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 (enExample
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)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (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)
  • Spectroscopy & Molecular Physics (AREA)
  • Medical Informatics (AREA)
  • Evolutionary Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (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
US61/802,174 2013-03-15
US61/791,409 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
US61/835,931 2013-06-17
US61/836,123 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 (enExample) 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 (enExample)
EP (5) EP4549566A3 (enExample)
JP (5) JP2016501531A (enExample)
KR (1) KR20150105956A (enExample)
CN (2) CN110872583A (enExample)
AU (4) AU2013359199C1 (enExample)
BR (1) BR112015013784A2 (enExample)
DK (2) DK3327127T3 (enExample)
ES (2) ES2883590T3 (enExample)
IL (2) IL239317B (enExample)
MX (2) MX389793B (enExample)
RU (1) RU2721275C2 (enExample)
SG (3) SG10201912328UA (enExample)
WO (1) WO2014093622A2 (enExample)
ZA (1) ZA201504132B (enExample)

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
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
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
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 (877)

* 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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CN102638971B (zh) 2009-07-08 2015-10-07 科马布有限公司 动物模型及治疗分子
US9445581B2 (en) 2012-03-28 2016-09-20 Kymab Limited Animal models and therapeutic molecules
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
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
ES2705236T3 (es) 2010-05-12 2019-03-22 Univ Columbia Procedimientos para producir células enteroendocrinas que producen y secretan insulina
BR112013024337A2 (pt) 2011-03-23 2017-09-26 Du Pont locus de traço transgênico complexo em uma planta, planta ou semente, método para produzir em uma planta um locus de traço transgênico complexo e construto de expressão
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
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
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
BR112014006390A2 (pt) 2011-09-19 2017-03-28 Kymab Ltd anticorpos, domínios variáveis e cadeias feitos especialmente para uso humano
WO2013045916A1 (en) 2011-09-26 2013-04-04 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
EP3604555B1 (en) 2011-10-14 2024-12-25 President and Fellows of Harvard College Sequencing by structure assembly
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
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
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
GB2502127A (en) 2012-05-17 2013-11-20 Kymab Ltd Multivalent antibodies and in vivo methods for their production
US10251377B2 (en) 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
CN104364380B (zh) 2012-04-25 2018-10-09 瑞泽恩制药公司 核酸酶介导的使用大靶向载体的靶向
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
EP2847215A1 (en) * 2012-05-07 2015-03-18 Synthes GmbH Methods and devices for treating intervertebral disc disease
PL4289948T3 (pl) 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2912175B1 (en) 2012-10-23 2018-08-22 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
CN105142669B (zh) 2012-12-06 2018-07-03 西格马-奥尔德里奇有限责任公司 基于crispr的基因组修饰和调控
PT2898075E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
CN105658796B (zh) 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
PL2784162T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Opracowanie systemów, metod oraz zoptymalizowanych kompozycji przewodnikowych do manipulacji sekwencyjnej
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
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
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
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
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
GB201306536D0 (en) 2013-04-10 2013-05-22 Gt Biolog Ltd Polypeptide and immune modulation
CA2908697C (en) 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
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
CN116083487A (zh) * 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
JP7065564B2 (ja) * 2013-05-29 2022-05-12 セレクティス Ｃａｓ９ニッカーゼ活性を用いて正確なｄｎａ切断をもたらすための方法
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) * 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
AU2014287397B2 (en) * 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
US9663782B2 (en) * 2013-07-19 2017-05-30 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
US11306328B2 (en) 2013-07-26 2022-04-19 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
MX363842B (es) * 2013-08-22 2019-04-05 Du Pont Métodos para producir modificaciones genéticas en un genoma vegetal sin incorporar un marcador transgénico seleccionable, y composiciones de éstas.
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI 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
EP3418379B1 (en) 2013-09-18 2020-12-09 Kymab Limited Methods, cells & organisms
ES2993142T3 (en) 2013-10-01 2024-12-23 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
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
WO2015071474A2 (en) * 2013-11-18 2015-05-21 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
HUE041331T2 (hu) 2013-12-11 2019-05-28 Regeneron Pharma Módszerek és készítmények a genom célzott módosításához
SG10201700961TA (en) 2013-12-11 2017-04-27 Regeneron Pharma Methods and compositions for the targeted modification of a genome
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080260B1 (en) 2013-12-12 2019-03-06 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 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
US9725710B2 (en) 2014-01-08 2017-08-08 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
JP2017510542A (ja) * 2014-01-31 2017-04-13 ファクター バイオサイエンス インコーポレイテッド 核酸生成及び送達のための方法及び製品
DE212015000061U1 (de) 2014-02-11 2017-09-03 The Regents Of The University Of Colorado, A Body Corporate CRISPR-ermöglichtes Multiplex Genom Engineering
KR20160130392A (ko) 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
EP3110971B1 (en) 2014-02-27 2019-05-08 The Broad Institute Inc. T cell balance gene expression and methods of use thereof
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US12460231B2 (en) 2014-04-02 2025-11-04 Editas Medicine, Inc. Crispr/CAS-related methods and compositions for treating primary open angle glaucoma
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
CA2947270A1 (en) * 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic acids targeting mdm2 or mycn
US10280419B2 (en) * 2014-05-09 2019-05-07 UNIVERSITé LAVAL Reduction of amyloid beta peptide production via modification of the APP gene using the CRISPR/Cas system
WO2015178343A1 (ja) * 2014-05-20 2015-11-26 国立大学法人北海道大学 ｓｉＲＮＡ細胞内送達のための脂質膜構造体
SG10201801654RA (en) 2014-05-28 2018-04-27 Childrens Hospital Med Ct Methods and systems for converting precursor cells into gastric tissues through directed differentiation
BR112016028023A2 (pt) * 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
HUE049776T2 (hu) 2014-06-06 2020-10-28 Regeneron Pharma Módszerek és készítmények egy célzott lókusz módosítására
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
