ES2987399T3 - Edición génica guiada por ARN y regulación génica - Google Patents

Edición génica guiada por ARN y regulación génica Download PDF

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Publication number
ES2987399T3
ES2987399T3 ES19168481T ES19168481T ES2987399T3 ES 2987399 T3 ES2987399 T3 ES 2987399T3 ES 19168481 T ES19168481 T ES 19168481T ES 19168481 T ES19168481 T ES 19168481T ES 2987399 T3 ES2987399 T3 ES 2987399T3
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Prior art keywords
gene
cas9
expression
activity
crispr
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English (en)
Spanish (es)
Inventor
Charles Gersbach
Isaac B Hilton
Pablo Perez-Pinera
Ami M Kabadi
Pratiksha I Thakore
David Ousterout
Joshua B Black
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Duke University
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Duke University
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Rheumatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Transplantation (AREA)
ES19168481T 2013-06-05 2014-06-05 Edición génica guiada por ARN y regulación génica Active ES2987399T3 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361831481P 2013-06-05 2013-06-05
US201361839127P 2013-06-25 2013-06-25
US201361904911P 2013-11-15 2013-11-15
US201461967466P 2014-03-19 2014-03-19
US201461981575P 2014-04-18 2014-04-18

Publications (1)

Publication Number Publication Date
ES2987399T3 true ES2987399T3 (es) 2024-11-14

Family

ID=52008753

Family Applications (3)

Application Number Title Priority Date Filing Date
ES19168481T Active ES2987399T3 (es) 2013-06-05 2014-06-05 Edición génica guiada por ARN y regulación génica
ES14806852T Active ES2991293T3 (es) 2013-06-05 2014-06-05 Edición génica guiada por ARN y regulación génica
ES18172956T Active ES3029138T3 (en) 2013-06-05 2014-06-05 Rna-guided gene editing and gene regulation

Family Applications After (2)

Application Number Title Priority Date Filing Date
ES14806852T Active ES2991293T3 (es) 2013-06-05 2014-06-05 Edición génica guiada por ARN y regulación génica
ES18172956T Active ES3029138T3 (en) 2013-06-05 2014-06-05 Rna-guided gene editing and gene regulation

Country Status (9)

Country Link
US (4) US10704060B2 (enExample)
EP (5) EP3004370B1 (enExample)
JP (5) JP7085716B2 (enExample)
KR (4) KR102307280B1 (enExample)
CN (3) CN105658805B (enExample)
AU (5) AU2014274840B2 (enExample)
CA (1) CA2914519A1 (enExample)
ES (3) ES2987399T3 (enExample)
WO (1) WO2014197748A2 (enExample)

