JP2016501531A - 配列操作および治療適用のための系、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 - Google Patents

配列操作および治療適用のための系、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 Download PDF

Info

Publication number
JP2016501531A
JP2016501531A JP2015547543A JP2015547543A JP2016501531A JP 2016501531 A JP2016501531 A JP 2016501531A JP 2015547543 A JP2015547543 A JP 2015547543A JP 2015547543 A JP2015547543 A JP 2015547543A JP 2016501531 A JP2016501531 A JP 2016501531A
Authority
JP
Japan
Prior art keywords
sequence
crispr
target
tracr
polynucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2015547543A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016501531A5 (cg-RX-API-DMAC7.html
Inventor
フェン・ジャン
マティアス・ハイデンライク
フェイ・ラン
ウーカシュ・スウィーチ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Broad Institute Inc
Harvard University
Original Assignee
Broad Institute Inc
Harvard University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49883289&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP2016501531(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Broad Institute Inc, Harvard University filed Critical Broad Institute Inc
Publication of JP2016501531A publication Critical patent/JP2016501531A/ja
Publication of JP2016501531A5 publication Critical patent/JP2016501531A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, 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
    • C12N15/11—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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201—Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8213—Targeted insertion of genes into the plant genome by homologous recombination
    • 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
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • 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
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/20—Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/30—Detection of binding sites or motifs
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/50—Mutagenesis
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10—Sequence alignment; Homology search
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Environmental Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Theoretical Computer Science (AREA)
  • Analytical Chemistry (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Medical Informatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Evolutionary Biology (AREA)
  • Rheumatology (AREA)
JP2015547543A 2012-12-12 2013-12-12 配列操作および治療適用のための系、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 Pending JP2016501531A (ja)

Applications Claiming Priority (27)

Application Number Priority Date Filing Date Title
US201261736527P 2012-12-12 2012-12-12
US61/736,527 2012-12-12
US201361748427P 2013-01-02 2013-01-02
US61/748,427 2013-01-02
US201361758468P 2013-01-30 2013-01-30
US61/758,468 2013-01-30
US201361769046P 2013-02-25 2013-02-25
US61/769,046 2013-02-25
US201361791409P 2013-03-15 2013-03-15
US201361802174P 2013-03-15 2013-03-15
US61/791,409 2013-03-15
US61/802,174 2013-03-15
US201361806375P 2013-03-28 2013-03-28
US61/806,375 2013-03-28
US201361814263P 2013-04-20 2013-04-20
US61/814,263 2013-04-20
US201361819803P 2013-05-06 2013-05-06
US61/819,803 2013-05-06
US201361828130P 2013-05-28 2013-05-28
US61/828,130 2013-05-28
US201361836123P 2013-06-17 2013-06-17
US201361835931P 2013-06-17 2013-06-17
US61/836,123 2013-06-17
US61/835,931 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US61/847,537 2013-07-17
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019039953A Division JP7013406B2 (ja) 2012-12-12 2019-03-05 配列操作および治療適用のための系、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

Publications (2)

Publication Number Publication Date
JP2016501531A true JP2016501531A (ja) 2016-01-21
JP2016501531A5 JP2016501531A5 (cg-RX-API-DMAC7.html) 2017-02-16

Family

ID=49883289

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2015547543A Pending JP2016501531A (ja) 2012-12-12 2013-12-12 配列操作および治療適用のための系、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
JP2019039953A Active JP7013406B2 (ja) 2012-12-12 2019-03-05 配列操作および治療適用のための系、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
JP2022006048A Active JP7228059B2 (ja) 2012-12-12 2022-01-19 配列操作および治療適用のための系、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
JP2023018822A Active JP7736729B2 (ja) 2012-12-12 2023-02-10 配列操作および治療適用のための系、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
JP2025142590A Pending JP2025181841A (ja) 2012-12-12 2025-08-28 配列操作および治療適用のための系、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

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2019039953A Active JP7013406B2 (ja) 2012-12-12 2019-03-05 配列操作および治療適用のための系、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
JP2022006048A Active JP7228059B2 (ja) 2012-12-12 2022-01-19 配列操作および治療適用のための系、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
JP2023018822A Active JP7736729B2 (ja) 2012-12-12 2023-02-10 配列操作および治療適用のための系、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
JP2025142590A Pending JP2025181841A (ja) 2012-12-12 2025-08-28 配列操作および治療適用のための系、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

Country Status (15)

Country Link
US (10) US20140179770A1 (cg-RX-API-DMAC7.html)
EP (5) EP3327127B1 (cg-RX-API-DMAC7.html)
JP (5) JP2016501531A (cg-RX-API-DMAC7.html)
KR (1) KR20150105956A (cg-RX-API-DMAC7.html)
CN (2) CN105164264B (cg-RX-API-DMAC7.html)
AU (4) AU2013359199C1 (cg-RX-API-DMAC7.html)
BR (1) BR112015013784A2 (cg-RX-API-DMAC7.html)
DK (2) DK2931897T3 (cg-RX-API-DMAC7.html)
ES (2) ES2658401T3 (cg-RX-API-DMAC7.html)
IL (2) IL239317B (cg-RX-API-DMAC7.html)
MX (2) MX389793B (cg-RX-API-DMAC7.html)
RU (1) RU2721275C2 (cg-RX-API-DMAC7.html)
SG (3) SG10201912328UA (cg-RX-API-DMAC7.html)
WO (1) WO2014093622A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201504132B (cg-RX-API-DMAC7.html)

Cited By (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521554A (ja) * 2013-06-04 2016-07-25 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性転写制御
JP2016534125A (ja) * 2013-08-29 2016-11-04 テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション Ｈｉｖ感染のｒｎａガイド処置のための方法および組成物
JP2020513832A (ja) * 2017-03-22 2020-05-21 ノバルティス アーゲー 免疫腫瘍学のための組成物および方法
JPWO2019017321A1 (ja) * 2017-07-18 2020-05-28 国立大学法人京都大学 遺伝子変異導入方法
JP2021500864A (ja) * 2017-09-29 2021-01-14 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法
US10930367B2 (en) 2012-12-12 2021-02-23 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for Cas enzymes or CRISPR-Cas systems for target sequences and conveying results thereof
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
JP2021513866A (ja) * 2018-02-27 2021-06-03 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル アンジェルマン症候群を処置する方法および組成物
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
JP2021519067A (ja) * 2018-03-23 2021-08-10 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 常染色体優性疾患のための遺伝子編集
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法
JP2021192624A (ja) * 2013-03-15 2021-12-23 ザ ジェネラル ホスピタル コーポレイション 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2022514214A (ja) * 2018-12-05 2022-02-10 インテリア セラピューティクス，インコーポレーテッド 修飾されたアミン脂質
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
US11578312B2 (en) 2015-06-18 2023-02-14 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
US11591581B2 (en) 2013-12-12 2023-02-28 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11597919B2 (en) 2013-12-12 2023-03-07 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
WO2023038020A1 (ja) 2021-09-07 2023-03-16 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法
US11624078B2 (en) 2014-12-12 2023-04-11 The Broad Institute, Inc. Protected guide RNAS (pgRNAS)
US12018275B2 (en) 2013-06-17 2024-06-25 The Broad Institute, Inc. Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy
US12037583B2 (en) 2015-12-04 2024-07-16 Novartis Ag Compositions and methods for immunooncology
US12123032B2 (en) 2015-06-18 2024-10-22 The Broad Institute, Inc. CRISPR enzyme mutations reducing off-target effects
US12251450B2 (en) 2013-12-12 2025-03-18 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12435320B2 (en) 2014-12-24 2025-10-07 The Broad Institute, Inc. CRISPR having or associated with destabilization domains
US12441995B2 (en) 2013-06-17 2025-10-14 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof

Families Citing this family (862)