KR102425438B1 (ko) 2014-06-23 2022-07-27 더 제너럴 하스피탈 코포레이션 서열결정에 의해 평가된 DSB의 게놈 전체에 걸친 비편향된 확인 (GUIDE-Seq)
MX384887B (es) 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
WO2015200901A1 (en) 2014-06-26 2015-12-30 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
EP3161128B1 (en) 2014-06-26 2018-09-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
US10081816B1 (en) * 2014-07-03 2018-09-25 Nant Holdings Ip, Llc Mechanical transfection devices and methods
US10760040B1 (en) 2014-07-03 2020-09-01 NanoCav, 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
WO2016007347A1 (en) 2014-07-11 2016-01-14 E. I. Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
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
BR122023024818A2 (pt) * 2014-07-11 2023-12-26 Pioneer Hi-Bred International, Inc. Rna guia, polinucleotídeo e complexo de ribonucleoproteínas
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
CA2956224A1 (en) 2014-07-30 2016-02-11 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
JP6837429B2 (ja) * 2014-08-11 2021-03-03 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム Ｃｒｉｓｐｒ／ｃａｓ９媒介遺伝子編集による筋ジストロフィーの予防
CN107429241B (zh) * 2014-08-14 2025-10-24 百奥赛图(北京)医药科技股份有限公司 Dna敲入系统
EP3686279B1 (en) 2014-08-17 2023-01-04 The Broad Institute, Inc. Genome editing using cas9 nickases
AU2015308910B2 (en) * 2014-08-27 2017-12-07 Caribou Biosciences, Inc. Methods for increasing Cas9-mediated engineering efficiency
US10570418B2 (en) 2014-09-02 2020-02-25 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification
CA2956487A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
WO2016049024A2 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
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
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
EP3795580A1 (en) 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
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
DK3204022T3 (da) 2014-10-09 2021-03-08 Celularity Inc Placenta-afledte adhærente celle-exosomer og anvendelser deraf
US20170354745A1 (en) * 2014-10-09 2017-12-14 The Regents Of The University Of California Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
DK3207124T3 (da) 2014-10-15 2019-08-12 Regeneron Pharma Fremgangsmåder og sammensætninger til generering eller bevaring af pluripotente celler
US11174506B2 (en) 2014-10-17 2021-11-16 Howard Hughes Medical Institute Genomic probes
AU2015331848B2 (en) 2014-10-17 2022-03-03 Children's Hospital Medical Center, D/B/A Cincinnati Children's Hospital Medical Center In vivo model of human small intestine using pluripotent stem cells and methods of making and using same
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3212788A2 (en) 2014-10-27 2017-09-06 The Broad Institute, Inc. Compositions, methods and use of synthetic lethal screening
US11071790B2 (en) 2014-10-29 2021-07-27 Massachusetts Eye And Ear Infirmary Method for efficient delivery of therapeutic molecules in vitro and in vivo
DK3212789T3 (da) * 2014-10-31 2020-07-27 Massachusetts Inst Technology Massiv parallel kombinatorisk genetik til crispr
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions 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
US20180057839A1 (en) 2014-11-26 2018-03-01 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
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
CN113699116A (zh) 2014-12-10 2021-11-26 明尼苏达大学董事会 用于治疗疾病的遗传修饰的细胞、组织和器官
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
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)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
JP6840077B2 (ja) 2014-12-19 2021-03-10 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． 単一ステップの複数標的化を通じた標的化された遺伝子修飾のための方法及び組成物
US10196613B2 (en) 2014-12-19 2019-02-05 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
WO2016100974A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
PL3193901T3 (pl) 2014-12-23 2018-10-31 4D Pharma Research Limited Polipeptyd pyrynowy i immunomodulacja
CN113862175A (zh) 2014-12-23 2021-12-31 4D制药研究有限公司 免疫调控
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
SG11201704272YA (en) 2014-12-31 2017-06-29 Synthetic Genomics Inc Compositions and methods for high efficiency in vivo genome editing
WO2016114972A1 (en) 2015-01-12 2016-07-21 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
LT3250691T (lt) 2015-01-28 2023-09-11 Caribou Biosciences, Inc. Crispr hibridiniai dnr/rnr polinukleotidai ir naudojimo būdai
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
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
US10584321B2 (en) * 2015-02-13 2020-03-10 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
WO2016138574A1 (en) 2015-03-02 2016-09-09 Sinai Health System Homologous recombination factors
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
US10450576B2 (en) 2015-03-27 2019-10-22 E I Du Pont De Nemours And Company Soybean U6 small nuclear RNA gene promoters and their use in constitutive expression of small RNA genes in plants
CA3000187A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
ES2959608T3 (es) * 2015-04-03 2024-02-27 Dana Farber Cancer Inst Inc Composición y métodos de edición del genoma de células B
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
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
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016187717A1 (en) * 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
US20160346360A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
MX2017015582A (es) 2015-06-01 2018-09-06 Univ Temple Metodos y composiciones para tratamiento guiado por arn de infeccion por vih.
EP3302053B1 (en) 2015-06-02 2021-03-17 Monsanto Technology LLC Compositions and methods for delivery of a polynucleotide into a plant
GB201509578D0 (en) * 2015-06-03 2015-07-15 Univ Singapore Vectors
WO2016196655A1 (en) 2015-06-03 2016-12-08 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
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
RS59446B1 (sr) 2015-06-15 2019-11-29 4D Pharma Res Ltd Blautia stercosis i wexlerae za upotrebu u lečenju inflamatornih i autoimunskih bolesti
MA41010B1 (fr) 2015-06-15 2020-01-31 4D Pharma Res Ltd Compositions comprenant des souches bactériennes
MA41060B1 (fr) 2015-06-15 2019-11-29 4D Pharma Res Ltd Compositions comprenant des souches bactériennes
PE20180267A1 (es) 2015-06-15 2018-02-06 4D Pharma Res Ltd Composiciones que comprenden cepas bacterianas
MD3206700T2 (ro) 2015-06-15 2019-12-31 4D Pharma Res Ltd Compoziții conținând tulpini bacteriene
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
WO2016205688A2 (en) * 2015-06-18 2016-12-22 Bowles Robert D Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP4159856A1 (en) 2015-06-18 2023-04-05 The Broad Institute, Inc. Novel crispr enzymes and systems
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP3316159A4 (en) 2015-06-25 2019-08-14 Advanced Telecommunications Research Institute International PREDICTIVE EQUIPMENT BASED ON A SYSTEM ASSOCIATED WITH SEVERAL INSTITUTIONS AND PREDICTION PROGRAM
EP3313416A4 (en) * 2015-06-26 2019-02-20 The Trustees of Columbia University in the City of New York GENETICALLY MODIFIED IPS CELLS WITH A MARKER FOR THE EXPRESSION OF NEUROGENIN3, TPH2, FOXO1 AND / OR INSULINGENES
US11414657B2 (en) 2015-06-29 2022-08-16 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