Families Citing this family (288)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
GB201122458D0 (en) * 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013119602A1 (en) 2012-02-06 2013-08-15 President And Fellows Of Harvard College Arrdc1-mediated microvesicles (armms) and uses thereof
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
BR122019025681B1 (pt) * 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
NZ732507A (en) * 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
SG10201800213VA (en) * 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
BR112016013201B1 (pt) * 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
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
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
ES2821149T3 (es) * 2014-03-12 2021-04-23 Prec Biosciences Inc Eliminación del exón del gen de la distrofina mediante nucleasas modificadas genéticamente
DK3119797T3 (da) 2014-03-18 2021-03-15 Univ Massachusetts Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
EP3122880B1 (en) 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
MX2016014066A (es) 2014-04-28 2017-05-03 Recombinetics Inc Edicion de genes multiples en cerdos.
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3180034B1 (en) * 2014-08-11 2022-04-20 The Board of Regents of The University of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
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
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US9816080B2 (en) * 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
WO2016089866A1 (en) * 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
JP6529110B2 (ja) * 2014-12-01 2019-06-12 国立大学法人 東京大学 複数のユニットが多重に連結したｄｎａカセットおよび該カセットを含むベクターの製造方法
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3237615B2 (en) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr having or associated with destabilization domains
EP3239298A4 (en) * 2014-12-26 2018-06-13 Riken Gene knockout method
CA2972454C (en) * 2014-12-31 2024-09-10 Synthetic Genomics Inc Compositions and methods for high efficiency in vivo genome editing
EP3256487A4 (en) * 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
AU2016225178B2 (en) * 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
DK3064587T3 (da) 2015-03-06 2019-09-30 Leibniz Inst Fuer Pflanzenbiochemie Ipb Bibliotek af syntetiske promotere til koordineret genekspression i eukaryotiske celler eller organismer
EP3748004A1 (en) * 2015-04-01 2020-12-09 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US11674144B2 (en) * 2015-04-16 2023-06-13 California Institute Of Technology Fractional regulation of transcription
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
JP2018522249A (ja) 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CN109536474A (zh) 2015-06-18 2019-03-29 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
US10648020B2 (en) 2015-06-18 2020-05-12 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
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
CN105255886B (zh) * 2015-07-09 2018-09-14 青岛市畜牧兽医研究所 一对靶向猪RelA基因的sgRNA
WO2017015637A1 (en) * 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
WO2017035342A2 (en) 2015-08-27 2017-03-02 University Of Washington Drug discovery platform for duchenne cardiomyopathy
JP6937513B2 (ja) * 2015-09-11 2021-09-22 国立研究開発法人理化学研究所 転写因子結合部位特異的なｄｎａ脱メチル化方法
WO2017049407A1 (en) * 2015-09-23 2017-03-30 UNIVERSITé LAVAL Modification of the dystrophin gene and uses thereof
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
IL297017A (en) 2015-10-08 2022-12-01 Harvard College Multiplexed genome editing
EP4089175A1 (en) * 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
ES3040945T3 (en) * 2015-10-28 2025-11-06 Vertex Pharma Materials and methods for treatment of duchenne muscular dystrophy
WO2017077394A2 (en) 2015-11-04 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
MX2018005377A (es) * 2015-11-30 2018-11-09 Univ Duke Dianas terapeuticas para la correccion del gen de la distrofina humana por edicion genica y metodos de uso.
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
SG10202112024PA (en) 2016-01-11 2021-12-30 Univ Leland Stanford Junior Chimeric proteins and methods of immunotherapy
CA3011458A1 (en) * 2016-01-14 2017-07-20 The Brigham And Women's Hospital, Inc. Genome editing for treating glioblastoma
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
JP6998313B2 (ja) * 2016-02-11 2022-02-04 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 細胞のゲノムにおける変異ジストロフィン遺伝子を修飾する方法及び組成物
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