* 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
NO2792236T3 (cg-RX-API-DMAC7.html) 2009-07-08 2018-04-14
US9445581B2 (en) 2012-03-28 2016-09-20 Kymab Limited Animal models and therapeutic molecules
JP2013533847A (ja) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ コレステロール関連障害のａａｖベースの治療
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
CN103429739B (zh) 2010-05-12 2018-11-13 哥伦比亚大学纽约管理委员会 制备产生和分泌胰岛素的肠内分泌细胞的方法
AR085533A1 (es) 2011-03-23 2013-10-09 Pioneer Hi Bred Int Metodos de produccion de un locus de rasgos transgenicos complejo
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2758535B1 (en) 2011-09-19 2016-11-09 Kymab Limited Antibodies, variable domains&chains tailored for human use
EP2761008A1 (en) 2011-09-26 2014-08-06 Kymab Limited Chimaeric surrogate light chains (slc) comprising human vpreb
GB201117313D0 (en) 2011-10-07 2011-11-16 Gt Biolog Ltd Bacterium for use in medicine
JP2014531908A (ja) 2011-10-14 2014-12-04 プレジデント アンド フェローズ オブ ハーバード カレッジ 構造アッセンブリによる配列決定
US9253965B2 (en) 2012-03-28 2016-02-09 Kymab Limited Animal models and therapeutic molecules
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
WO2013139861A1 (en) 2012-03-20 2013-09-26 Luc Montagnier Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9637739B2 (en) * 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US10251377B2 (en) 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
GB2502127A (en) 2012-05-17 2013-11-20 Kymab Ltd Multivalent antibodies and in vivo methods for their production
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
KR101904508B1 (ko) 2012-04-25 2018-10-04 리제너론 파마슈티칼스 인코포레이티드 큰 표적화 벡터를 사용한 뉴클레아제-중재된 표적화
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US20130297023A1 (en) * 2012-05-07 2013-11-07 Hee-Jeong Im Sampen Methods and Devices For Treating Intervertebral Disc Disease
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP4556564A3 (en) 2012-10-23 2025-09-03 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
KR20150105635A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
US20140186919A1 (en) 2012-12-12 2014-07-03 Feng Zhang Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US9970024B2 (en) * 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering
US10544405B2 (en) * 2013-01-16 2020-01-28 Emory University Cas9-nucleic acid complexes and uses related thereto
EP3578666A1 (en) 2013-03-12 2019-12-11 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9788534B2 (en) 2013-03-18 2017-10-17 Kymab Limited Animal models and therapeutic molecules
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CN115261411A (zh) * 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
GB201306536D0 (en) 2013-04-10 2013-05-22 Gt Biolog Ltd Polypeptide and immune modulation
KR20200136508A (ko) 2013-04-16 2020-12-07 리제너론 파마슈티칼스 인코포레이티드 랫트 게놈의 표적화된 변형
US9783618B2 (en) 2013-05-01 2017-10-10 Kymab Limited Manipulation of immunoglobulin gene diversity and multi-antibody therapeutics
US9783593B2 (en) 2013-05-02 2017-10-10 Kymab Limited Antibodies, variable domains and chains tailored for human use
US11707056B2 (en) 2013-05-02 2023-07-25 Kymab Limited Animals, repertoires and methods
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
DK3004349T3 (en) * 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6718813B2 (ja) * 2013-07-10 2020-07-08 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 Ｒｎａ誘導性遺伝子制御および編集のための直交性ｃａｓ９タンパク質
SI3019619T1 (sl) * 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
EP3022304B1 (en) * 2013-07-19 2018-12-26 Larix Biosciences LLC Methods and compositions for producing double allele knock outs
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 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
MX2016002306A (es) 2013-08-22 2016-07-08 Du Pont Promotor u6 de polimerasa iii de soja y metodos de uso.
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
ES2844174T3 (es) * 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
SG11201602236SA (en) 2013-10-01 2016-04-28 Kymab Ltd Animal models and therapeutic molecules
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3071695A2 (en) * 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
JP6174811B2 (ja) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ゲノムの標的改変のための方法及び組成物
PL3080279T3 (pl) 2013-12-11 2019-03-29 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do celowanej modyfikacji genomu
WO2015089419A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CN105899657A (zh) 2013-12-12 2016-08-24 布罗德研究所有限公司 用于改变基因产物表达的crispr-cas系统和方法、结构信息以及诱导型模块化cas酶
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3092049A1 (en) 2014-01-08 2016-11-16 Flodesign Sonics Inc. Acoustophoresis device with dual acoustophoretic chamber
MX372651B (es) * 2014-01-31 2020-06-16 Factor Bioscience Inc ARN SINTÉTICO PARA SU Uso EN EL TRATAMIENTO DE LA EPIDERMÓLISIS AMPOLLOSA DISTRÓFICA.
WO2015123339A1 (en) 2014-02-11 2015-08-20 The Regents Of The University Of Colorado, A Body Corporate Crispr enabled multiplexed genome engineering
US10286084B2 (en) 2014-02-18 2019-05-14 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
WO2015130968A2 (en) 2014-02-27 2015-09-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
WO2015153780A1 (en) * 2014-04-02 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
WO2015168800A1 (en) * 2014-05-09 2015-11-12 UNIVERSITé LAVAL Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
EP3147277B1 (en) * 2014-05-20 2023-10-04 National University Corporation Hokkaido University Lipid membrane structure for sirna intracellular delivery
CN106661548B (zh) 2014-05-28 2020-12-11 儿童医院医疗中心 用于经由定向分化将前体细胞转化为胃组织的方法和系统
US10066241B2 (en) * 2014-05-30 2018-09-04 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
RU2704283C9 (ru) 2014-06-06 2020-02-07 Регенерон Фармасьютикалз, Инк. Способы и композиции для модификации целевого локуса
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
CA2953499C (en) 2014-06-23 2023-10-24 Regeneron Pharmaceuticals, Inc. Nuclease-mediated dna assembly
IL286474B2 (en) 2014-06-23 2023-11-01 Massachusetts Gen Hospital Genome-wide random identification of DSBS assessed by sequencing (guide-sequence)
EP3160503B1 (en) 2014-06-26 2021-02-17 The Trustees of Columbia University in the City of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
PT3161128T (pt) 2014-06-26 2018-11-21 Regeneron Pharma Métodos e composições para modificações genéticas alvejadas e métodos de utilização
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
US10760040B1 (en) 2014-07-03 2020-09-01 NanoCav, LLC Mechanical transfection devices and methods
US10081816B1 (en) * 2014-07-03 2018-09-25 Nant Holdings Ip, Llc Mechanical transfection devices and methods
EP2966170A1 (en) * 2014-07-10 2016-01-13 Heinrich-Pette-Institut Leibniz-Institut für experimentelle Virologie-Stiftung bürgerlichen Rechts - HBV inactivation
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
CA2954686A1 (en) * 2014-07-11 2016-01-14 Pioneer Hi-Bred International, Inc. Agronomic trait modification using guide rna/cas endonuclease systems and methods of use
AU2015288157A1 (en) 2014-07-11 2017-01-19 E. I. Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
US9850521B2 (en) * 2014-08-01 2017-12-26 Agilent Technologies, Inc. In vitro assay buffer for Cas9
CA2959130A1 (en) * 2014-08-11 2016-02-18 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
US11071289B2 (en) 2014-08-14 2021-07-27 Biocytogen Boston Corp DNA knock-in system
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
DK3186376T3 (da) * 2014-08-27 2019-05-06 Caribou Biosciences Inc Fremgangsmåder til forbedring af cas9-medieret manipulationseffektivitet
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
WO2016040030A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CN104293831A (zh) * 2014-09-28 2015-01-21 上海云舜生物技术有限公司 建立高血压小鼠模型的方法及其用途
WO2016054554A1 (en) 2014-10-03 2016-04-07 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
JP6723230B2 (ja) * 2014-10-09 2020-07-15 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 神経障害性疼痛を治療するためのｃｓｆ１−ｄａｐ１２経路のメンバー遺伝子の標的破壊
US9943612B2 (en) 2014-10-09 2018-04-17 Seattle Children's Hospital Long poly(A) plasmids and methods for introduction of long poly(A) sequences into the plasmid
AU2015330855A1 (en) 2014-10-09 2017-04-27 Celularity Inc. Placenta-derived adherent cell exosomes and uses thereof
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3207124B1 (en) 2014-10-15 2019-06-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
WO2016061464A1 (en) 2014-10-17 2016-04-21 Children's Hospital Center, D/B/A Cincinnati Children's Hospital Medical Center In vivo model of human small intetine using pluripotent stem cells and methods of making and using same
EP3207131B1 (en) * 2014-10-17 2022-09-28 Howard Hughes Medical Institute Genomic probes
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
WO2016069591A2 (en) 2014-10-27 2016-05-06 The Broad Institute Inc. Compositions, methods and use of synthetic lethal screening
EP3212221B1 (en) * 2014-10-29 2023-12-06 Massachusetts Eye & Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
EP3212789B1 (en) * 2014-10-31 2020-04-22 Massachusetts Institute of Technology Massively parallel combinatorial genetics for crispr
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
EP3521437B1 (en) 2014-11-21 2025-01-15 Regeneron Pharmaceuticals, Inc. Methods for targeted genetic modification using paired guide rnas
WO2016086197A1 (en) 2014-11-25 2016-06-02 The Brigham And Women's Hospital, Inc. Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease
EP3224362B1 (en) 2014-11-26 2024-09-25 The Regents of The University of California Therapeutic compositions comprising transcription factors and methods of making and using the same
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016094679A1 (en) 2014-12-10 2016-06-16 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016100857A1 (en) 2014-12-19 2016-06-23 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
SG11201704646YA (en) 2014-12-19 2017-07-28 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
HRP20180847T1 (hr) 2014-12-23 2018-08-24 4D Pharma Research Limited Pirinski polipeptid i imunološka modulacija
CA2971108C (en) 2014-12-23 2019-09-17 4D Pharma Research Limited A bacteroides thetaiotaomicron strain and use thereof in reducing inflammation
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
AU2015373893B2 (en) 2014-12-31 2021-07-29 Synthetic Genomics, Inc. Compositions and methods for high efficiency in vivo genome editing
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
US10059940B2 (en) 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
SI3250691T1 (sl) 2015-01-28 2023-10-30 Caribou Biosciences, Inc. Hibridni dna/rna-polinukleotidi crispr in postopki za uporabo
EP3250689B1 (en) 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3262193A2 (en) 2015-02-26 2018-01-03 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
JP6871169B2 (ja) 2015-03-02 2021-05-12 シナイ ヘルス システム 相同組換え因子
WO2016141224A1 (en) * 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CN104673816A (zh) * 2015-03-05 2015-06-03 广东医学院 一种pCr-NHEJ载体及其构建方法及其用于细菌基因定点敲除的应用
CN106032540B (zh) * 2015-03-16 2019-10-25 中国科学院上海生命科学研究院 CRISPR/Cas9核酸内切酶体系的腺相关病毒载体构建及其用途
GB2536650A (en) 2015-03-24 2016-09-28 Augmedics Ltd Method and system for combining video-based and optic-based augmented reality in a near eye display
US20180066253A1 (en) * 2015-03-26 2018-03-08 Yale University Methods and compositions for modifying endothelial cells
BR112017017260A2 (pt) 2015-03-27 2018-04-17 Du Pont construções de dna, vetor, célula, plantas, semente, método de expressão de rna e método de modificação de local alvo
CA3000187A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
EP4335918A3 (en) * 2015-04-03 2024-04-17 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
CA2981715A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
CA2988854A1 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
WO2016187717A1 (en) * 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
US20160346360A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
KR20230110368A (ko) 2015-06-01 2023-07-21 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염의 rna-가이드된 치료를 위한 방법 및 조성물
UY36703A (es) 2015-06-02 2016-12-30 Monsanto Technology Llc Composiciones y métodos para la administración de un polinucleótido en una planta
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
CN108368502B (zh) 2015-06-03 2022-03-18 内布拉斯加大学董事委员会 使用单链dna的dna编辑
GB201509578D0 (en) * 2015-06-03 2015-07-15 Univ Singapore Vectors
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
JP6439037B2 (ja) 2015-06-15 2018-12-19 フォーディー ファーマ リサーチ リミテッド４Ｄ Ｐｈａｒｍａ Ｒｅｓｅａｒｃｈ Ｌｉｍｉｔｅｄ 細菌株を含む組成物
MA41060B1 (fr) 2015-06-15 2019-11-29 4D Pharma Res Ltd Compositions comprenant des souches bactériennes
MA41010B1 (fr) 2015-06-15 2020-01-31 4D Pharma Res Ltd Compositions comprenant des souches bactériennes
HUE045413T2 (hu) 2015-06-15 2019-12-30 4D Pharma Res Ltd Baktériumtörzseket tartalmazó készítmények
ES2753779T3 (es) 2015-06-15 2020-04-14 4D Pharma Res Ltd Blautia stercosis y wexlerae para la utilización en el tratamiento de enfermedades inflamatorias y autoinmunes
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
EP3310395A4 (en) * 2015-06-18 2019-05-22 Robert D. Bowles REGULATION OF RNA GUIDED TRANSCRIPTION AND METHODS OF USE THEREOF FOR THE TREATMENT OF LOMBALGIA
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016208776A1 (ja) 2015-06-25 2016-12-29 株式会社国際電気通信基礎技術研究所 多器官連関システムを基盤とした予測装置、及び予測プログラム
WO2016210416A2 (en) * 2015-06-26 2016-12-29 The Trustees Of Columbia University In The City Of New York Genetically modified ips cells that carry a marker to report expression of neurogenin3, tph2, foxo1 and/or insulin genes
CA2990699A1 (en) 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
US20170000743A1 (en) * 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
US10456452B2 (en) 2015-07-02 2019-10-29 Poseida Therapeutics, Inc. Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles
WO2017007825A1 (en) * 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
CN116212052A (zh) * 2015-07-25 2023-06-06 哈比卜·弗罗斯特 用于为癌症和其他病理状态提供治疗或治愈的系统、装置和方法
DK3329001T3 (da) * 2015-07-28 2021-12-20 Danisco Us Inc Genomredigeringssystemer og anvendelsesfremgangsmåder
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
GB2592821B (en) * 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
US10842812B2 (en) 2015-08-12 2020-11-24 The General Hospital Corporation Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders
WO2017027910A1 (en) 2015-08-14 2017-02-23 The University Of Sydney Connexin 45 inhibition for therapy
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
EP4345454A3 (en) * 2015-08-25 2024-07-17 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP3341477B1 (en) 2015-08-28 2022-03-23 The General Hospital Corporation Engineered crispr-cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
AU2016319110B2 (en) 2015-09-11 2022-01-27 The General Hospital Corporation Full interrogation of nuclease DSBs and sequencing (FIND-seq)
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
US11667911B2 (en) 2015-09-24 2023-06-06 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/CAS-mediated genome editing
WO2017059313A1 (en) 2015-09-30 2017-04-06 The General Hospital Corporation Comprehensive in vitro reporting of cleavage events by sequencing (circle-seq)
WO2017062754A1 (en) * 2015-10-07 2017-04-13 New York University Compositions and methods for enhancing crispr activity by polq inhibition
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
CN109153980B (zh) 2015-10-22 2023-04-14 布罗德研究所有限公司 Vi-b型crispr酶和系统
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075265A1 (en) 2015-10-28 2017-05-04 The Broad Institute, Inc. Multiplex analysis of single cell constituents
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
HK1252878A1 (zh) 2015-11-03 2019-06-06 President And Fellows Of Harvard College 用於含有基质的三维核酸的体积成像的方法和设备
EP3373938A4 (en) * 2015-11-09 2019-05-15 Seattle Children's Hospital (DBA Seattle Children's Research Institute) NOVEL RNA-BASED VECTOR SYSTEM FOR TEMPORARY AND STABLE GENE EXPRESSION
EP3374494A4 (en) * 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY
WO2017083852A1 (en) 2015-11-13 2017-05-18 MOORE, Tara Methods for the treatment of corneal dystrophies
CA3005633C (en) 2015-11-16 2023-11-21 Research Institute Of Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopathies
CN106893739A (zh) 2015-11-17 2017-06-27 香港中文大学 用于靶向基因操作的新方法和系统
WO2017087708A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
GB201520497D0 (en) 2015-11-20 2016-01-06 4D Pharma Res Ltd Compositions comprising bacterial strains
HUE036362T2 (hu) 2015-11-20 2018-07-30 4D Pharma Res Ltd Baktériumtörzseket tartalmazó készítmények
CN109072235B (zh) 2015-11-23 2023-02-28 加利福尼亚大学董事会 通过核递送crispr/cas9追踪并操纵细胞rna
EP3380635A2 (en) * 2015-11-25 2018-10-03 Integrated DNA Technologies Inc. Methods for variant detection
US20210395799A1 (en) * 2015-11-25 2021-12-23 Integrated Dna Technologies, Inc. Methods for variant detection
EP4644567A2 (en) 2015-11-30 2025-11-05 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
CN105624196A (zh) * 2015-12-24 2016-06-01 江苏大学 一种建立cyp2c11基因敲除大鼠模型的方法
CN105624191A (zh) * 2015-12-24 2016-06-01 江苏大学 一种建立cyp2d1基因敲除大鼠模型的方法
CN105548420B (zh) * 2015-12-30 2019-08-02 上海泽润生物科技有限公司 Mops残留量的检测方法
EP4219689A3 (en) 2015-12-30 2023-12-20 Novartis AG Immune effector cell therapies with enhanced efficacy
KR102808969B1 (ko) 2016-01-11 2025-05-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 키메라 단백질 및 면역요법 방법
SG11201805792PA (en) * 2016-01-11 2018-08-30 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
US10053693B2 (en) 2016-01-19 2018-08-21 Mubin I. Syed Method for controlling obesity using minimally invasive means
US20190264186A1 (en) 2016-01-22 2019-08-29 The Broad Institute Inc. Crystal structure of crispr cpf1
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
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US12122997B2 (en) 2016-02-15 2024-10-22 Temple University—Of the Commonwealth System of Higher Education Excision of retroviral nucleic acid sequences
US10973930B2 (en) 2016-02-18 2021-04-13 The Penn State Research Foundation Generating GABAergic neurons in brains
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
WO2017147196A1 (en) 2016-02-22 2017-08-31 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
US10286073B2 (en) * 2016-02-23 2019-05-14 Ilisa Tech, Inc. Magnetic control of gene delivery in vivo
SG11201807025SA (en) * 2016-02-26 2018-09-27 Lanzatech New Zealand Ltd Crispr/cas systems for c-1 fixing bacteria
EP3520801A1 (en) 2016-03-04 2019-08-07 4D Pharma Plc Compositions comprising bacterial blautia strains for treating visceral hypersensitivity
GB201612191D0 (en) 2016-07-13 2016-08-24 4D Pharma Plc Compositions comprising bacterial strains
EP3423083A4 (en) 2016-03-04 2019-10-02 Indoor Biotechnologies Inc. FEL-D-1 KNOCKOUTS AND ASSOCIATED COMPOSITIONS AND METHODS BASED ON CRISPR-CAS9 GENERALIZATION
EP3429567B1 (en) 2016-03-16 2024-01-10 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
CN109414504B (zh) * 2016-03-29 2022-04-08 无限生物制药公司 药物组合物或食品组合物及评价活性成分体内效果的方法
CN114594268A (zh) 2016-03-29 2022-06-07 无限生物制药公司 用以预防或治疗肾脏疾病的活性组分的候选物质的筛选装置和筛选方法
CN109475646A (zh) * 2016-03-30 2019-03-15 因特利亚治疗公司 用于crispr/cas成分的脂质纳米颗粒制剂
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR102424476B1 (ko) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
EP3445856A1 (en) 2016-04-19 2019-02-27 The Broad Institute Inc. Novel crispr enzymes and systems
JP7259182B2 (ja) 2016-04-25 2023-04-18 プレジデント アンド フェローズ オブ ハーバード カレッジ ｉｎ ｓｉｔｕ分子検出のためのハイブリダイゼーション連鎖反応法
WO2017189542A1 (en) * 2016-04-26 2017-11-02 University Of Georgia Research Foundation, Inc. Plants having reduced methylation of cytosine nucleotides and methods of use
EP3449004B1 (en) 2016-04-29 2021-02-24 Poseida Therapeutics, Inc. Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
WO2017192997A1 (en) 2016-05-05 2017-11-09 Children's Hospital Medical Center Methods for the in vitro manufacture of gastric fundus tissue and compositions related to same
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
NZ747857A (en) * 2016-05-20 2023-01-27 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
US10017760B2 (en) 2016-06-24 2018-07-10 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
CN109863143B (zh) 2016-07-13 2021-10-15 威泰克斯制药公司 提高基因组编辑效率的方法、组合物和试剂盒
TW201821093A (zh) 2016-07-13 2018-06-16 英商４Ｄ製藥有限公司 包含細菌菌株之組合物
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2018021855A1 (ko) * 2016-07-28 2018-02-01 기초과학연구원 Cas9 단백질 및 가이드 RNA를 포함하는 안질환 치료용 약학 조성물
SG11201901305YA (en) 2016-07-29 2019-03-28 Regeneron Pharma Mice comprising mutations resulting in expression of c-truncated fibrillin-1
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
AU2017308473B2 (en) * 2016-08-12 2021-08-19 Toolgen Incorporated Manipulated immunoregulatory element and immunity altered thereby
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