US10456452B2 (en) 2015-07-02 2019-10-29 Poseida Therapeutics, Inc. Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles
US20170000743A1 (en) * 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
ES2948559T3 (es) * 2015-07-25 2023-09-14 Habib Frost Un sistema, un dispositivo y un método para proporcionar una terapia o una cura para el cáncer y otros estados patológicos
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US20180208945A1 (en) * 2015-07-28 2018-07-26 Danisco Us Inc. Genome editing systems and methods of use
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
IL295616A (en) * 2015-07-31 2022-10-01 Us Health Adapted cells and treatment methods
WO2017027810A2 (en) 2015-08-12 2017-02-16 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
AU2016308339A1 (en) 2015-08-18 2018-04-12 Baylor College Of Medicine Methods and compositions for altering function and structure of chromatin loops and/or domains
CN108351350B (zh) * 2015-08-25 2022-02-18 杜克大学 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
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
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
WO2017044843A1 (en) 2015-09-11 2017-03-16 The General Hospital Corporation Full interrogation of nuclease dsbs and sequencing (find-seq)
PL3350333T3 (pl) 2015-09-17 2022-03-07 Modernatx, Inc. Polinukleotydy zawierające region stabilizujący ogon
EP3786294A1 (en) 2015-09-24 2021-03-03 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US9850484B2 (en) 2015-09-30 2017-12-26 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
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US11970710B2 (en) 2015-10-13 2024-04-30 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
WO2017070605A1 (en) 2015-10-22 2017-04-27 The Broad Institute Inc. Type vi-b crispr enzymes and systems
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
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
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
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
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
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3371329B1 (en) 2015-11-03 2026-03-25 President and Fellows of Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
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
EP4036228A1 (en) 2015-11-13 2022-08-03 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
US11617783B2 (en) 2015-11-16 2023-04-04 Research Institute At Nationwide Children's Hospital Repairing a mutant human titin gene using CRISPR technology
US11905521B2 (en) 2015-11-17 2024-02-20 The Chinese University Of Hong Kong Methods and systems for targeted gene manipulation
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
WO2017085520A1 (en) 2015-11-20 2017-05-26 4D Pharma Research Limited Compositions comprising bacterial strains
EP4163374A1 (en) 2015-11-23 2023-04-12 The Regents of the University of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
US20210395799A1 (en) * 2015-11-25 2021-12-23 Integrated Dna Technologies, Inc. Methods for variant detection
CN116064747A (zh) * 2015-11-25 2023-05-05 综合基因技术公司 用于变体检测的方法
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. 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
CN109072195A (zh) 2015-12-30 2018-12-21 诺华股份有限公司 具有增强功效的免疫效应细胞疗法
MX2018008344A (es) 2016-01-11 2018-12-06 Univ Leland Stanford Junior Proteinas quimericas y metodos para regular la expresion genica.
JP7012645B2 (ja) 2016-01-11 2022-01-28 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー キメラタンパク質および免疫治療の方法
US10053693B2 (en) 2016-01-19 2018-08-21 Mubin I. Syed Method for controlling obesity using minimally invasive means
JP6914274B2 (ja) 2016-01-22 2021-08-04 ザ・ブロード・インスティテュート・インコーポレイテッド Ｃｒｉｓｐｒｃｐｆ１の結晶構造
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
EP3413928B1 (en) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017142835A1 (en) * 2016-02-15 2017-08-24 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
CN116763942A (zh) 2016-02-18 2023-09-19 宾州研究基金会 脑内生成gaba能神经元
US20190249172A1 (en) 2016-02-18 2019-08-15 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
EP3420102B1 (en) 2016-02-22 2024-04-03 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
EP3420089B1 (en) * 2016-02-26 2021-12-29 LanzaTech NZ, Inc. Crispr/cas systems for c-1 fixing bacteria
JP6441536B2 (ja) 2016-03-04 2018-12-19 フォーディー ファーマ ピーエルシー４Ｄ Ｐｈａｒｍａ Ｐｌｃ 細菌株を含む組成物
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
GB201612191D0 (en) 2016-07-13 2016-08-24 4D Pharma Plc Compositions comprising bacterial strains
JP2019515654A (ja) 2016-03-16 2019-06-13 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 肥満及び／又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
WO2017161325A1 (en) 2016-03-17 2017-09-21 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
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
EP3466446B1 (en) * 2016-03-29 2023-12-27 Karydo Therapeutix, Inc. Pharmaceutical composition or food composition, and method for assessing effect of active ingredient in vivo
SMT202500211T1 (it) * 2016-03-30 2025-07-22 Intellia Therapeutics Inc Formulazioni di nanoparticelle lipidiche per componenti crispr/cas
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017189308A1 (en) 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CA3210120C (en) 2016-04-25 2024-04-09 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
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
ES2865481T3 (es) 2016-04-29 2021-10-15 Poseida Therapeutics Inc Micelas basadas en poli(histidina) para la complejación y el aporte de proteínas y ácidos nucleicos
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
CN109415685B (zh) 2016-05-05 2023-07-04 儿童医院医疗中心 用于体外制造胃底组织的方法和与其相关的组合物
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN109475109B (zh) 2016-05-20 2021-10-29 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
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
KR20190019168A (ko) 2016-06-17 2019-02-26 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会（法人团体） 用于生成条形码化组合文库的方法
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US20210222164A1 (en) 2016-06-29 2021-07-22 The Broad Institute, Inc. Crispr-cas systems having destabilization domain
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
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
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
CN109789185A (zh) * 2016-07-28 2019-05-21 基础科学研究院 用于治疗眼部疾病的含cas9蛋白和向导rna的药物组合物
RU2721125C1 (ru) 2016-07-29 2020-05-18 Регенерон Фармасьютикалз, Инк. Мыши, содержащие мутации, вследствие которых экспрессируется укороченный на с-конце фибриллин-1
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
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
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
ES2998057T3 (en) * 2016-08-12 2025-02-18 Toolgen Inc Manipulated immunoregulatory element and immunity altered thereby
CN110312799A (zh) * 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035364A1 (en) 2016-08-17 2018-02-22 The Broad Institute Inc. Product and methods useful for modulating and evaluating immune responses
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
AU2017313917B2 (en) * 2016-08-18 2023-12-21 The Regents Of The University Of California CRISPR-Cas genome engineering via a modular AAV delivery system