IL313038A (en) * 2016-03-14 2024-07-01 Editas Medicine Inc Methods and preparations related to CRISPR/CAS - for the treatment of diseases in the hemoglobin cell
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
KR102424476B1 (ko) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
ES2957660T3 (es) 2016-05-05 2024-01-23 Univ Duke Composiciones relacionadas con crispr/cas para tratar la distrofia muscular de duchenne
CA3026321C (en) * 2016-06-02 2023-10-03 Sigma-Aldrich Co. Llc Using programmable dna binding proteins to enhance targeted genome modification
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN106119269B (zh) * 2016-06-23 2019-12-06 百奥迈科生物技术有限公司 一种在大肠杆菌中制备线性单链dna的方法
EP3481956A4 (en) 2016-07-05 2020-07-08 The Johns Hopkins University COMPOSITIONS AND METHODS COMPRISING CRISPR-GUIDED RNA ENHANCEMENTS USING THE H1 PROMOTER
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2018031762A1 (en) * 2016-08-10 2018-02-15 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
IL264872B2 (en) * 2016-08-18 2025-02-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
AU2017313616B2 (en) * 2016-08-19 2022-12-08 Institute For Basic Science Artificially engineered angiogenesis regulatory system
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CN107880132B (zh) * 2016-09-30 2022-06-17 北京大学 一种融合蛋白及使用其进行同源重组的方法
EP3518981A4 (en) 2016-10-03 2020-06-10 President and Fellows of Harvard College THERAPEUTIC RNA DELIVERY THROUGH ARRDC1 MICROVESICLES
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
CN106318947A (zh) * 2016-10-17 2017-01-11 北京大北农科技集团股份有限公司 基因组编辑系统及其用途
FR3058160A1 (fr) * 2016-10-28 2018-05-04 Universite Pierre Et Marie Curie - Paris 6 (Upmc) Procede de differenciation de cellules souches pluripotentes en cardiomyocytes
CN109689693B (zh) * 2016-11-03 2022-06-28 深圳华大生命科学研究院 提高基因编辑效率的方法和系统
CA3044531A1 (en) * 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
MX2019006426A (es) 2016-12-01 2019-08-14 Sangamo Therapeutics Inc Reguladores de tau, y composiciones y metodos para su administracion.
JP7026906B2 (ja) * 2016-12-12 2022-03-01 アステラス製薬株式会社 転写調節融合ポリペプチド
US12286727B2 (en) 2016-12-19 2025-04-29 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
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US20220283144A1 (en) * 2017-01-06 2022-09-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods identifying and using stem cell differentiation markers
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
WO2018147343A1 (en) * 2017-02-07 2018-08-16 Edigene Corporation Method of treating diseases associated with elevated kras expression using crispr-gndm system
CN110312797A (zh) 2017-02-10 2019-10-08 齐默尔根公司 组装和编辑用于多个宿主的多个dna构建体的模块化通用质粒设计策略
US20190380314A1 (en) * 2017-02-23 2019-12-19 President And Fellows Of Harvard College Methods of Genetic Modification of a Cell
WO2018160695A1 (en) * 2017-02-28 2018-09-07 Trustees Of Boston University Opto-genetic modulator
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
WO2018169983A1 (en) * 2017-03-13 2018-09-20 President And Fellows Of Harvard College Methods of modulating expression of target nucleic acid sequences in a cell
WO2018170184A1 (en) * 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11771703B2 (en) 2017-03-17 2023-10-03 The Johns Hopkins University Targeted epigenetic therapy against distal regulatory element of TGFβ2 expression
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CN107012158B (zh) * 2017-03-28 2020-09-11 东南大学 一种端粒酶启动基因表达方法及其应用
KR102758434B1 (ko) * 2017-03-30 2025-01-21 고쿠리츠 다이가쿠 호진 교토 다이가쿠 게놈 편집에 의한 엑손 스키핑 유도 방법
CN107058358B (zh) * 2017-04-01 2020-06-09 中国科学院微生物研究所 一种双spacer序列识别切割CRISPR-Cas9载体构建及其在疣孢菌中的应用
EP3385373A1 (en) 2017-04-05 2018-10-10 Centro de Neurociências e Biologia Celular Compositions for reprogramming cells into dendritic cells or antigen presenting cells, methods and uses thereof
JP7303743B2 (ja) 2017-04-05 2023-07-05 アスガード セラピューティクス エービー 細胞を樹状細胞または抗原提示細胞にリプログラムするための組成物、その方法および使用
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症（ａｌｓ）を処置する方法
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US12139720B2 (en) * 2017-06-02 2024-11-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant lentiviral vector for stem cell- based gene therapy of sickle cell disorder
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
AU2018283372B2 (en) 2017-06-16 2024-06-13 Avery Therapeutics, Inc. Three dimensional tissue compositions and methods of use