EP3500671B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method of selecting target sequences for the design of guide rnas
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11630103B2 (en) 2016-08-17 2023-04-18 The Broad Institute, Inc. Product and methods useful for modulating and evaluating immune responses
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
KR20230155013A (ko) 2016-08-20 2023-11-09 아벨리노 랩 유에스에이, 인크. 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법
WO2019165322A1 (en) * 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
US20220056440A1 (en) * 2016-08-20 2022-02-24 Tara Moore Crispr gene editing for autosomal dominant diseases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
IL247752A0 (en) 2016-09-11 2016-11-30 Yeda Res & Dev Compositions and methods for modulating gene expression for site-directed mutagenesis
WO2018053053A1 (en) 2016-09-13 2018-03-22 The Broad Institute, Inc. Proximity-dependent biotinylation and uses thereof
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
DK3516056T3 (da) * 2016-09-23 2025-02-17 Dsm Ip Assets Bv Et guide-rna-ekspressionssystem til en værtscelle
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
EA201990860A1 (ru) 2016-09-30 2019-10-31 Рнк-направляемые модифицирующие нуклеиновые кислоты ферменты и способы их применения
KR20230170126A (ko) 2016-09-30 2023-12-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Rna-가이드된 핵산 변형 효소 및 이의 사용 방법
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN110462034A (zh) * 2016-10-07 2019-11-15 综合Dna技术公司 化脓链球菌cas9突变基因和由其编码的多肽
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
KR20190127655A (ko) 2016-10-19 2019-11-13 프로디자인 소닉스, 인크. 음향학에 의한 친화성 세포 추출
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
NZ753051A (en) 2016-11-04 2023-03-31 Children’S Hospital Medical Center Liver organoid compositions and methods of making and using same
WO2018089386A1 (en) 2016-11-11 2018-05-17 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action
CN110248957B (zh) * 2016-11-14 2023-12-19 株式会社图尔金 经人工操纵的sc功能控制系统
KR102807995B1 (ko) 2016-12-05 2025-05-16 칠드런즈 호스피탈 메디칼 센터 결장 유사장기 및 이를 제조 및 사용하는 방법
AU2017371324B2 (en) 2016-12-09 2024-06-13 Massachusetts Institute Of Technology CRISPR effector system based diagnostics
WO2018107103A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr-systems for modifying a trait of interest in a plant
GB201621123D0 (en) 2016-12-12 2017-01-25 4D Pharma Plc Compositions comprising bacterial strains
KR20240172763A (ko) 2016-12-14 2024-12-10 리간달 인코포레이티드 핵산 및/또는 단백질 적재물 전달을 위한 조성물 및 방법
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
KR20230125856A (ko) * 2016-12-23 2023-08-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
CN110214192A (zh) * 2016-12-27 2019-09-06 中外制药株式会社 用于评价测试物质的免疫原性的方法
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
CN106632693B (zh) * 2017-01-19 2021-05-25 上海科技大学 具有多个核定位序列的SpyCas9蛋白及其应用
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11591621B2 (en) 2017-02-07 2023-02-28 Modalis Therapeutics Corporation Method of treating diseases associated with elevated KRAS expression using CRISPR-GNDM system
US20190367924A1 (en) * 2017-02-17 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
GB2574769A (en) * 2017-03-03 2019-12-18 Univ California RNA Targeting of mutations via suppressor tRNAs and deaminases
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
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11739308B2 (en) 2017-03-15 2023-08-29 The Broad Institute, Inc. Cas13b orthologues CRISPR enzymes and systems
US11174515B2 (en) 2017-03-15 2021-11-16 The Broad Institute, Inc. CRISPR effector system based diagnostics
KR20190140918A (ko) 2017-03-15 2019-12-20 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 검출을 위한 crispr 이펙터 시스템 기반 진단
US11104937B2 (en) 2017-03-15 2021-08-31 The Broad Institute, Inc. CRISPR effector system based diagnostics
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
EP3600382A4 (en) * 2017-03-21 2020-12-30 Anthony P. Shuber TREATMENT OF CANCER WITH CAS ENDONUCLEASE COMPLEXES
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
WO2018183921A1 (en) 2017-04-01 2018-10-04 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
US11618928B2 (en) 2017-04-12 2023-04-04 The Broad Institute, Inc. CRISPR effector system based diagnostics for malaria detection
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
EP3610009A1 (en) 2017-04-12 2020-02-19 The Broad Institute, Inc. Novel type vi crispr orthologs and systems
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
JP7248586B2 (ja) 2017-04-14 2023-03-29 チルドレンズ ホスピタル メディカル センター 複数ドナー幹細胞組成物およびそれを作製する方法
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
WO2018195129A1 (en) 2017-04-17 2018-10-25 University Of Maryland, College Park Embryonic cell cultures and methods of using the same
EP3612629A1 (en) 2017-04-18 2020-02-26 The Broad Institute, Inc. Compositions for detecting secretion and methods of use
CN111031790A (zh) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 产生基因修改的动物的方法
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
US11696954B2 (en) * 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
US11072816B2 (en) 2017-05-03 2021-07-27 The Broad Institute, Inc. Single-cell proteomic assay using aptamers
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP7235391B2 (ja) 2017-05-08 2023-03-08 ツールゲン インコーポレイテッド 人工的に操作された免疫細胞
US20210278416A1 (en) 2017-05-09 2021-09-09 The Broad Institute, Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209324A2 (en) 2017-05-11 2018-11-15 The Broad Institute, Inc. Methods and compositions of use of cd8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
JP7368235B2 (ja) * 2017-05-12 2023-10-24 プレジデント アンド フェローズ オブ ハーバード カレッジ ユニバーサル早期がん診断
WO2018213459A1 (en) * 2017-05-16 2018-11-22 Wisconsin Alumni Research Foundation System and method for en masse patterning of molecule structures
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3063739A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3064171A1 (en) 2017-05-22 2018-11-29 4D Pharma Research Limited Compositions comprising bacterial strains
EP3630942B1 (en) 2017-05-24 2022-11-30 4D Pharma Research Limited Compositions comprising bacterial strain
CA3063449A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
EP3635113A4 (en) 2017-06-05 2021-03-17 Fred Hutchinson Cancer Research Center SAFE GENOMIC HAVERS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND NANOPARTICLES MODIFIED TO ENABLE TARGETED GENETIC THERAPIES
US10428319B2 (en) 2017-06-09 2019-10-01 Editas Medicine, Inc. Engineered Cas9 nucleases
JP2020523326A (ja) 2017-06-14 2020-08-06 フォーディー ファーマ リサーチ リミテッド４Ｄ Ｐｈａｒｍａ Ｒｅｓｅａｒｃｈ Ｌｉｍｉｔｅｄ 細菌株を含む組成物
JP6884889B2 (ja) 2017-06-14 2021-06-09 フォーディー ファーマ リサーチ リミテッド４Ｄ Ｐｈａｒｍａ Ｒｅｓｅａｒｃｈ Ｌｉｍｉｔｅｄ 細菌株を含む組成物
US11897953B2 (en) 2017-06-14 2024-02-13 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
SMT202100069T1 (it) 2017-06-14 2021-03-15 4D Pharma Res Limited Composizioni comprendenti ceppi batterici
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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
MX2019015413A (es) 2017-06-23 2020-07-20 Univ Kentucky Res Found Metodo.
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
WO2019006107A1 (en) * 2017-06-28 2019-01-03 University Of South Florida MODIFIED UBE3A GENE FOR GENE THERAPY APPROACH TO ANGELMAN'S SYNDROME
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US12415000B2 (en) 2017-07-07 2025-09-16 The Broad Institute, Inc. CRISPR system based antiviral therapy
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US12049643B2 (en) 2017-07-14 2024-07-30 The Broad Institute, Inc. Methods and compositions for modulating cytotoxic lymphocyte activity
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
US12239646B2 (en) 2017-07-19 2025-03-04 Dana-Farber Cancer Institute, Inc. Cancer diagnostic and treatment
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019028023A2 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR EVALUATING CRISPR / CAS MEDIATED DISRUPTION OR EXCISION AND CRISPR / CAS INDUCED RECOMBINATION USING IN VIVO EXOGENIC DONOR NUCLEIC ACID
BR112020001364A2 (pt) * 2017-07-31 2020-08-11 Regeneron Pharmaceuticals, Inc. métodos para testar e modificar a capacidade de uma crispr/cas nuclease.
US20190032156A1 (en) 2017-07-31 2019-01-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
BR102017016440A2 (pt) 2017-07-31 2019-03-19 Universidade Federal Do Rio Grande Do Sul Composição para terapia gênica do sistema nervoso central, processo de obtenção e uso da mesma
JP7581046B2 (ja) 2017-08-23 2024-11-12 ザ ジェネラル ホスピタル コーポレイション 変更されたＰＡＭ特異性を有する遺伝子操作されたＣＲＩＳＰＲ－Ｃａｓ９ヌクレアーゼ
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
US20190083457A1 (en) * 2017-09-15 2019-03-21 The Borad Of Trustees Of The Leland Stanford Junior University Mitochondrial aldehyde dehydrogenase-2 modulators for protecting, expanding and increasing the potency of hematopoietic stem cells
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
CN118006607A (zh) 2017-09-22 2024-05-10 马萨诸塞大学 Sod1双表达载体及其用途
AU2018341349A1 (en) 2017-09-27 2020-04-09 E. I. Du Pont De Nemours And Company Soil application of crop protection agents
US12295978B2 (en) * 2017-09-28 2025-05-13 Shiftbio Inc. Recombinant plasma membrane-based vesicle, for treating cancer
EP4276185A3 (en) 2017-09-29 2024-02-21 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
WO2019070755A1 (en) 2017-10-02 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER
WO2019071048A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
CA3076518A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. Crispr effector system based diagnostics
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
EP3694603A4 (en) 2017-10-10 2021-07-14 Children's Hospital Medical Center ESOPHAGUS TISSUE AND / OR ORGANOID COMPOSITIONS AND METHOD FOR MANUFACTURING THEREOF
US11725228B2 (en) 2017-10-11 2023-08-15 The General Hospital Corporation Methods for detecting site-specific and spurious genomic deamination induced by base editing technologies
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
BR112020007823A2 (pt) 2017-10-20 2020-10-27 Fred Hutchinson Cancer Research Center sistemas e métodos para produzir células b geneticamente modificadas para expressar anticorpos selecionados
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
US12227753B2 (en) 2017-11-01 2025-02-18 The Regents Of The University Of California CasY compositions and methods of use
WO2019090173A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
US12221720B2 (en) 2017-11-13 2025-02-11 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
CN111448313A (zh) 2017-11-16 2020-07-24 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
US11332736B2 (en) 2017-12-07 2022-05-17 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
US12458411B2 (en) 2017-12-07 2025-11-04 Augmedics Ltd. Spinous process clamp
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． 音響トランスデューサドライバ及びコントローラ
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US12379372B2 (en) 2017-12-21 2025-08-05 Children's Hospital Medical Center Digitalized human organoids and methods of using same
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
AU2019207409B2 (en) 2018-01-12 2023-02-23 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
MA51619A (fr) 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
EP3740483B1 (en) 2018-01-17 2023-01-11 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
FI3746568T3 (fi) 2018-01-29 2023-12-12 Broad Inst Inc Crispr-efektorijärjestelmään perustuva diagnostiikka
KR20250099758A (ko) 2018-01-31 2025-07-02 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
US20210032300A1 (en) * 2018-02-02 2021-02-04 The Penn State Research Foundation Methods and materials for treating brain injuries
WO2019157304A1 (en) * 2018-02-09 2019-08-15 Empirico Inc. Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions
CN112020554B (zh) 2018-02-23 2024-10-22 先锋国际良种公司 新颖cas9直系同源物
ES2953541T3 (es) 2018-03-14 2023-11-14 Arbor Biotechnologies Inc Sistemas y enzimas novedosos de direccionamiento a ADN y ARN de CRISPR
KR101889683B1 (ko) * 2018-03-14 2018-08-17 유병탁 철골 구조 시공 방법
CN112041444A (zh) 2018-03-14 2020-12-04 阿伯生物技术公司 新型crispr dna靶向酶及系统
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
JP7558563B2 (ja) 2018-03-15 2024-10-01 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
WO2019183123A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
WO2019204378A1 (en) 2018-04-17 2019-10-24 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
US12338486B2 (en) 2018-04-19 2025-06-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded DNA
SI3560330T1 (sl) 2018-04-24 2022-08-31 KWS SAAT SE & Co. KGaA Rastline z izboljšano prebavljivostjo in markerskimi haplotipi
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
US12281301B2 (en) 2018-04-27 2025-04-22 The Broad Institute, Inc. Sequencing-based proteomics
CA3097914A1 (en) * 2018-04-30 2019-11-07 Oregon Health & Science University Methods of gene therapy
EP3787543A4 (en) 2018-05-02 2022-01-19 Augmedics Ltd. REGISTRATION OF A FIDUCIAL MARKER FOR AN AUGMENTED REALITY SYSTEM
JP7498499B2 (ja) * 2018-05-03 2024-06-12 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 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復
US20210386829A1 (en) 2018-05-04 2021-12-16 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
CA3100019A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108707628B (zh) * 2018-05-28 2021-11-23 上海海洋大学 斑马鱼notch2基因突变体的制备方法
WO2019232542A2 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
CN112543812A (zh) 2018-06-26 2021-03-23 麻省理工学院 基于crispr效应系统的扩增方法、系统和诊断
BR112020026246A2 (pt) 2018-06-26 2021-04-20 The Broad Institute Inc. composições, sistemas e métodos de amplificação com base em nickase dupla crispr
KR20210024009A (ko) 2018-06-26 2021-03-04 더 브로드 인스티튜트, 인코퍼레이티드 Crispr/cas 및 트랜스포사제 기반 증폭 조성물, 시스템 및 방법
US11629179B2 (en) 2018-06-29 2023-04-18 Stichting Het Nederlands Kanker Instituut—Antoni van Leeuwenhoek Ziekenhuis TWEAK-receptor agonists for use in combination with immunotherapy of a cancer
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CN112543810A (zh) 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
CA3105658A1 (en) 2018-07-13 2020-01-16 The Regents Of The University Of California Retrotransposon-based delivery vehicle and methods of use thereof
US20210284981A1 (en) * 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
SG11202100533VA (en) 2018-07-26 2021-02-25 Childrens Hospital Med Ct Hepato-biliary-pancreatic tissues and methods of making same
KR20210053898A (ko) 2018-07-31 2021-05-12 더 브로드 인스티튜트, 인코퍼레이티드 신규 crispr 효소 및 시스템
GB201812603D0 (en) 2018-08-02 2018-09-19 British American Tobacco Investments Ltd Method
EP3833761A1 (en) 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems
KR102103103B1 (ko) 2018-08-16 2020-04-21 (주)라트바이오 인위적 뉴클레아제를 생산하는 형질전환 동물 및 형질전환 배아
SG11202101576UA (en) 2018-08-18 2021-03-30 Harvard College In situ gene editing
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020041384A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. 3-phenyl-2-cyano-azetidine derivatives, inhibitors of rna-guided nuclease activity
WO2020041387A1 (en) 2018-08-20 2020-02-27 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
WO2020068304A2 (en) 2018-08-20 2020-04-02 The Broad Institute, Inc. Inhibitors of rna-guided nuclease target binding and uses thereof
WO2020041456A1 (en) 2018-08-22 2020-02-27 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
EP3847650A1 (en) 2018-09-06 2021-07-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP4268831A3 (en) 2018-09-12 2024-05-22 Fred Hutchinson Cancer Center Reducing cd33 expression to selectively protect therapeutic cells
SG11202102431YA (en) 2018-09-12 2021-04-29 Childrens Hospital Med Ct Organoid compositions for the production of hematopoietic stem cells and derivatives thereof
EP3852813A4 (en) 2018-09-18 2022-06-22 VNV Newco Inc. ARC-BASED CAPSIDS AND THEIR USES
WO2020072816A1 (en) 2018-10-03 2020-04-09 The Broad Institute, Inc. Crispr effector system based diagnostics for hemorrhagic fever detection
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US20220401460A1 (en) 2018-10-10 2022-12-22 Dana-Farber Cancer Institute, Inc. Modulating resistance to bcl-2 inhibitors
WO2020077236A1 (en) 2018-10-12 2020-04-16 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
CN109295081B (zh) * 2018-10-17 2020-06-12 中国人民解放军第四军医大学 一种LDLR-Lamp2b融合基因、表达载体及其应用
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US20210388389A1 (en) 2018-10-30 2021-12-16 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
CA3112839A1 (en) 2018-11-01 2020-05-07 E. I. Du Pont De Nemours And Company Systems and methods for manufacture of controlled release of agricultural cores
GB201817971D0 (en) 2018-11-02 2018-12-19 British American Tobacco Investments Ltd Method
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
AU2019374883A1 (en) 2018-11-09 2021-05-20 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
CN113881706B (zh) * 2018-11-13 2024-12-13 四川横竖生物科技股份有限公司 基于hNPY的基因过表达嵌合动物模型、工程猴模型及其应用
MX2021005701A (es) 2018-11-14 2021-09-23 Broad Inst Inc Sistema crispr a base de sistemas y métodos de diagnóstico de gotas.
CN113302312A (zh) 2018-11-14 2021-08-24 博德研究所 使用sherlock检测方法对高度进化的病毒变体进行多重化
GB201818715D0 (en) 2018-11-16 2019-01-02 British American Tobacco Investments Ltd Method
US11766296B2 (en) 2018-11-26 2023-09-26 Augmedics Ltd. Tracking system for image-guided surgery
EP3891276A4 (en) * 2018-12-05 2022-07-27 Fred Hutchinson Cancer Center PRODUCTION OF GENETICALLY MODIFIED CELLS WITH REDUCED AND MINIMAL MANIPULATION
US12258594B2 (en) 2018-12-05 2025-03-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
US20250043366A1 (en) 2018-12-13 2025-02-06 The Broad Institute, Inc. Tiled assays using crispr-cas based detection
CA3117228A1 (en) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
CA3120799A1 (en) 2018-12-20 2020-06-25 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US20220064635A1 (en) * 2019-01-07 2022-03-03 Emendobio Inc. Crispr compositions and methods for promoting gene editing of adenosine deaminase 2 (ada2)
EP3911632A1 (en) 2019-01-15 2021-11-24 Empirico Inc. Prodrugs of alox-15 inhibitors and methods of using the same
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB201900940D0 (en) 2019-01-23 2019-03-13 British American Tobacco Investments Ltd Method
US20220119871A1 (en) 2019-01-28 2022-04-21 The Broad Institute, Inc. In-situ spatial transcriptomics
US11965159B2 (en) 2019-01-29 2024-04-23 The Broad Institute, Inc. Compositions and methods for regulating proteins and nucleic acids activities
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020163856A1 (en) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Modified mitochondrion and methods of use thereof
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2020168075A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
WO2020172343A2 (en) 2019-02-19 2020-08-27 Massachusetts Institute Of Technology Methods for treating injuries
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2020181101A1 (en) 2019-03-07 2020-09-10 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
US20220154282A1 (en) 2019-03-12 2022-05-19 The Broad Institute, Inc. Detection means, compositions and methods for modulating synovial sarcoma cells
EP3938499A1 (en) 2019-03-12 2022-01-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Cas9 variants with enhanced specificity
US12419915B2 (en) 2019-03-13 2025-09-23 The Broad Institute, Inc. Microglial progenitors for regeneration of functional microglia in the central nervous system and therapeutics uses thereof
WO2020186235A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate intestinal innate lymphoid cells
US20220154258A1 (en) 2019-03-14 2022-05-19 The Broad Institute, Inc. Crispr effector system based multiplex diagnostics
WO2020186223A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Sherlock assays for tick-borne diseases
US20220154222A1 (en) 2019-03-14 2022-05-19 The Broad Institute, Inc. Novel nucleic acid modifiers
CA3127814C (en) 2019-03-18 2024-05-14 Regeneron Pharmaceuticals, Inc. Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
EP3942023A1 (en) 2019-03-18 2022-01-26 The Broad Institute, Inc. Compositions and methods for modulating metabolic regulators of t cell pathogenicity
US20220152148A1 (en) 2019-03-18 2022-05-19 The Broad Institute, Inc. Modulation of type 2 immunity by targeting clec-2 signaling
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
KR102695765B1 (ko) 2019-03-18 2024-08-20 리제너론 파마슈티칼스 인코포레이티드 타우 시딩 또는 응집의 유전적 변형제를 식별하기 위한 CRISPR/Cas 스크리닝 플랫폼
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
CN111733225A (zh) * 2019-03-25 2020-10-02 北京大学第一医院 一种可用于辅助氯吡格雷精准用药的prkg1基因snp标志物
EP3947687A1 (en) 2019-03-29 2022-02-09 Massachusetts Institute of Technology Constructs for continuous monitoring of live cells
WO2020206046A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Methods and compositions for cell therapy