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
US20220056440A1 (en) * 2016-08-20 2022-02-24 Tara Moore Crispr gene editing for autosomal dominant diseases
CN109963945A (zh) 2016-08-20 2019-07-02 阿维利诺美国实验室股份有限公司 单一向导rna、crispr/cas9系统及其使用方法
WO2019165322A1 (en) * 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2569252A (en) 2016-08-31 2019-06-12 Harvard College Methods of combining the detection of biomolecules into a single assay using fluorescent in situ sequencing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
JP7215994B2 (ja) 2016-09-06 2023-01-31 ザ チルドレンズ メディカル センター コーポレーション 人工多能性幹細胞由来の免疫細胞
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
US12188059B2 (en) 2016-09-13 2025-01-07 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
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
DK3516056T3 (da) * 2016-09-23 2025-02-17 Dsm Ip Assets Bv Et guide-rna-ekspressionssystem til en værtscelle
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018064352A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
JP2019532644A (ja) 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
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
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
EP4338799A3 (en) 2016-10-18 2024-06-05 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
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
EP3534976A4 (en) 2016-11-04 2020-09-16 Children's Hospital Medical Center PATHOLOGICAL MODELS OF HEPATIC ORGANOIDS AND ASSOCIATED METHODS OF MANUFACTURE AND USE
US12227578B2 (en) 2016-11-11 2025-02-18 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through T cell action
WO2018088694A2 (ko) 2016-11-14 2018-05-17 주식회사 툴젠 인위적으로 조작된 sc 기능 조절 시스템
CA3045145A1 (en) 2016-12-05 2018-06-14 Children's Hospital Medical Center Colonic organoids and methods of making and using same
WO2018107103A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr-systems for modifying a trait of interest in a plant
JP7228514B2 (ja) 2016-12-09 2023-02-24 ザ・ブロード・インスティテュート・インコーポレイテッド Ｃｒｉｓｐｒエフェクターシステムベースの診断法
GB201621123D0 (en) 2016-12-12 2017-01-25 4D Pharma Plc Compositions comprising bacterial strains
CA3045134A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload delivery
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018119354A1 (en) * 2016-12-23 2018-06-28 President And Fellows Of Harvard College Gene editing of pcsk9
JP7369524B2 (ja) * 2016-12-27 2023-10-26 中外製薬株式会社 被験物質の免疫原性を評価する方法
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
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
WO2018136758A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. 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
WO2018147343A1 (en) * 2017-02-07 2018-08-16 Edigene Corporation Method of treating diseases associated with elevated kras expression using crispr-gndm system
WO2018152418A1 (en) * 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
EP3589751A4 (en) * 2017-03-03 2021-11-17 The Regents of The University of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA 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
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US11104937B2 (en) 2017-03-15 2021-08-31 The Broad Institute, Inc. CRISPR effector system based diagnostics
CN120330163A (zh) 2017-03-15 2025-07-18 博德研究所 新型cas13b直向同源物crispr酶和系统
US11174515B2 (en) 2017-03-15 2021-11-16 The Broad Institute, Inc. CRISPR effector system based diagnostics
WO2018170340A1 (en) 2017-03-15 2018-09-20 The Broad Institute, Inc. Crispr effector system based diagnostics for virus detection
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
WO2018175502A2 (en) * 2017-03-21 2018-09-27 Shuber Anthony P Treating cancer with cas endonuclease complexes
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US12161694B2 (en) 2017-03-24 2024-12-10 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
US11963966B2 (en) 2017-03-31 2024-04-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
US11913075B2 (en) 2017-04-01 2024-02-27 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
WO2018191553A1 (en) 2017-04-12 2018-10-18 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
US11618928B2 (en) 2017-04-12 2023-04-04 The Broad Institute, Inc. CRISPR effector system based diagnostics for malaria detection
CA3059757A1 (en) 2017-04-12 2018-10-18 Massachusetts Institute Of Technology Novel type vi crispr orthologs and systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
WO2018191673A1 (en) 2017-04-14 2018-10-18 Children's Hospital Medical Center Multi donor stem cell compositions and methods of making same
US11913015B2 (en) 2017-04-17 2024-02-27 University Of Maryland, College Park Embryonic cell cultures and methods of using the same
MX2019012398A (es) 2017-04-18 2020-09-25 Broad Inst Inc Composiciones para detectar secreciones y metodos de uso.
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
BR112019021719A2 (pt) 2017-04-21 2020-06-16 The General Hospital Corporation Variantes de cpf1 (cas12a) com especificidade para pam alterada
WO2018201090A1 (en) * 2017-04-28 2018-11-01 Exicure, Inc. Synthesis of spherical nucleic acids using lipophilic moieties
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
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
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
AU2018264636B2 (en) 2017-05-08 2022-03-17 Toolgen Incorporated Artificially manipulated immune cell
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
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3622062A4 (en) 2017-05-10 2020-10-14 The Regents of the University of California DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9
US12226479B2 (en) 2017-05-11 2025-02-18 The General Hospital Corporation Methods and compositions of use of CD8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
EP3622091A4 (en) * 2017-05-12 2021-02-17 President and Fellows of Harvard College UNIVERSAL EARLY CANCER DIAGNOSIS
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
EP3624942A1 (en) * 2017-05-16 2020-03-25 Wisconsin Alumni Research Foundation System and method for en masse patterning of molecule structures
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP7364472B2 (ja) 2017-05-18 2023-10-18 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化された核酸編集のための系、方法、及び組成物
HUE054164T2 (hu) 2017-05-22 2021-08-30 4D Pharma Res Ltd Baktériumtörzseket tartalmazó készítmények
WO2018215782A1 (en) 2017-05-24 2018-11-29 4D Pharma Research Limited Compositions comprising bacterial strain
EP3630970A4 (en) 2017-05-25 2020-12-30 The General Hospital Corporation BASIC EDITORS WITH IMPROVED PRECISION AND SPECIFICITY
US11603544B2 (en) 2017-06-05 2023-03-14 Fred Hutchinson Cancer Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
EP3635104A1 (en) 2017-06-09 2020-04-15 Editas Medicine, Inc. Engineered cas9 nucleases
MA49010B1 (fr) 2017-06-14 2020-11-30 4D Pharma Res Ltd Compositions comprenant une souche bactérienne du genre megasphaera et leurs utilisations
WO2018232195A1 (en) 2017-06-14 2018-12-20 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
LT3600363T (lt) 2017-06-14 2021-02-10 4D Pharma Research Limited Kompozicijos, apimančios bakterijų kamienus
SMT202000695T1 (it) 2017-06-14 2021-01-05 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