US20200140896A1 (en) * 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US20200158716A1 (en) * 2017-07-17 2020-05-21 Massachusetts Institute Of Technology Cell atlas of healthy and diseased barrier tissues
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
CN107488649A (zh) * 2017-08-25 2017-12-19 南方医科大学 一种Cpf1和p300核心结构域的融合蛋白、相应的DNA靶向激活系统和应用
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019055536A1 (en) * 2017-09-14 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR ENHANCING THE EFFICACY OF CELL TRANSPLANTATION
CN107513531B (zh) * 2017-09-21 2020-02-21 无锡市妇幼保健院 用于内源性过表达lncRNA-XIST的gRNA靶点序列及其应用
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
CN107722125B (zh) * 2017-09-28 2021-05-07 中山大学 一种人工转录激活因子dCas9-TV及其编码基因与应用
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
WO2019089623A1 (en) * 2017-10-30 2019-05-09 Children's Hospital Medical Center Fusion proteins for use in improving gene correction via homologous recombination
BR112020008654A2 (pt) * 2017-11-01 2020-11-10 The Regents Of The University Of California composições casz e métodos de uso
WO2019090160A2 (en) * 2017-11-03 2019-05-09 Hunterian Medicine Llc Compositions and methods of use thereof for the treatment of duchenne muscular dystrophy
KR102132198B1 (ko) * 2017-12-14 2020-07-09 단국대학교 산학협력단 Tert 프로모터 돌연변이에 특이적인 crispr 시스템 및 그의 이용
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
MX2020006800A (es) 2017-12-28 2020-09-03 Takeda Pharmaceuticals Co Lipidos cationicos.
CN111527204B (zh) 2017-12-28 2023-09-01 国立大学法人京都大学 靶基因改造用组合物
CN108559731A (zh) * 2018-01-15 2018-09-21 南方医科大学 一种可调控基因表达的人类胚胎干细胞系及其应用
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
SG11202007382TA (en) * 2018-02-15 2020-08-28 Sigma Aldrich Co Llc Engineered cas9 systems for eukaryotic genome modification
CN110229814B (zh) * 2018-03-06 2025-11-14 中国科学院动物研究所 改进的向导rna
SG11202008956XA (en) 2018-03-14 2020-10-29 Editas Medicine Inc Systems and methods for the treatment of hemoglobinopathies
CN112334577B (zh) 2018-04-19 2023-10-17 加利福尼亚大学董事会 用于基因编辑的组合物和方法
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
CN112166194A (zh) * 2018-04-23 2021-01-01 杜克大学 通过dna甲基化的靶向编辑下调snca的表达
WO2019210279A1 (en) * 2018-04-27 2019-10-31 The Regents Of The University Of California De novo formation of the biliary system by hepatocyte transdifferentiation
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12297241B2 (en) 2018-07-06 2025-05-13 The Regents Of The University Of California Method to engineer transplantable human tissues
CN109055375B (zh) * 2018-07-26 2021-11-12 东南大学 一种crispr辅助反式增强子激活基因表达的方法及其应用
CN110760511B (zh) * 2018-07-27 2021-09-07 广东赤萌医疗科技有限公司 一种用于治疗杜氏肌营养不良症的gRNA、表达载体、CRISPR-Cas9系统
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
GB201813011D0 (en) 2018-08-10 2018-09-26 Vib Vzw Means and methods for drought tolerance in crops
CN112703250A (zh) * 2018-08-15 2021-04-23 齐默尔根公司 CRISPRi在高通量代谢工程中的应用
EP3836974A4 (en) 2018-08-18 2022-06-15 President and Fellows of Harvard College IN SITU GENE EDIT
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
JP7250031B2 (ja) * 2018-09-25 2023-03-31 公益財団法人微生物化学研究会 新規ウイルスベクターおよびその製造方法と使用方法
CN113226333A (zh) 2018-10-02 2021-08-06 桑格摩生物治疗股份有限公司 用于调控Tau蛋白的方法和组合物
CA3113817A1 (en) * 2018-10-09 2020-04-16 The University Of North Carolina At Chapel Hill Regulated gene editing system
WO2020081604A2 (en) * 2018-10-15 2020-04-23 Avery Therapeutics, Inc. Cell-free compositions and methods for restoration or enhancement of tissue function
WO2020081922A1 (en) 2018-10-18 2020-04-23 University Of Utah Research Foundation Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020092725A1 (en) * 2018-11-01 2020-05-07 Montana State University Gene modulation with crispr system type i
WO2020097083A1 (en) * 2018-11-06 2020-05-14 Cellino Biotech, Inc. Systems for cell control
BR112021009481A2 (pt) * 2018-11-16 2021-08-17 Astellas Pharma Inc. método para tratamento da distrofia muscular direcionando gene de utrofina.
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220079972A1 (en) * 2019-01-28 2022-03-17 The Administrators Of The Tulane Educational Fund Crispr-based methods and novel compositions for treating vascular disorders
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
CN109929865B (zh) * 2019-03-11 2022-03-08 东南大学 基于gal4-uas系统的crispr辅助反式增强子激活基因表达的方法及其应用
DE112020001306T5 (de) * 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US20220145249A1 (en) * 2019-04-11 2022-05-12 Agency For Science, Technology And Research A Method of Altering a Differentiation Status of a Cell
JP2022526669A (ja) * 2019-04-12 2022-05-25 デューク ユニバーシティ ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物