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
BR112021019512A2 (pt) 2019-04-03 2022-02-15 Regeneron Pharma Métodos para inserir uma sequência de codificação de proteína de ligação ao antígeno e para tratar ou efetuar a profilaxia de uma doença em um animal, animal, célula, genoma, ácido nucleico doador exógeno, gene de porto seguro, e, agente de nuclease ou um ou mais ácidos nucleicos
IL286905B2 (en) 2019-04-04 2024-06-01 Regeneron Pharma Non-human animals containing the human coagulation factor 12 locus
CA3133359C (en) 2019-04-04 2023-04-11 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220213469A1 (en) 2019-04-30 2022-07-07 The Broad Institute, Inc. Methods and compositions for barcoding nucleic acid libraries and cell populations
BR112021021979A2 (pt) * 2019-05-03 2022-12-06 Specific Biologics Inc Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas
WO2020231702A1 (en) * 2019-05-11 2020-11-19 Youngsuk Yi Compositions and methods containing exosomes
CN114096684B (zh) 2019-05-13 2024-11-19 科沃施种子欧洲股份两合公司 玉米的耐旱性
WO2020232271A1 (en) 2019-05-14 2020-11-19 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
GB201906768D0 (en) 2019-05-14 2019-06-26 British American Tobacco Investments Ltd Method
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
CN114555813A (zh) * 2019-05-21 2022-05-27 国家医疗保健研究所 治疗雷特综合征的胆固醇24-水解酶的表达载体
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
WO2020243371A1 (en) 2019-05-28 2020-12-03 Massachusetts Institute Of Technology Methods and compositions for modulating immune responses
WO2020243661A1 (en) 2019-05-31 2020-12-03 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CA3141807A1 (en) 2019-05-31 2020-12-03 Children's Hospital Medical Center Methods of generating and expanding hematopoietic stem cells
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
US12188045B2 (en) 2019-06-07 2025-01-07 KSQ Therapeutics, Inc. Guide RNA combinations and methods of use
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
ES3034102T3 (en) 2019-06-14 2025-08-13 Regeneron Pharma Models of tauopathy
WO2020257514A1 (en) * 2019-06-21 2020-12-24 Children's Medical Center Corporation Methods and compositions for allele specific gene editing
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
GB201909563D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
GB201909562D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
US12178666B2 (en) 2019-07-29 2024-12-31 Augmedics Ltd. Fiducial marker
US11980506B2 (en) 2019-07-29 2024-05-14 Augmedics Ltd. Fiducial marker
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021030627A1 (en) 2019-08-13 2021-02-18 The General Hospital Corporation Methods for predicting outcomes of checkpoint inhibition and treatment thereof
US12421557B2 (en) 2019-08-16 2025-09-23 The Broad Institute, Inc. Methods for predicting outcomes and treating colorectal cancer using a cell atlas
EP4488287A3 (en) 2019-08-21 2025-03-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
US20220333208A1 (en) 2019-09-03 2022-10-20 The Broad Institute, Inc. Crispr effector system based multiplex cancer diagnostics
US20220325296A1 (en) 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
JP2022548031A (ja) 2019-09-13 2022-11-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 脂質ナノ粒子によって送達されるｃｒｉｓｐｒ／ｃａｓシステムを使用する動物における転写調節
US12264367B2 (en) 2019-09-19 2025-04-01 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
US20220389451A1 (en) 2019-09-20 2022-12-08 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
AU2020348879A1 (en) 2019-09-20 2022-04-14 Massachusetts Institute Of Technology Novel type VI CRISPR enzymes and systems
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US12195725B2 (en) 2019-10-03 2025-01-14 Dana-Farber Cancer Institute, Inc. Compositions and methods for modulating and detecting tissue specific TH17 cell pathogenicity
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US11793787B2 (en) 2019-10-07 2023-10-24 The Broad Institute, Inc. Methods and compositions for enhancing anti-tumor immunity by targeting steroidogenesis
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021077000A1 (en) 2019-10-16 2021-04-22 The Broad Institute, Inc. Engineered muscle targeting compositions
AU2020366566A1 (en) 2019-10-17 2022-04-21 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
US11844800B2 (en) 2019-10-30 2023-12-19 Massachusetts Institute Of Technology Methods and compositions for predicting and preventing relapse of acute lymphoblastic leukemia
AU2020379046B2 (en) 2019-11-08 2025-03-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy
US12195723B2 (en) 2019-11-08 2025-01-14 The Broad Institute, Inc. Engineered antigen presenting cells and uses thereof
CN110684801A (zh) * 2019-11-13 2020-01-14 武汉华美生物工程有限公司 转导cas9基因到哺乳动物细胞进行基因编辑的方法
EP4061338A1 (en) 2019-11-22 2022-09-28 President And Fellows Of Harvard College Ionic liquids for drug delivery
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
US12241830B2 (en) 2019-12-06 2025-03-04 Broad Institute, Inc. Living biosensors
EP3835309A1 (en) 2019-12-13 2021-06-16 KWS SAAT SE & Co. KGaA Method for increasing cold or frost tolerance in a plant
US11382712B2 (en) 2019-12-22 2022-07-12 Augmedics Ltd. Mirroring in image guided surgery
IL292896A (en) * 2019-12-23 2022-07-01 Univ California Crispr-cas effector polypeptides and methods of use thereof
US20230340537A1 (en) 2020-01-17 2023-10-26 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
KR20220130158A (ko) 2020-01-23 2022-09-26 더 칠드런스 메디칼 센터 코포레이션 인간 만능 줄기 세포로부터의 무-간질 t 세포 분화
US12165747B2 (en) 2020-01-23 2024-12-10 The Broad Institute, Inc. Molecular spatial mapping of metastatic tumor microenvironment
WO2021151073A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
EP4093863A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation CRISPR-CAS ENZYMES WITH ENHANCED ON-TARGET ACTIVITY
CN113281130B (zh) * 2020-02-20 2024-08-23 中国科学院上海高等研究院 一种用于大尺寸脑组织的高效的高尔基染色方法
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
MX2022010666A (es) 2020-02-28 2023-01-30 Huigene Therapeutics Co Ltd Sistema crispr-cas de tipo vi-e y de tipo vi-f y usos del mismo.
US11965161B2 (en) 2020-03-04 2024-04-23 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
CN111321150B (zh) * 2020-03-13 2020-12-04 广西壮族自治区水产科学研究院 一种LvCTL4基因及编码的蛋白质、蛋白质的获取方法、表达载体、重组菌和应用
WO2021188734A1 (en) 2020-03-17 2021-09-23 The Broad Institute, Inc. Crispr system high throughput diagnostic systems and methods
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US11453907B2 (en) 2020-03-23 2022-09-27 The Broad Institute, Inc. Crispr effector system based coronavirus diagnostics
MX2022012189A (es) 2020-03-31 2023-01-05 Arbor Biotechnologies Inc Composiciones que comprenden una variante del polipéptido cas12i2 y usos de las mismas.
US12057197B2 (en) 2020-04-03 2024-08-06 Creyon Bio, Inc. Oligonucleotide-based machine learning
KR20220165764A (ko) 2020-04-09 2022-12-15 아아르. 제이. 레날드즈 토바코 캄파니 니코티아나 타바쿰에서 니코틴 수준을 조절하는 방법
US20230121577A1 (en) * 2020-04-15 2023-04-20 The Regents Of The University Of California Compositions and Methods for Treatment
US20230165909A1 (en) 2020-04-21 2023-06-01 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
WO2021216622A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
EP4149622A4 (en) * 2020-05-12 2024-12-18 Myogene Bio LLC IMMUNOSUPPRESSIVE AGENTS AND METHODS OF RE-DOSING VIRAL ADMINISTRATION FOR GENE THERAPY
CN111521826A (zh) * 2020-05-21 2020-08-11 江海松 外周血外泌体中Notch3蛋白作为分子标记的应用及一种检测试剂盒
EP4176434A4 (en) * 2020-05-29 2024-09-11 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)
US20230279418A1 (en) 2020-05-29 2023-09-07 KWS SAAT SE & Co. KGaA Plant haploid induction
US11389252B2 (en) 2020-06-15 2022-07-19 Augmedics Ltd. Rotating marker for image guided surgery
KR102687692B1 (ko) * 2020-07-08 2024-07-24 경상국립대학교산학협력단 미세상동성 기반 말단 결합을 통한 유전자 교정에 이용되는 공여자 핵산 및 이의 용도
WO2022011356A2 (en) * 2020-07-10 2022-01-13 President And Fellows Of Harvard College Aav vector delivery systems
CN111979241B (zh) * 2020-07-23 2021-10-01 上海市第一人民医院 制备视网膜色素变性非人哺乳动物模型的方法
WO2022018667A1 (en) 2020-07-24 2022-01-27 Pfizer Inc. Combination therapies using cdk2 and cdc25a inhibitors
CN116194138A (zh) 2020-08-07 2023-05-30 博德研究所 治疗性靶向癌症中经由xpr1:kidins220蛋白质复合物的磷酸盐调节异常
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
US12239385B2 (en) 2020-09-09 2025-03-04 Augmedics Ltd. Universal tool adapter
JP2023541418A (ja) 2020-09-10 2023-10-02 モンサント テクノロジー エルエルシー 減数分裂及び生殖系列プロモーターを使用する遺伝子編集及び部位特異的組込み事象の増加
CN112159809B (zh) * 2020-09-22 2021-06-22 广州瑞风生物科技有限公司 靶向CTGF基因的gRNA及其应用
WO2022068912A1 (en) 2020-09-30 2022-04-07 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
WO2022188039A1 (en) 2021-03-09 2022-09-15 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
HUE071824T2 (hu) 2020-10-15 2025-09-28 Hoffmann La Roche Nukleinsav-konstruktumok VA RNS transzkripcióhoz
RU2749307C1 (ru) * 2020-10-30 2021-06-08 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) Новая компактная нуклеаза CAS9 II типа из Anoxybacillus flavithermus
EP4001429A1 (en) 2020-11-16 2022-05-25 Antibodies-Online GmbH Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq)
US20240002839A1 (en) 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
CN114796493A (zh) * 2021-01-28 2022-07-29 中国科学技术大学 血管源性bace1作为脑小血管损伤相关疾病的治疗靶点的用途
AU2022220332A1 (en) 2021-02-11 2023-08-24 Arbor Biotechnologies, Inc. Compositions comprising a variant cas12i4 polypeptide and uses thereof
CN112997966A (zh) * 2021-03-08 2021-06-22 国家卫生健康委科学技术研究所 一种基于CRISPR/Cas9技术敲入miRNA-125a的小鼠模型及构建方法
CN115427561B (zh) 2021-03-09 2024-06-04 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
WO2022204543A1 (en) * 2021-03-25 2022-09-29 The Regents Of The University Of California Methods and materials for treating huntington's disease
US20240247285A1 (en) 2021-05-10 2024-07-25 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
CN113180015A (zh) * 2021-05-25 2021-07-30 四川省农业机械研究设计院 一种蚕房及三段式流水线养蚕方法
JP2024522112A (ja) 2021-05-27 2024-06-11 中国科学院動物研究所 エンジニアリングされたＣａｓ１２ｉヌクレアーゼ、エフェクタータンパク質及びその用途
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4347826A1 (en) 2021-06-02 2024-04-10 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4355351A1 (en) * 2021-06-18 2024-04-24 Innovative Cellular Therapeutics Holdings, Ltd. Modified cells targeting tumor ecm and therapy thereof
US11896445B2 (en) 2021-07-07 2024-02-13 Augmedics Ltd. Iliac pin and adapter
WO2023004367A2 (en) 2021-07-20 2023-01-26 The Broad Institute, Inc. Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
WO2023004422A2 (en) 2021-07-22 2023-01-26 Arbor Biotechnologies, Inc. Crispr-associated transposon systems and methods of using same
US12150821B2 (en) 2021-07-29 2024-11-26 Augmedics Ltd. Rotating marker and adapter for image-guided surgery
US20250179591A1 (en) 2021-07-30 2025-06-05 KWS SAAT SE & Co. KGaA Plants with improved digestibility and marker haplotypes
CN113607963B (zh) * 2021-08-11 2024-05-24 河南省人民医院 一种预测视网膜衰老进程的蛋白标志物、筛选方法及其应用
EP4384608A1 (en) 2021-08-12 2024-06-19 Arbor Biotechnologies, Inc. Compositions comprising a variant cas12i3 polypeptide and uses thereof
CN113373178A (zh) * 2021-08-13 2021-09-10 上海南方模式生物科技股份有限公司 一种tlr8基因人源化动物模型的构建方法及应用
WO2023021450A1 (en) 2021-08-18 2023-02-23 Augmedics Ltd. Stereoscopic display and digital loupe for augmented-reality near-eye display
JP2024534945A (ja) 2021-09-10 2024-09-26 アジレント・テクノロジーズ・インク 化学修飾を有するプライム編集のためのガイドｒｎａ
EP4596697A3 (en) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions and methods for nucleic acid modifications
CN118382429A (zh) 2021-10-08 2024-07-23 哈佛大学校长及研究员协会 用于药物递送的离子液体
CN113913428A (zh) * 2021-10-14 2022-01-11 南通大学 靶向Tfap2a基因的小干扰RNA及其在制备治疗慢性疼痛药物方面的应用
EP4405463A1 (en) 2021-10-14 2024-07-31 Lonza Sales AG Modified producer cells for extracellular vesicle production
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
US20240415980A1 (en) 2021-10-28 2024-12-19 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
CN119120424A (zh) 2021-11-02 2024-12-13 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023093862A1 (en) 2021-11-26 2023-06-01 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
WO2023108047A1 (en) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
CN114107231B (zh) * 2021-12-13 2023-08-18 重庆大学 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用
GB202118058D0 (en) 2021-12-14 2022-01-26 Univ Warwick Methods to increase yields in crops
CN115843318B (zh) * 2021-12-14 2023-07-18 中国医学科学院药用植物研究所 基于全基因组分析与基因组编辑的植物物种鉴定方法与应用
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
US20250059239A1 (en) 2021-12-17 2025-02-20 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
IL313765A (en) 2021-12-22 2024-08-01 Tome Biosciences Inc Joint provision of a gene editor structure and a donor template
KR20240128067A (ko) 2021-12-29 2024-08-23 브리스톨-마이어스 스큅 컴퍼니 랜딩 패드 세포주의 생성
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023150518A1 (en) 2022-02-01 2023-08-10 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
CA3248748A1 (en) 2022-02-02 2023-08-10 Inscripta, Inc. NUCLEIC ACID-GUIDED NICKASE FUSION PROTEINS
WO2023150623A2 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
AU2023214690A1 (en) 2022-02-03 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
AU2023215810A1 (en) 2022-02-03 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
MX2024009605A (es) 2022-02-04 2024-08-14 Nicoventures Trading Ltd Metodo para modular el contenido de alcaloides en plantas de tabaco.
EP4487875A4 (en) 2022-02-28 2025-07-30 National Univ Corporation Tokai National Higher Education And Research System MEDICINE FOR A DISEASE CAUSED BY A FRAME-SHIFT MUTATION
CN114480497B (zh) * 2022-02-28 2023-09-15 湖南师范大学 一种ep400基因敲除斑马鱼心力衰竭模型的构建及其应用的方法
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
GB202205148D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205561D0 (en) 2022-04-14 2022-06-01 Nicoventures Trading Ltd Method
GB202205562D0 (en) 2022-04-14 2022-06-01 Nicoventures Trading Ltd Method
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023203521A1 (en) 2022-04-21 2023-10-26 Augmedics Ltd. Systems and methods for medical image visualization
GB202206107D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
GB202206109D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
JP2025514304A (ja) 2022-04-29 2025-05-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
CA3256953A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
WO2023225564A1 (en) 2022-05-18 2023-11-23 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
CA3253951A1 (en) 2022-05-19 2023-11-23 Lyell Immunopharma, Inc. NR4A3 Targeted Polynucleotides and Their Uses
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
CN115053861A (zh) * 2022-06-30 2022-09-16 南方医科大学南方医院 一种基于免疫激活的精神分裂症动物模型的构建方法和应用
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
CN120225541A (zh) 2022-07-14 2025-06-27 博德研究所 通过与转铁蛋白受体的相互作用实现cns范围的基因递送的aav衣壳
CN120344660A (zh) 2022-07-18 2025-07-18 雷纳嘉德医疗管理公司 基因编辑组分、系统和使用方法
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024020557A1 (en) 2022-07-22 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising a variant nuclease and uses thereof
WO2024020567A1 (en) 2022-07-22 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising a variant polypeptide and uses thereof
CN115109799A (zh) * 2022-07-26 2022-09-27 华中科技大学同济医学院附属协和医院 一种小分子肽的融合质粒及其在抗肿瘤药物中的应用
EP4561636A1 (en) 2022-07-29 2025-06-04 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
EP4565603A1 (en) 2022-08-05 2025-06-11 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43
CN115247149B (zh) * 2022-08-22 2023-06-16 华域生物科技(天津)有限公司 适用于nk细胞的培养基组合物及培养方法
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4577660A1 (en) 2022-08-26 2025-07-02 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
JP2025531829A (ja) 2022-09-13 2025-09-25 オーグメディックス リミテッド 画像誘導医療介入のための拡張現実アイウェア
EP4590688A1 (en) 2022-09-21 2025-07-30 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024124044A1 (en) 2022-12-07 2024-06-13 The Brigham And Women’S Hospital, Inc. Compositions and methods targeting sat1 for enhancing anti¬ tumor immunity during tumor progression
EP4638766A1 (en) 2022-12-20 2025-10-29 NanoCell Therapeutics Holdings B.V. Integrating and self-inactivating viral vectors and constructs and uses thereof
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024155597A1 (en) 2023-01-17 2024-07-25 Inscripta, Inc. Methods and compositions of co-expression of t7rna polymerase and inhibitory rna aptamers
WO2024155830A2 (en) * 2023-01-18 2024-07-25 Fred Hutchinson Cancer Center Viral particle producer cells with landing pad-integrated viral vectors
GB202300905D0 (en) 2023-01-20 2023-03-08 Nicoventures Trading Ltd Method
EP4654982A1 (en) 2023-01-27 2025-12-03 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof
WO2024163842A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
GB202303077D0 (en) 2023-03-02 2023-04-19 Univ Oslo Brassica plants with improved seed retention
WO2024192141A1 (en) 2023-03-13 2024-09-19 Dana-Farber Cancer Institute, Inc. Treatment of cancers having a drug-resistant mesenchymal cell state
KR20250163949A (ko) * 2023-03-20 2025-11-21 버브 테라퓨틱스, 인크. 심혈관 질환의 치료를 위한 lpa 유전자의 생체내 닉카제-기반 편집
WO2024197185A1 (en) 2023-03-21 2024-09-26 The Broad Institute, Inc. Methods and compositions for dissecting organelle physiology
CN121013904A (zh) 2023-03-30 2025-11-25 儿童医院医学中心 临床级类器官
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
TW202449159A (zh) 2023-04-19 2024-12-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於產生文庫之組合物及方法
WO2024226838A2 (en) 2023-04-25 2024-10-31 The Brigham And Women's Hospital, Inc. Treatment of autoimmune diseases having a pathogenic t cell state
WO2024227131A1 (en) * 2023-04-27 2024-10-31 Rensselaer Polytechnic Institute Recombinant enzyme for the accurate insertion of dna sequences in eukaryotic cells
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024238817A2 (en) * 2023-05-16 2024-11-21 Fallon Jim Deceased Systems and methods for an intranasal drug delivery system
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2024238977A2 (en) 2023-05-18 2024-11-21 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
AU2024274792A1 (en) 2023-05-23 2025-10-23 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2024259309A1 (en) 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders
WO2024263961A2 (en) 2023-06-23 2024-12-26 Children's Hospital Medical Center Methods of matrix-free suspension culture
WO2025006419A1 (en) 2023-06-26 2025-01-02 Flagship Pioneering Innovations Vii, Llc Engineered plasmodia and related methods
AU2024309884A1 (en) 2023-06-30 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
AR133385A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Inserción anti-tfr:gaa y anti-cd63:gaa para el tratamiento de la enfermedad de pompe
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025049788A1 (en) 2023-08-29 2025-03-06 The Broad Institute, Inc. Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025054202A1 (en) 2023-09-05 2025-03-13 Sana Biotechnology, Inc. Method of screening a sample comprising a transgene with a unique barcode
WO2025059533A1 (en) 2023-09-13 2025-03-20 The Broad Institute, Inc. Crispr enzymes and systems
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
WO2025064591A1 (en) * 2023-09-19 2025-03-27 The Regents Of The University Of California In vivo gene editing of tau locus via liponanoparticle delivery
GB202314578D0 (en) 2023-09-22 2023-11-08 Univ Manchester Methods of producing homoplasmic modified plants or parts thereof
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025072803A1 (en) 2023-09-29 2025-04-03 Children's Hospital Medical Center Ntrk2 signaling-mediated alveolar capillary injury and repair
WO2025075911A1 (en) 2023-10-02 2025-04-10 Inscripta, Inc. Engineered gpp synthases
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025085423A1 (en) 2023-10-19 2025-04-24 The Broad Institute, Inc. Method of multi-locus crispri targeting with a single truncated guide
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025096916A1 (en) 2023-11-03 2025-05-08 The Broad Institute, Inc. Multi-site editing in living cells
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025151838A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
WO2025166152A1 (en) 2024-02-02 2025-08-07 The Broad Institute, Inc. Multiplexed perturbation and decoding in pooled genetic screens
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025212920A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Multi-zonal liver organoids
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217202A1 (en) 2024-04-08 2025-10-16 Children's Hospital Medical Center Bile duct organoid
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
CN118376795B (zh) * 2024-05-10 2025-10-31 厦门大学 尿液中n-糖基化蛋白组合作为靶标在早期诊断阿尔茨海默病中的应用
WO2025244671A1 (en) 2024-05-22 2025-11-27 The Scripps Research Institute Compositions for use in treating haploinsufficiency diseases
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025259669A1 (en) 2024-06-10 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and systems for characterizing modified oligonucleotides