BR112019027710A2 (pt) 2017-06-23 2020-08-18 University Of Kentucky Research Foundation métodos de modulação do teor de alcaloides e do teor de uma nitrosamina específica do tabaco (tsna), uso de pelo menos um gene nic1 erf, método para produzir uma planta, planta modificada, material de propagação vegetal, uso de uma planta, folha colhida de uma planta, folha processada, material de tabaco curado, mistura de tabaco, produto da indústria do tabaco, produto de tabaco, uso de uma célula de tabaco, artigo de fumo combustível, uso de uma sequência de nucleotídeos, mutante de uma planta, progênie ou semente
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
RU2019143627A (ru) * 2017-06-28 2021-07-28 Юниверсити Оф Соус Флорида Модифицированный ген ube3a для генной терапии синдрома ангельмана
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019010422A1 (en) 2017-07-07 2019-01-10 The Broad Institute, Inc. ANTIVIRAL THERAPY BASED ON THE CRISPR SYSTEM
US12548638B2 (en) 2017-07-07 2026-02-10 The Broad Institute, Inc. Methods for designing guide sequences for guided nucleases
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
EP3654993A4 (en) 2017-07-17 2021-08-25 The Broad Institute, Inc. CELL ATLAS OF HEALTHY HUMAN COLUMN AND HUMAN COLUMN WITH COLITIS ULCEROSA
WO2019018694A1 (en) 2017-07-19 2019-01-24 Dana-Farber Cancer Institute, Inc. DIAGNOSIS AND TREATMENT OF CANCER
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
SG11201912235PA (en) * 2017-07-31 2020-01-30 Regeneron Pharma Cas-transgenic mouse embryonic stem cells and mice and uses thereof
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
EP3585160A2 (en) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Crispr reporter non-human animals and uses thereof
AU2018309714A1 (en) 2017-07-31 2020-01-30 Regeneron Pharmaceuticals, Inc. Assessment of CRISPR/Cas-induced recombination with an exogenous donor nucleic acid in vivo
EP3672612A4 (en) 2017-08-23 2021-09-29 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES WITH MODIFIED PAM SPECIFICITY
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019055169A1 (en) * 2017-09-15 2019-03-21 The Board Of Trustees Of The Leland Stanford Junior University MITOCHONDRIAL ALDEHYDE DEHYDROGENASE 2 MODULATORS FOR THE PROTECTION, EXPANSION AND INCREASE IN THE POWER OF HEMATOPOIETIC STEM CELLS
KR20200066616A (ko) 2017-09-21 2020-06-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법 및 조성물
JP7397488B2 (ja) 2017-09-22 2023-12-13 ユニバーシティ オブ マサチューセッツ Ｓｏｄ１二重発現ベクターおよびその使用
WO2019067452A1 (en) * 2017-09-27 2019-04-04 Pioneer Hi-Bred International, Inc. GROUND APPLICATION OF CROP PROTECTION AGENTS
JP2020535160A (ja) * 2017-09-28 2020-12-03 タンデム カンパニー，リミテッド 癌治療用の新規な組換え原形質膜をベースとする小胞体
ES2962277T3 (es) 2017-09-29 2024-03-18 Regeneron Pharma Roedores que comprenden un locus Ttr humanizado y métodos de uso
US12043870B2 (en) 2017-10-02 2024-07-23 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
CA3077086A1 (en) 2017-10-04 2019-04-11 President And Fellows Of Harvard College Systems, methods, and compositions for targeted nucleic acid editing
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
CA3076518A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. Crispr effector system based diagnostics
JP2020536529A (ja) 2017-10-10 2020-12-17 チルドレンズ ホスピタル メディカル センター 食道組織および／または臓器組成物およびそれを作製する方法
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
US11680296B2 (en) 2017-10-16 2023-06-20 Massachusetts Institute Of Technology Mycobacterium tuberculosis host-pathogen interaction
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CA3079681A1 (en) 2017-10-20 2019-04-25 Fred Hutchinson Cancer Research Center Systems and methods to produce b cells genetically modified to express selected antibodies
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
WO2019089804A1 (en) 2017-11-01 2019-05-09 The Regents Of The University Of California Casy compositions and methods of use
CA3081346A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH
US12171783B2 (en) 2017-11-13 2024-12-24 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in H3K27M gliomas
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
JP7423520B2 (ja) 2017-11-16 2024-01-29 アストラゼネカ・アクチエボラーグ Ｃａｓ９ベースノックイン方針の効力を改善するための組成物及び方法
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
US12521201B2 (en) 2017-12-07 2026-01-13 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
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126626A1 (en) 2017-12-21 2019-06-27 Children's Hospital Medical Center Digitalized human organoids and methods of using same
US20230193242A1 (en) 2017-12-22 2023-06-22 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
US20200362366A1 (en) 2018-01-12 2020-11-19 Basf Se Gene underlying the number of spikelets per spike qtl in wheat on chromosome 7a
ES2940786T3 (es) 2018-01-17 2023-05-11 Vertex Pharma Compuestos de quinoxalinona, composiciones, métodos y kits para aumentar la eficiencia de edición del genoma
BR112020014315A2 (pt) 2018-01-17 2020-12-08 Vertex Pharmaceuticals Incorporated Inibidores de dna-pk
EA202091709A1 (ru) 2018-01-17 2020-11-10 Вертекс Фармасьютикалз Инкорпорейтед Ингибиторы днк-пк
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
BR112020015093A2 (pt) 2018-01-29 2020-12-08 The Broad Institute, Inc. Diagnóstico baseado no sistema de efetor crispr
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
US20210032300A1 (en) * 2018-02-02 2021-02-04 The Penn State Research Foundation Methods and materials for treating brain injuries
US20210361693A1 (en) * 2018-02-09 2021-11-25 Empirico Inc. Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions
WO2019165168A1 (en) 2018-02-23 2019-08-29 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR101889683B1 (ko) * 2018-03-14 2018-08-17 유병탁 철골 구조 시공 방법
DK3765615T3 (da) 2018-03-14 2023-08-21 Arbor Biotechnologies Inc Nye enzymer og systemer til målretning af crispr dna
US12195770B2 (en) 2018-03-14 2025-01-14 Arbor Biotechnologies, Inc. CRISPR DNA and RNA targeting enzymes and systems
AU2019236204B2 (en) 2018-03-15 2026-02-26 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
KR20240038811A (ko) 2018-03-19 2024-03-25 리제너론 파마슈티칼스 인코포레이티드 CRISPR/Cas 시스템을 사용한 동물에서의 전사 조절
US20210155959A1 (en) 2018-04-06 2021-05-27 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
EP3560330B1 (en) 2018-04-24 2022-06-15 KWS SAAT SE & Co. KGaA Plants with improved digestibility and marker haplotypes
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
WO2019211741A1 (en) 2018-05-02 2019-11-07 Augmedics Ltd. Registration of a fiducial marker for an augmented reality system
CN112512595A (zh) * 2018-05-03 2021-03-16 哈佛学院校长同事会 心脏、骨骼肌和肌肉干细胞中的体内同源性定向修复
WO2019213660A2 (en) 2018-05-04 2019-11-07 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
KR20250134703A (ko) 2018-05-11 2025-09-11 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12522812B2 (en) 2018-05-16 2026-01-13 Arbor Biotechnologies, Inc. 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
CA3101066A1 (en) 2018-06-26 2020-01-02 The Broad Institute, Inc. Crispr/cas and transposase based amplification compositions, systems and methods
BR112020026246A2 (pt) 2018-06-26 2021-04-20 The Broad Institute Inc. composições, sistemas e métodos de amplificação com base em nickase dupla crispr
US20210269866A1 (en) 2018-06-26 2021-09-02 The Broad Institute, Inc. Crispr effector system based amplification methods, systems, and diagnostics