EP3952925A4 (en) * 2019-04-12 2024-01-24 The Regents of the University of California COMPOSITIONS AND METHODS FOR MODIFYING DYSTROPHIN GENES
US20220184229A1 (en) * 2019-04-14 2022-06-16 Duke University Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
EP3930766A4 (en) * 2019-04-14 2023-02-22 Duke University CRISPR/CAS-BASED GENE EDITING COMPOSITION TO RESTORE DYSTROPHIN FUNCTION
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020232373A1 (en) * 2019-05-15 2020-11-19 Board Of Regents, The University Of Texas System Crispr methods for treating cancers
AU2020280107A1 (en) * 2019-05-23 2021-12-23 American Molecular Laboratories, Inc. Methods for detecting a level of H. pylori in a fecal sample
CN112266418A (zh) * 2019-07-08 2021-01-26 中国科学院微生物研究所 改进的基因组编辑系统及其应用
JP7708737B2 (ja) 2019-07-18 2025-07-15 ユニバーシティー オブ ロチェスター 細胞の細胞型選択的免疫保護
WO2021051054A1 (en) * 2019-09-13 2021-03-18 The Regents Of The University Of Colorado, A Body Corporate Methods and compositions for cell and tissue rejuvenation
US20220409744A1 (en) * 2019-09-23 2022-12-29 Vigeneron Gmbh Method of transactivating a homologous gene of a gene of interest and an in vitro method of diagnosing a disease
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021075555A1 (ja) * 2019-10-18 2021-04-22 株式会社Ｅｐｉｇｅｎｅｒｏｎ 標的核酸の検出方法、核酸結合分子の検出方法、及び核酸結合能の評価方法
AU2020378619A1 (en) 2019-11-06 2022-06-09 Association Institut De Myologie Combined therapy for muscular diseases
WO2021113536A1 (en) * 2019-12-03 2021-06-10 Duke University Systems and methods for lipid nanoparticle delivery of gene editing machinery
WO2021127057A1 (en) * 2019-12-16 2021-06-24 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
EP4126224A4 (en) * 2020-04-27 2024-07-03 Duke University HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
EP4125349A4 (en) * 2020-04-27 2024-07-10 Duke University GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
WO2021231575A1 (en) * 2020-05-12 2021-11-18 Myogene Bio Llc Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
WO2022008557A2 (en) 2020-07-08 2022-01-13 UCB Biopharma SRL Modulation of cftr expression
US20230313186A1 (en) * 2020-07-24 2023-10-05 The Trustees Of The University Of Pennsylvania Trophin genome editing for treating duchenne muscular dystrophy (dmd)
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
AU2021345112A1 (en) 2020-09-15 2023-04-27 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
US20240043818A1 (en) * 2020-10-15 2024-02-08 Northeastern University Engineered Cells for Increased Collagen Production
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
CN112662674B (zh) * 2021-01-12 2023-04-11 广州瑞风生物科技有限公司 靶向编辑VEGFA基因外显子区域的gRNA及其应用
CN114854791A (zh) * 2021-02-04 2022-08-05 北京中因科技有限公司 一种新型CRISPR-Cas9系统载体及其应用
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
CN112779259B (zh) * 2021-03-18 2023-05-26 新疆畜牧科学院生物技术研究所（新疆畜牧科学院中国-澳大利亚绵羊育种研究中心） 一种用于精准编辑绵羊OCT4基因的sgRNA、扩增用引物和应用
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
CN113637658A (zh) * 2021-08-19 2021-11-12 中国农业科学院棉花研究所 基于dCas9-oToV的基因转录系统及其应用
EP4377465A4 (en) * 2021-09-10 2025-05-21 The Regents of the University of California TEMPERATURE-REGULATED CRISPR-CAS SYSTEMS AND METHODS OF USE THEREOF
WO2023050158A1 (zh) * 2021-09-29 2023-04-06 深圳先进技术研究院 一种实现多碱基编辑的方法
EP4463548A1 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250290100A1 (en) * 2022-04-26 2025-09-18 University of Massaachusetts SINGLE pegRNA-MEDIATED LARGE INSERTIONS
CN114941032B (zh) * 2022-06-13 2025-05-06 大连医科大学 Rnf122在制备抗肿瘤药物中的应用
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
CN119487186A (zh) 2022-06-24 2025-02-18 欧洲分子生物学实验室 用于在目标基因座特异性引入表观遗传性修饰的基于crispr的模块化工具
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics Inc TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
CA3264214A1 (en) 2022-08-19 2024-02-22 Tune Therapeutics Inc Compositions, systems and methods of regulating the hepatitis B virus by targeted gene suppression
CN120239746A (zh) 2022-09-19 2025-07-01 图恩疗法股份有限公司 用于调节t细胞功能的组合物、系统和方法
WO2024081937A2 (en) * 2022-10-13 2024-04-18 Duke University Cas12a fusion proteins and methods of using same
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
EP4662319A2 (en) * 2023-02-10 2025-12-17 Integrate Bioscience LLC Personalized crispr profiling for cancer
WO2024206714A1 (en) * 2023-03-31 2024-10-03 Mammoth Biosciences, Inc. Engineered effector proteins, compositions, systems and methods of use thereof
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
CN118291415B (zh) * 2024-03-14 2024-11-19 内蒙古大学 一种CRISPR::Cr-P300M巴豆酰化修饰编辑工具及其应用