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004519245A (ja) * 2001-03-16 2004-07-02 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
WO2011011767A1 (en) * 2009-07-24 2011-01-27 Sigma-Aldrich Co. Method for genome editing
WO2011064736A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
WO2011146121A1 (en) * 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
JP2012510812A (ja) * 2008-12-04 2012-05-17 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014089290A1 (en) * 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation

Family Cites Families (254)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US303910A (en) 1884-08-19 Enbaker
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
DE122007000007I1 (de) 1986-04-09 2007-05-16 Genzyme Corp Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern
JPS6395765U (cg-RX-API-DMAC7.html) 1986-12-11 1988-06-21
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4783989A (en) 1987-03-05 1988-11-15 Atlantic Richfield Company Vapor pressure measurement apparatus and method
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5593970A (en) 1990-06-11 1997-01-14 Biochem Pharma Inc. Heterocyclic anthracycline analogs
US5210015A (en) 1990-08-06 1993-05-11 Hoffman-La Roche Inc. Homogeneous assay system using the nuclease activity of a nucleic acid polymerase
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US7745416B2 (en) 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
EP0831854A4 (en) 1995-06-06 2001-01-24 Isis Pharmaceuticals Inc OLIGONUCLEOTIDS WITH PHOSPHOROTHIOATE BINDINGS OF HIGH CHIRAL PURITY
WO1997000321A1 (en) 1995-06-14 1997-01-03 Commonwealth Scientific And Industrial Research Organisation Immune response modulators and uses therefor
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
JP4303418B2 (ja) 1997-10-24 2009-07-29 ライフ テクノロジーズ コーポレーション 組換え部位を有する核酸を使用する組換えクローニング
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7868149B2 (en) 1999-07-20 2011-01-11 Monsanto Technology Llc Plant genome sequence and uses thereof
US6479808B1 (en) 1999-07-07 2002-11-12 The United States Of America As Represented By The Administrator Of The National Aeronautics And Space Administration Method and systems for collecting data from multiple fields of view
US6603061B1 (en) 1999-07-29 2003-08-05 Monsanto Company Agrobacterium-mediated plant transformation method
GB0024550D0 (cg-RX-API-DMAC7.html) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
WO2002080851A2 (en) 2001-04-05 2002-10-17 The Johns Hopkins University Chimeric vaccines
IL159756A0 (en) 2001-07-12 2004-06-20 Univ Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
AU2002327430A1 (en) 2001-08-08 2003-02-24 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
WO2003045304A2 (en) 2001-09-26 2003-06-05 Mayo Foundation For Medical Education And Research Mutable vaccines
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
US20090100536A1 (en) 2001-12-04 2009-04-16 Monsanto Company Transgenic plants with enhanced agronomic traits
JP2005512598A (ja) 2001-12-21 2005-05-12 オックスフォード バイオメディカ （ユーケー） リミテッド Ｅｉａｖなどのレンチウイルス発現ベクターを使用するトランスジェニック生物の作製方法
US7206639B2 (en) 2002-03-15 2007-04-17 Sarcos Investments Lc Cochlear drug delivery system and method
AU2003224897A1 (en) 2002-04-09 2003-10-27 Kenneth L. Beattie Oligonucleotide probes for genosensor chips
AU2003274404A1 (en) 2002-06-11 2003-12-22 The Scripps Research Institute Artificial transcription factors
EP2338478B1 (en) 2002-06-28 2014-07-23 Protiva Biotherapeutics Inc. Method for producing liposomes
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
AU2003283976B2 (en) 2002-09-27 2009-12-10 Cold Spring Harbor Laboratory Cell-based RNA interference and related methods and compositions
US9534224B2 (en) * 2002-11-15 2017-01-03 Trustees Of Boston University Cis/trans riboregulators
US20060178297A1 (en) 2003-01-28 2006-08-10 Troy Carol M Systems and methods for silencing expression of a gene in a cell and uses thereof
AU2004257191B2 (en) 2003-07-03 2009-05-07 The Regents Of The University Of California Genome mapping of functional DNA elements and cellular proteins
JP4951338B2 (ja) 2003-07-16 2012-06-13 プロチバ バイオセラピューティクス インコーポレイティッド 脂質に封入された干渉ｒｎａ
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
JP4842821B2 (ja) 2003-09-15 2011-12-21 プロチバ バイオセラピューティクス インコーポレイティッド ポリエチレングリコール修飾脂質化合物およびその使用
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
US8673634B2 (en) 2003-11-13 2014-03-18 Massachusetts Eye & Ear Infirmary Method for the treatment of hearing loss
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
WO2005074511A2 (en) 2004-01-27 2005-08-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for homozygous gene inactivation using collections of pre-defined nucleotide sequences complementary to chromosomal transcripts
WO2005072793A1 (en) 2004-01-29 2005-08-11 The Charles Stark Draper Laboratory, Inc. Implantable drug delivery apparatus
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
CA2573702C (en) 2004-07-16 2013-10-15 The Government Of The United States Of America As Represented By The Sec Retary Of The Department Of Health And Human Services Vaccine constructs and combination of vaccines designed to improve the breadth of the immune response to diverse strains and clades of hiv
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006116756A1 (en) * 2005-04-28 2006-11-02 Benitec, Limited. Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
DK3284833T3 (da) 2005-08-26 2022-02-07 Dupont Nutrition Biosci Aps Anvendelse af crispr-associerede gener (cas)
WO2007030743A2 (en) 2005-09-08 2007-03-15 Massachusetts Eye & Ear Infirmary Cochlear implants containing biological cells and uses thereof
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
EP1989307B1 (en) 2006-02-08 2012-08-08 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
EP2835429B1 (en) 2006-05-11 2017-12-13 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
JP5453097B2 (ja) 2006-11-14 2014-03-26 セレクティス Ｈｐｒｔ遺伝子からのｄｎａ標的配列を切断するメガヌクレアーゼ変異型及びその使用
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
US8173792B2 (en) 2007-02-09 2012-05-08 The Board Of Trustees Of The Leland Stanford Junior University Method for regulating protein function in cells using synthetic small molecules
BRPI0808704B1 (pt) 2007-03-02 2022-01-18 Dupont Nutrition Biosciences Aps Método para gerar uma cultura inicial compreendendo pelo menos duas cepas variantes resistentes a bacteriófagos, cultura iniciadora e método de fermentação
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
CN101815446A (zh) 2007-05-29 2010-08-25 施莲·特萨特斯-斯特劳斯 带有内置防护垫的衣服
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
WO2009019528A1 (en) 2007-08-03 2009-02-12 Cellectis Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof
CA2696209C (en) 2007-08-14 2016-10-25 Fred Hutchinson Cancer Research Center Needle array assembly and method for delivering therapeutic agents
EP2225002A4 (en) 2007-12-31 2011-06-22 Nanocor Therapeutics Inc RNA INTERFERENCE FOR THE TREATMENT OF CARDIAC SUFFICIENCIES
WO2009100438A2 (en) 2008-02-07 2009-08-13 Massachusetts Eye & Ear Infirmary Compounds that enhance atoh-1 expression
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
JP5475753B2 (ja) 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
US20090280262A1 (en) 2008-05-08 2009-11-12 Chung Yuan Christian University Method for forming composite membrane with porous coating layer and apparatus thereof
EP2293800B1 (en) 2008-06-06 2016-10-05 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
JP2011526619A (ja) 2008-06-30 2011-10-13 サイレンシード リミテッド 局所ドラッグデリバリーシステム、その方法、および、その組成物
EP2309980A1 (en) 2008-07-08 2011-04-20 S.I.F.I. Societa' Industria Farmaceutica Italiana Ophthalmic compositions for treating pathologies of the posterior segment of the eye
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
WO2010014218A2 (en) 2008-07-29 2010-02-04 Academia Sinica Puf-a and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
CN102177235A (zh) 2008-09-08 2011-09-07 赛莱克蒂斯公司 切割来自谷氨酰胺合成酶基因的dna靶序列的大范围核酸酶变体及其用途
EP2334794B8 (en) 2008-09-15 2017-04-19 The Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
JP5791505B2 (ja) 2008-10-20 2015-10-07 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． トランスチレチンの発現を阻害するための組成物および方法
CA2741119C (en) 2008-10-29 2019-02-12 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
CN104910025B (zh) 2008-11-07 2019-07-16 麻省理工学院 氨基醇类脂质和其用途
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
US20110023153A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
WO2010068816A1 (en) 2008-12-10 2010-06-17 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
WO2010107493A2 (en) 2009-03-20 2010-09-23 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
RU2556376C2 (ru) 2009-04-07 2015-07-10 ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи Опосредованная наночастицами доставка сиквенс-специфичных нуклеаз
WO2010129602A2 (en) 2009-05-04 2010-11-11 Fred Hutchinson Cancer Research Center Cocal vesiculovirus envelope pseudotyped retroviral vectors
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
KR20100133319A (ko) 2009-06-11 2010-12-21 주식회사 툴젠 표적 특이적인 게놈의 재배열을 위한 표적 특이적 뉴클레아제 및 이의 용도
US8385868B2 (en) 2009-06-15 2013-02-26 Agc Automotive Americas R&D, Inc. Diversity antenna system and method utilizing a threshold value
CA2767127A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP2449106B1 (en) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
EP2727600B1 (en) 2009-07-28 2019-03-27 Sangamo Therapeutics, Inc. Zinc finger fusion proteins for repressing a huntington gene
US8927807B2 (en) 2009-09-03 2015-01-06 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
CA2784547A1 (en) 2009-12-23 2011-06-30 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Influenza targets
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
WO2011139336A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using nucleases
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP3078753B1 (en) 2010-05-10 2018-04-18 The Regents of The University of California Methods using endoribonuclease compositions
WO2011141825A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
ES2536791T3 (es) 2010-05-28 2015-05-28 Oxford Biomedica (Uk) Ltd Administración de vectores lentivirales al cerebro
EP3489359A1 (en) 2010-07-23 2019-05-29 Sigma Aldrich Co. LLC Genome editing using targeting endonucleases and single-stranded nucleic acids
DK2601611T3 (da) 2010-08-02 2021-02-01 Integrated Dna Tech Inc Fremgangsmåder til forudsigelse af stabilitet og smeltetemperaturer for nukleinsyreduplekser
WO2012027675A2 (en) 2010-08-26 2012-03-01 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US9549901B2 (en) 2010-09-03 2017-01-24 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
ES2562421T3 (es) 2010-10-12 2016-03-04 The Children's Hospital Of Philadelphia Métodos y composiciones para tratar la hemofilia B
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
JP6072772B2 (ja) 2011-04-22 2017-02-01 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 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
SG194089A1 (en) 2011-04-27 2013-11-29 Amyris Inc Methods for genomic modification
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
WO2012164565A1 (en) * 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
AU2012318562A1 (en) 2011-10-06 2014-04-10 Sangamo Therapeutics, Inc. Methods and compositions for regulating HIV infection
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
EP2591770B1 (en) 2011-11-14 2016-03-16 Silenseed Ltd Compositions for siRNA delivery and methods of manufacturing and using same
CA2855653A1 (en) 2011-11-18 2013-05-23 Universite Laval Methods and products for increasing frataxin levels and uses thereof
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
US20130137173A1 (en) 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
AU2012352180A1 (en) 2011-12-16 2014-07-31 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
RU2018109507A (ru) 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を治療するための方法および組成物
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
KR101945578B1 (ko) 2012-06-01 2019-02-07 드렉셀유니버시티 B형 간염 바이러스 cccdna 전사의 조절
FR2993379B1 (fr) 2012-07-10 2015-09-04 Morpho Procede de traitement comparatif securise
FR2995325B1 (fr) 2012-09-11 2015-03-13 Ct Tech De L Ind Des Papiers Cartons Et Celluloses Procede de fabrication d'un support fibreux multicouche par fractionnement et stratification
CA2886684C (en) 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
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
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
KR20150105635A (ko) 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
US20140186919A1 (en) 2012-12-12 2014-07-03 Feng Zhang Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
WO2014093709A1 (en) 2012-12-12 2014-06-19 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
US20140310830A1 (en) * 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US9970024B2 (en) 2012-12-17 2018-05-15 President And Fellows Of Harvard College RNA-guided human genome engineering
US10544405B2 (en) 2013-01-16 2020-01-28 Emory University Cas9-nucleic acid complexes and uses related thereto
US11135273B2 (en) 2013-02-07 2021-10-05 The Rockefeller University Sequence specific antimicrobials
WO2014124226A1 (en) 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US9163837B2 (en) 2013-02-27 2015-10-20 Siemens Aktiengesellschaft Flow conditioner in a combustor of a gas turbine engine
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
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
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
HUE034635T2 (en) 2013-03-27 2018-02-28 Wilco Ag A method for checking and / or testing devices in a row and equipment for performing such a procedure
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
DK3004349T3 (en) 2013-05-29 2018-06-06 Cellectis Sa A method for producing precise DNA cleavage using CAS9 nickase activity
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
WO2014197568A2 (en) 2013-06-04 2014-12-11 President And Fellows Of Harvard College Rna-guideded transcriptional regulation
EP4435110A3 (en) 2013-06-05 2025-01-01 Duke University Rna-guided gene editing and gene regulation
EP3620524A1 (en) 2013-06-17 2020-03-11 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN106062197A (zh) 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
JP7118588B2 (ja) 2013-08-29 2022-08-16 テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション Ｈｉｖ感染のｒｎａガイド処置のための方法および組成物
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3071695A2 (en) 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
CN105899657A (zh) 2013-12-12 2016-08-24 布罗德研究所有限公司 用于改变基因产物表达的crispr-cas系统和方法、结构信息以及诱导型模块化cas酶
WO2015089419A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
CN103668472B (zh) 2013-12-31 2014-12-24 北京大学 利用CRISPR/Cas9系统构建真核基因敲除文库的方法
CN103779358A (zh) * 2014-01-27 2014-05-07 京东方科技集团股份有限公司 一种阵列基板及其制作方法、显示装置
WO2015113063A1 (en) 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
CA2951707A1 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
JP6323228B2 (ja) 2014-07-18 2018-05-16 富士電機株式会社 電力変換装置
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
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
DE102014221482A1 (de) 2014-10-22 2016-04-28 Ford Global Technologies, Llc Verfahren zur Ansteuerung einer Spannungsquelle zum Laden einer Batterie eines Kraftfahrzeugs
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
EP3907285A1 (en) 2015-05-06 2021-11-10 Snipr Technologies Limited Altering microbial populations & modifying microbiota
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
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
JP6186470B2 (ja) 2016-04-20 2017-08-23 パイオニア株式会社 音響装置、音量制御方法、音量制御プログラム及び記録媒体
WO2018154439A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
KR102085165B1 (ko) 2017-11-10 2020-03-05 주식회사 엘지화학 유기 금속 화합물 및 이를 포함하는 유기 발광 소자
US11116729B2 (en) 2019-01-17 2021-09-14 Georgia Tech Research Corporation Drug delivery systems containing oxidized cholesterols
US20220273566A1 (en) 2019-07-29 2022-09-01 Georgia Tech Research Corporation Nanomaterials containing constrained lipids and uses thereof