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
US12391928B2 (en) 2018-06-29 2025-08-19 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
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
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3105658A1 (en) 2018-07-13 2020-01-16 The Regents Of The University Of California Retrotransposon-based delivery vehicle and methods of use thereof
WO2020023529A1 (en) * 2018-07-24 2020-01-30 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
EP3826652B1 (en) 2018-07-26 2026-02-25 Children's Hospital Medical Center Hepato-biliary-pancreatic tissues and methods of making same
WO2020028555A2 (en) 2018-07-31 2020-02-06 The Broad Institute, Inc. Novel crispr enzymes and systems
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
JP2021533784A (ja) 2018-08-18 2021-12-09 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 インサイチュ遺伝子編集
US12460244B2 (en) 2018-08-20 2025-11-04 The Broad Institute, Inc. Inhibitors of RNA-guided nuclease activity and uses thereof
WO2020068304A2 (en) 2018-08-20 2020-04-02 The Broad Institute, Inc. Inhibitors of rna-guided nuclease target binding and uses thereof
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
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
WO2020051507A1 (en) 2018-09-06 2020-03-12 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP3849568A4 (en) 2018-09-12 2022-06-15 Children's Hospital Medical Center Organoid compositions for the production of hematopoietic stem cells and derivatives thereof
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
JP7344300B2 (ja) 2018-09-18 2023-09-13 ブイエヌブイ ニューコ インク． Ａｒｃベースのカプシドおよびその使用
US12467925B2 (en) 2018-10-03 2025-11-11 The Broad Institute, Inc. CRISPR effector system based diagnostics for hemorrhagic fever detection
WO2020077135A1 (en) 2018-10-10 2020-04-16 Dana-Farber Cancer Institute, Inc. Modulating resistance to bcl-2 inhibitors
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
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
WO2020081730A2 (en) 2018-10-16 2020-04-23 Massachusetts Institute Of Technology Methods and compositions for modulating microenvironment
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
WO2020092453A1 (en) 2018-10-29 2020-05-07 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
WO2020092163A1 (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
AU2019374883A1 (en) 2018-11-09 2021-05-20 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
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
CN111172192B (zh) * 2018-11-13 2024-04-23 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构建方法及其应用
US20220002789A1 (en) 2018-11-14 2022-01-06 The Broad Institute, Inc. Multiplexing highly evolving viral variants with sherlock detection method
AU2019379160A1 (en) 2018-11-14 2021-06-24 Massachusetts Institute Of Technology CRISPR system based droplet diagnostic systems and methods
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
US12258594B2 (en) 2018-12-05 2025-03-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof
AU2019392748A1 (en) * 2018-12-05 2021-06-10 Fred Hutchinson Cancer Center Reduced and minimal manipulation manufacturing of genetically-modified cells
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
WO2020124050A1 (en) 2018-12-13 2020-06-18 The Broad Institute, Inc. Tiled assays using crispr-cas based detection
US12365888B2 (en) 2018-12-14 2025-07-22 Pioneer Hi-Bred International, Inc. CRISPR-Cas systems for genome editing
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
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
US11690362B2 (en) 2018-12-20 2023-07-04 Regeneran 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)
US11116778B2 (en) 2019-01-15 2021-09-14 Empirico Inc. Prodrugs of ALOX-15 inhibitors and methods of using the same
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
GB201900940D0 (en) 2019-01-23 2019-03-13 British American Tobacco Investments Ltd Method
US12529092B2 (en) 2019-01-28 2026-01-20 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
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
US12460230B2 (en) 2019-02-10 2025-11-04 The J. David Gladstone Institutes Modified mitochondrion and methods of use thereof
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
CN119896751A (zh) * 2019-02-13 2025-04-29 比姆医疗股份有限公司 使用腺苷酸脱氨酶碱基编辑器破坏疾病相关基因的剪接受体位点，包括用于治疗遗传性疾病
US20230053540A1 (en) 2019-02-19 2023-02-23 Massachusetts Institute Of Technology Treatment of liver injury
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
EP3935156A4 (en) 2019-03-07 2022-12-28 The Regents of The University of California CRISPR-CAS EFFECTIVE 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
US20220154158A1 (en) 2019-03-12 2022-05-19 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Cas9 variants with enhanced specificity
WO2020186237A1 (en) 2019-03-13 2020-09-17 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
US20220154222A1 (en) 2019-03-14 2022-05-19 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2020186223A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Sherlock assays for tick-borne diseases
WO2020186231A2 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Crispr effector system based multiplex diagnostics
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020190932A1 (en) 2019-03-18 2020-09-24 Regeneron Pharmaceuticals, Inc. Crispr/cas screening platform to identify genetic modifiers of tau seeding or aggregation
WO2020191069A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Modulation of type 2 immunity by targeting clec-2 signaling
WO2020191079A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Compositions and methods for modulating metabolic regulators of t cell pathogenicity
SG11202108090XA (en) 2019-03-18 2021-08-30 Regeneron Pharma Crispr/cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
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
US20220162649A1 (en) 2019-04-01 2022-05-26 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2020206046A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Methods and compositions for cell therapy
AU2020256225B9 (en) 2019-04-03 2025-04-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
US11111504B2 (en) 2019-04-04 2021-09-07 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
WO2020206139A1 (en) 2019-04-04 2020-10-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
WO2020214842A1 (en) 2019-04-17 2020-10-22 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
AU2020268959B2 (en) 2019-05-03 2026-02-19 Specific Biologics Inc. Lipid-encapsulated dual-cleaving endonuclease for DNA and gene editing
US20230190819A1 (en) * 2019-05-11 2023-06-22 Youngsuk Yi Compositions and methods containing exosomes
UA128175C2 (uk) 2019-05-13 2024-04-24 Квс Саат Се Енд Ко. Кгаа Посухостійкість у кукурудзи
GB201906768D0 (en) 2019-05-14 2019-06-26 British American Tobacco Investments Ltd Method
US20220249701A1 (en) 2019-05-14 2022-08-11 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
WO2020236967A1 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Random crispr-cas deletion mutant
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
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
