method for nuclear reprogramming
US8521273B2 (en) 2008-01-29 2013-08-27 Gilbert H. KLIMAN Drug delivery devices, kits and methods therefor
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
US20130171732A1 (en) * 2011-10-06 2013-07-04 Sangamo Biosciences, Inc. Methods and compositions for regulating hiv infection
JP6144691B2 (ja) * 2011-11-16 2017-06-07 サンガモ セラピューティクス, インコーポレイテッド 修飾されたdna結合タンパク質およびその使用
CN104284669A (zh) * 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
PL4289948T3 (pl) * 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
PT2898075E (pt) * 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
AU2014207618A1 (en) * 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
KR20170108172A (ko) * 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
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

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WO2015071474A3 (en) 2015-08-27
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