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004519245A (ja) * 2001-03-16 2004-07-02 直哉 小林 肝臓細胞の増殖方法、該方法により得られる肝臓細胞、およびその用途
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
JP2012510812A (ja) * 2008-12-04 2012-05-17 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
WO2011011767A1 (en) * 2009-07-24 2011-01-27 Sigma-Aldrich Co. Method for genome editing
WO2011064736A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
WO2011146121A1 (en) * 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
WO2014065596A1 (en) * 2012-10-23 2014-05-01 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
WO2014089290A1 (en) * 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
BIOCHIMICA ET BIOPHYSICA ACTA, 2006, VOL.1758, P.355-363, JPN6017007681, ISSN: 0003913030 *
ELIFE, JAN 2013, VOL.2, E00471, JPN6017016126, ISSN: 0003913036 *
FEBS LETT., 2012, 586(15), P.2076-2083, EPUB 2012 FEB 28, JPN6018033752, ISSN: 0003913033 *
MOL. CELL. BIOL., 1987, 7(6), P.2087-2096, JPN6018033753, ISSN: 0003913034 *
PNAS, 2011, VOL.108, P.E351-E358, JPN6017007683, ISSN: 0003913032 *
SCIENCE, AUG 2012, VOL.337, P.816-821, SUPPLEMENTARY MATERIALS, JPN6016021641, ISSN: 0003913029 *
SCIENCE, JAN 2013, VOL.339, P.823-826, JPN6017016124, ISSN: 0003913035 *
THE JOURNAL OF CELL BIOLOGY, 1988, VOL.107, P.1279-1287, JPN6017007682, ISSN: 0003913031 *