AU2020283048A1 (en) 2019-05-31 2021-12-23 Children's Hospital Medical Center Shaped organoid compositions and methods of making same
EP3976066A4 (en) 2019-05-31 2023-06-28 Children's Hospital Medical Center Methods of generating and expanding hematopoietic stem cells
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
EP3801011A1 (en) 2019-06-04 2021-04-14 Regeneron Pharmaceuticals, Inc. Non-human animals 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
AU2020289581B2 (en) 2019-06-07 2024-11-21 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
CA3137765A1 (en) 2019-06-14 2020-12-17 Regeneron Pharmaceuticals, Inc. Models of tauopathy
WO2020257514A1 (en) * 2019-06-21 2020-12-24 Children's Medical Center Corporation Methods and compositions for allele specific gene editing
SG11202111943UA (en) 2019-07-02 2021-11-29 Hutchinson Fred Cancer Res Recombinant ad35 vectors and related gene therapy improvements
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
CN114245803A (zh) 2019-08-06 2022-03-25 儿童医疗中心有限公司 用于重构小神经胶质细胞的方法和组合物
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
US20220282333A1 (en) 2019-08-13 2022-09-08 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
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
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
AU2020347276A1 (en) 2019-09-12 2022-03-17 Massachusetts Institute Of Technology Engineered adeno-associated virus capsids
CA3153980A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
US12264367B2 (en) 2019-09-19 2025-04-01 The Broad Institute, Inc. Methods of in vivo evaluation of gene function
WO2021055855A1 (en) 2019-09-20 2021-03-25 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
EP4031660A1 (en) 2019-09-20 2022-07-27 The Broad Institute, Inc. 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
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
US20230357788A1 (en) 2019-10-17 2023-11-09 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
US12195723B2 (en) 2019-11-08 2025-01-14 The Broad Institute, Inc. Engineered antigen presenting cells and uses thereof
AU2020379046B2 (en) 2019-11-08 2025-03-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy
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
US20240016735A1 (en) 2019-11-22 2024-01-18 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
US20230028178A1 (en) * 2019-12-23 2023-01-26 The Regents Of The University Of 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
CN115397974A (zh) 2020-01-23 2022-11-25 儿童医疗中心有限公司 由人类多能干细胞进行无基质t细胞分化
US12165747B2 (en) 2020-01-23 2024-12-10 The Broad Institute, Inc. Molecular spatial mapping of metastatic tumor microenvironment
US12312613B2 (en) 2020-01-24 2025-05-27 The General Hospital Corporation Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
WO2021155063A1 (en) 2020-01-29 2021-08-05 Readcoor, Llc Compositions and methods for analyte detection
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
BR112022017070A2 (pt) * 2020-02-28 2022-11-16 Huigene Therapeutics Co Ltd Sistema crispr-cas tipo vi-e e tipo vi-f e usos do mesmo
JP2023515671A (ja) 2020-03-04 2023-04-13 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 免疫療法に対する腫瘍細胞の感作のための方法及び組成物
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
WO2021195079A1 (en) 2020-03-23 2021-09-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US11639523B2 (en) 2020-03-23 2023-05-02 The Broad Institute, Inc. Type V CRISPR-Cas systems and use thereof
CN115698278B (zh) 2020-03-31 2025-10-17 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途
WO2021202938A1 (en) 2020-04-03 2021-10-07 Creyon Bio, Inc. Oligonucleotide-based machine learning
US20230159945A1 (en) 2020-04-09 2023-05-25 R.J. Reynolds Tobacco Company Method
US20230121577A1 (en) * 2020-04-15 2023-04-20 The Regents Of The University Of California Compositions and Methods for Treatment
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
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
WO2021224633A1 (en) 2020-05-06 2021-11-11 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230175015A1 (en) * 2020-05-12 2023-06-08 Myogene Bio Llc Immunosuppressive agents and viral delivery re-dosing methods 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
JP2023527446A (ja) 2020-05-29 2023-06-28 カー・ヴェー・エス ザート エス・エー ウント コー． カー・ゲー・アー・アー 植物単数体の誘導
EP4176434A4 (en) * 2020-05-29 2024-09-11 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)
US11389252B2 (en) 2020-06-15 2022-07-19 Augmedics Ltd. Rotating marker for image guided surgery
WO2022010286A1 (ko) * 2020-07-08 2022-01-13 경상국립대학교산학협력단 미세상동성 기반 말단 결합을 통한 유전자 교정에 이용되는 공여자 핵산 및 이의 용도
US20230321278A1 (en) * 2020-07-10 2023-10-12 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
EP4192486A4 (en) 2020-08-07 2024-05-01 The Broad Institute, Inc. THERAPEUTIC TARGETING OF PHOSPHATE DISREGULATION IN CANCER VIA THE XPR1 PROTEIN COMPLEX: KIDINS220
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
US12502163B2 (en) 2020-09-09 2025-12-23 Augmedics Ltd. Universal tool adapter for image-guided surgery
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
CN114250228A (zh) * 2020-09-22 2022-03-29 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
WO2022079083A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for va rna transcription
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)
WO2022120022A1 (en) 2020-12-02 2022-06-09 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cell lines and methods of use thereof
CN114796493B (zh) * 2021-01-28 2026-02-10 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
US20240309346A1 (en) 2021-02-11 2024-09-19 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
EP4337769A1 (en) 2021-05-10 2024-03-20 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
WO2022256437A1 (en) 2021-06-02 2022-12-08 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
CA3225121A1 (en) 2021-07-20 2023-01-26 Benjamin DEVERMAN Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
EP4373928A2 (en) 2021-07-22 2024-05-29 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
CA3221517A1 (en) 2021-07-30 2023-02-02 Monika KLOIBER-MAITZ 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
US12475662B2 (en) 2021-08-18 2025-11-18 Augmedics Ltd. Stereoscopic display and digital loupe for augmented-reality near-eye display
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
EP4596697A3 (en) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions and methods for nucleic acid modifications
JP2024538700A (ja) 2021-10-08 2024-10-23 プレジデント・アンド・フェロウズ・オブ・ハーバード・カレッジ 薬物送達のためのイオン液体
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
KR20240082391A (ko) 2021-10-14 2024-06-10 론자 세일즈 아게 세포외 소포 생산을 위한 변형된 생산자 세포
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
EP4423271A2 (en) 2021-10-28 2024-09-04 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
CA3237482A1 (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
KR20240117571A (ko) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
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
WO2023108430A1 (zh) * 2021-12-14 2023-06-22 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
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
EP4448549A2 (en) 2021-12-17 2024-10-23 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
AU2022420615A1 (en) 2021-12-22 2024-07-04 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