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
AU4528493A (en) 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
PT681483E (pt) 1993-01-26 2005-11-30 Wyeth Corp Composicoes e metodos para distribuicao de material genetico
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US7329728B1 (en) * 1999-10-25 2008-02-12 The Scripps Research Institute Ligand activated transcriptional regulator proteins
WO2001083783A2 (en) 2000-04-28 2001-11-08 Genzyme Corporation In vivo loading of mhc
US20040175727A1 (en) 2002-11-04 2004-09-09 Advisys, Inc. Synthetic muscle promoters with activities exceeding naturally occurring regulatory sequences in cardiac cells
WO2004048583A2 (en) * 2002-11-22 2004-06-10 Institut Clayton De La Recherche Compositions and systems for the regulation of genes
US20070185042A1 (en) 2003-08-08 2007-08-09 The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College Sirna based methods for treating alzheimer's disease
ATE498685T1 (de) * 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
KR20090031938A (ko) 2006-07-05 2009-03-30 더 스크립스 리서치 인스티튜트 방향성 진화로 촉매 작용을 최적화시킨 키메라 징크 핑거 리컴비나제
PT2203173E (pt) * 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
EP2998402A1 (en) 2008-10-17 2016-03-23 Joule Unlimited Technologies, Inc. Ethanol production by microorganisms
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
WO2011036640A2 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
RU2712833C2 (ru) * 2009-10-22 2020-01-31 ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи Конструируемые белки с цинковыми пальцами, направленные на гены растений, вовлеченные в биосинтез жирных кислот
BR112012020257A8 (pt) 2010-02-11 2018-02-14 Recombinetics Inc métodos e aparelhos para produzir artiodátilos transgênicos
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
CN102643852B (zh) * 2011-02-28 2015-04-08 华东理工大学 光可控的基因表达系统
EP2729567B1 (en) 2011-07-08 2016-10-05 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenssis
US20130137173A1 (en) * 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
NZ627896A (en) * 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP2834357B1 (en) 2012-04-04 2017-12-27 Life Technologies Corporation Tal-effector assembly platform, customized services, kits and assays
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
FI3597749T3 (fi) * 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
US8884771B2 (en) * 2012-08-01 2014-11-11 Microchip Technology Incorporated Smoke detection using change in permittivity of capacitor air dielectric
AU2013308770B2 (en) * 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
BR122019025681B1 (pt) * 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
PT3363902T (pt) * 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
KR102874079B1 (ko) * 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
EP2973735B1 (en) 2013-03-15 2020-02-12 Edward J. Britt Energy conversion device and method for making and using same
AU2014265331B2 (en) * 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
RU2690935C2 (ru) * 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
JP7085716B2 (ja) * 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
CN110997908A (zh) * 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶

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