Cited By (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12252707B2 (en) 2012-12-12 2025-03-18 The Broad Institute, Inc. Delivery, Engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US11041173B2 (en) 2012-12-12 2021-06-22 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
US12454687B2 (en) 2012-12-12 2025-10-28 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, knock out libraries and applications thereof
US10930367B2 (en) 2012-12-12 2021-02-23 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for Cas enzymes or CRISPR-Cas systems for target sequences and conveying results thereof
JP2021192624A (ja) * 2013-03-15 2021-12-23 ザ ジェネラル ホスピタル コーポレイション 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2023061983A (ja) * 2013-03-15 2023-05-02 ザ ジェネラル ホスピタル コーポレイション 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
JP7376650B2 (ja) 2013-06-04 2023-11-08 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 Ｒｎａ誘導性転写制御
JP2022166077A (ja) * 2013-06-04 2022-11-01 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性転写制御
JP2016521554A (ja) * 2013-06-04 2016-07-25 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性転写制御
US11008588B2 (en) 2013-06-17 2021-05-18 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
US12018275B2 (en) 2013-06-17 2024-06-25 The Broad Institute, Inc. Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy
US10946108B2 (en) 2013-06-17 2021-03-16 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
US12441995B2 (en) 2013-06-17 2025-10-14 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
JP2016534125A (ja) * 2013-08-29 2016-11-04 テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション Ｈｉｖ感染のｒｎａガイド処置のための方法および組成物
US11285193B2 (en) 2013-08-29 2022-03-29 Temple University—Of the Commonwealth System of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US11291710B2 (en) 2013-08-29 2022-04-05 Temple University—Of the Commonwealth System of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US11298411B2 (en) 2013-08-29 2022-04-12 Temple University—Of the Commonwealth System of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US11597919B2 (en) 2013-12-12 2023-03-07 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US11407985B2 (en) 2013-12-12 2022-08-09 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing
US11591581B2 (en) 2013-12-12 2023-02-28 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
US12421506B2 (en) 2013-12-12 2025-09-23 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US12410435B2 (en) 2013-12-12 2025-09-09 The Broad Institute, Inc. Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US12258595B2 (en) 2013-12-12 2025-03-25 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems
US12251450B2 (en) 2013-12-12 2025-03-18 The Broad Institute, Inc. Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
US11624078B2 (en) 2014-12-12 2023-04-11 The Broad Institute, Inc. Protected guide RNAS (pgRNAS)
US12435320B2 (en) 2014-12-24 2025-10-07 The Broad Institute, Inc. CRISPR having or associated with destabilization domains
US11578312B2 (en) 2015-06-18 2023-02-14 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
US12168789B2 (en) 2015-06-18 2024-12-17 The Broad Institute, Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation
US12123032B2 (en) 2015-06-18 2024-10-22 The Broad Institute, Inc. CRISPR enzyme mutations reducing off-target effects
US12037583B2 (en) 2015-12-04 2024-07-16 Novartis Ag Compositions and methods for immunooncology
JP2020513832A (ja) * 2017-03-22 2020-05-21 ノバルティス アーゲー 免疫腫瘍学のための組成物および方法
US11851659B2 (en) 2017-03-22 2023-12-26 Novartis Ag Compositions and methods for immunooncology
JP7210028B2 (ja) 2017-07-18 2023-01-23 国立大学法人京都大学 遺伝子変異導入方法
JPWO2019017321A1 (ja) * 2017-07-18 2020-05-28 国立大学法人京都大学 遺伝子変異導入方法
JP7707140B2 (ja) 2017-09-29 2025-07-14 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法
JP2021500864A (ja) * 2017-09-29 2021-01-14 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法
JP2023029966A (ja) * 2017-09-29 2023-03-07 インテリア セラピューティクス，インコーポレイテッド Ｔｔｒ遺伝子編集およびａｔｔｒアミロイドーシスの治療用の組成物および方法
JP2021513866A (ja) * 2018-02-27 2021-06-03 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル アンジェルマン症候群を処置する方法および組成物
JP2021519067A (ja) * 2018-03-23 2021-08-10 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 常染色体優性疾患のための遺伝子編集
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法
JP7430917B2 (ja) 2018-06-12 2024-02-14 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法
JP2022514214A (ja) * 2018-12-05 2022-02-10 インテリア セラピューティクス，インコーポレーテッド 修飾されたアミン脂質
JP2024167309A (ja) * 2018-12-05 2024-12-03 インテリア セラピューティクス，インコーポレーテッド 修飾されたアミン脂質
JP7547335B2 (ja) 2018-12-05 2024-09-09 インテリア セラピューティクス，インコーポレーテッド 修飾されたアミン脂質
WO2023038020A1 (ja) 2021-09-07 2023-03-16 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法

Also Published As

Publication number Publication date
AU2025203782A1 (en) 2025-06-12
AU2019283878A1 (en) 2020-01-23
US20250250589A1 (en) 2025-08-07
MX389793B (es) 2025-03-20
MX2022001378A (es) 2022-06-23
SG10201912328UA (en) 2020-02-27
IL239317A0 (en) 2015-07-30
AU2013359199C1 (en) 2021-06-17
US12252707B2 (en) 2025-03-18
WO2014093622A9 (en) 2014-11-06
EP4299741A3 (en) 2024-02-28
EP3327127A1 (en) 2018-05-30
MX2015007550A (es) 2017-02-02
US20170107536A1 (en) 2017-04-20
EP2931897B1 (en) 2017-11-01
US20140179770A1 (en) 2014-06-26
SG11201504523UA (en) 2015-07-30
US20170152528A1 (en) 2017-06-01
JP7228059B2 (ja) 2023-02-22
IL293526A (en) 2022-08-01
AU2013359199B2 (en) 2019-10-31
WO2014093622A2 (en) 2014-06-19
US20150020223A1 (en) 2015-01-15
AU2013359199A1 (en) 2015-07-30
ES2883590T3 (es) 2021-12-09
AU2022203759A1 (en) 2022-06-23
JP7736729B2 (ja) 2025-09-09
CN105164264A (zh) 2015-12-16
AU2022203759B2 (en) 2025-02-27
WO2014093622A8 (en) 2014-12-04
EP3031921A1 (en) 2016-06-15
WO2014093622A3 (en) 2014-08-07
CN105164264B (zh) 2021-05-07
HK1252619A1 (en) 2019-05-31
HK1220725A1 (en) 2017-05-12
IL239317B (en) 2022-07-01
JP2025181841A (ja) 2025-12-11
ZA201504132B (en) 2022-03-30
US20140242699A1 (en) 2014-08-28
EP4549566A2 (en) 2025-05-07
US20210269831A1 (en) 2021-09-02
RU2721275C2 (ru) 2020-05-18
CA2894681A1 (en) 2014-06-19
KR20150105956A (ko) 2015-09-18
AU2019283878B2 (en) 2022-06-09
HK1215048A1 (en) 2016-08-12
EP4549566A3 (en) 2025-07-09
JP7013406B2 (ja) 2022-02-15
RU2015128057A (ru) 2019-03-27
JP2019103513A (ja) 2019-06-27
SG10201707569YA (en) 2017-10-30
EP3327127B1 (en) 2021-03-24
CN110872583A (zh) 2020-03-10
DK2931897T3 (en) 2018-02-05
EP3031921B1 (en) 2025-03-12
EP2931897A2 (en) 2015-10-21
JP2023073245A (ja) 2023-05-25
JP2022040308A (ja) 2022-03-10
US20250382642A1 (en) 2025-12-18
BR112015013784A2 (pt) 2017-07-11
ES2658401T3 (es) 2018-03-09
US20230399662A1 (en) 2023-12-14
US20240035048A1 (en) 2024-02-01
US11041173B2 (en) 2021-06-22
EP4299741A2 (en) 2024-01-03
DK3327127T3 (da) 2021-06-28

Similar Documents

Publication Publication Date Title
JP7228059B2 (ja) 配列操作および治療適用のための系、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
JP7689420B2 (ja) ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
JP6738729B2 (ja) 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
JP6702858B2 (ja) ウイルス成分を使用して障害および疾患をターゲティングするためのＣＲＩＳＰＲ−Ｃａｓ系および組成物の送達、使用および治療上の適用
JP6738728B2 (ja) 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
JP2017501149A6 (ja) 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
JP2017501149A (ja) 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
BR122024006902A2 (pt) Aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
BR112015031610B1 (pt) Composição para modificação de uma célula cerebral ou neuronal in vivo em um organismo eucariótico e uso de um vetor viral no tratamento de uma doença ou distúrbio cerebral ou neurológico

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20161212

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20161212

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170111

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20171204

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20180302

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180604

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20180606

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20181105

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190305

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20190305

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20190307

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20190401

C27A Decision to dismiss

Free format text: JAPANESE INTERMEDIATE CODE: C2711

Effective date: 20190520

C30A Notification sent

Free format text: JAPANESE INTERMEDIATE CODE: C3012

Effective date: 20190610