CA3241882A1 (en) 2021-12-29 2023-07-06 Bristol-Myers Squibb Company Generation of landing pad cell lines
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
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023150518A1 (en) 2022-02-01 2023-08-10 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
JP2025508677A (ja) 2022-02-02 2025-04-10 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 ポンペ病の処置のための抗ＴｆＲ：ＧＡＡ及び抗ＣＤ６３：ＧＡＡの挿入
EP4473097A1 (en) 2022-02-02 2024-12-11 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
US20250163392A1 (en) 2022-02-02 2025-05-22 Inscripta, Inc. Nucleic acid-guided nickase fusion proteins
US20250215443A1 (en) 2022-02-03 2025-07-03 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
WO2023148476A1 (en) 2022-02-03 2023-08-10 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
EP4472405A1 (en) 2022-02-04 2024-12-11 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
US20250161489A1 (en) 2022-02-28 2025-05-22 National University Corporation Tokai National Higher Education And Research System Medicine for disease caused by 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
CA3255225A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California COMPOSITIONS AND METHODS OF GENETIC COMPLEMENTATION
GB202205148D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205562D0 (en) 2022-04-14 2022-06-01 Nicoventures Trading Ltd Method
GB202205561D0 (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
GB202206109D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
GB202206107D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
EP4514981A2 (en) 2022-04-29 2025-03-05 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
EP4526324A1 (en) 2022-05-18 2025-03-26 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
CN119947735A (zh) 2022-05-19 2025-05-06 莱尔免疫制药公司 靶向nr4a3的多核苷酸及其用途
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
EP4532720A2 (en) 2022-05-31 2025-04-09 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
EP4544051A2 (en) 2022-06-24 2025-04-30 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
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
CA3262097A1 (en) 2022-07-14 2024-01-18 The Broad Institute, Inc. AAV capsids that allow gene distribution across the central nervous system via interactions with the transferrin receptor
AU2023311964A1 (en) 2022-07-18 2025-01-30 Renagade Therapeutics Management Inc. Gene editing components, systems, and methods of use
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4558626A1 (en) 2022-07-22 2025-05-28 Arbor Biotechnologies, Inc. Compositions comprising a variant polypeptide and uses thereof
WO2024020557A1 (en) 2022-07-22 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising a variant nuclease 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
AU2023314808A1 (en) 2022-07-29 2025-03-20 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
CA3261296A1 (en) 2022-08-05 2024-02-08 Regeneron Pharmaceuticals, Inc. TDP-43 VARIANTS RESISTANT TO AGGREGATION
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
US20260055146A1 (en) 2022-08-24 2026-02-26 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
US20250386788A1 (en) 2022-08-26 2025-12-25 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
IL319523A (en) 2022-09-13 2025-05-01 Augmedics Ltd Augmented reality glasses for image-guided medical intervention
WO2024064838A1 (en) 2022-09-21 2024-03-28 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
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
EP4602174A1 (en) 2022-10-13 2025-08-20 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
EP4612184A1 (en) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024107765A2 (en) 2022-11-14 2024-05-23 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
JP2026503704A (ja) 2023-01-27 2026-01-29 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 修飾されたラブドウイルス糖タンパク質およびその使用
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
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
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
CN121358859A (zh) * 2023-03-20 2026-01-16 维乎医疗有限公司 用于治疗心血管疾病的lpa基因的基于切口酶的体内编辑
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
EP4698665A1 (en) 2023-04-18 2026-02-25 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
EP4698666A1 (en) 2023-04-18 2026-02-25 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
US20240352485A1 (en) 2023-04-19 2024-10-24 Flagship Pioneering Innovations Vii, Llc Compositions and methods for the production of libraries
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
CN121712899A (zh) * 2023-04-27 2026-03-20 伦斯勒理工学院 用于在真核细胞中准确插入dna序列的重组酶
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
EP4713433A2 (en) 2023-05-18 2026-03-25 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
WO2024243340A1 (en) 2023-05-23 2024-11-28 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
KR20260026049A (ko) 2023-06-15 2026-02-25 리제너론 파아마슈티컬스, 인크. 청력 장애에 대한 유전자 치료
CN120500356A (zh) 2023-06-16 2025-08-15 益杰立科新加坡有限公司 靶向乙型肝炎病毒基因的表观编辑工具
WO2024263961A2 (en) 2023-06-23 2024-12-26 Children's Hospital Medical Center Methods of matrix-free suspension culture
TW202509220A (zh) 2023-06-26 2025-03-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 經工程化之瘧原蟲及相關方法
AU2024309884A1 (en) 2023-06-30 2025-12-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
AU2024317483A1 (en) 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
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
WO2025049788A1 (en) 2023-08-29 2025-03-06 The Broad Institute, Inc. Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish
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
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
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
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
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
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
US20250276092A1 (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
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
WO2025212920A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Multi-zonal liver organoids
WO2025211949A1 (ko) * 2024-04-05 2025-10-09 재단법인대구경북과학기술원 ＳｅｒｐｉｎＡ１ｅ 단백질을 유효성분으로 포함하는 기억력, 인지기능 개선용 또는 뇌신경질환의 예방 또는 치료용 조성물
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
US20250388890A1 (en) 2024-06-20 2025-12-25 Regeneron Pharmaceuticals, Inc. ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome
WO2026047626A1 (en) 2024-08-30 2026-03-05 Inceptor Bio, Llc Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy
CN119570025A (zh) * 2024-11-19 2025-03-07 中国药科大学 一种聚乙烯亚胺衍生物及其作为基因药物载体应用

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 (enExample) 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
AU7979491A (en) 1990-05-03 1991-11-27 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
AU752704B2 (en) 1997-10-24 2002-09-26 Invitrogen Corporation Recombinational cloning using nucleic acids having recombination sites
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 (enExample) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
ES2404689T3 (es) 2001-04-05 2013-05-28 The Johns Hopkins University Vacunas quiméricas que comprenden el dominio lumenal de LAMP-1 o LAMP-2
IL159756A0 (en) 2001-07-12 2004-06-20 Univ Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
US20040002468A1 (en) 2001-08-08 2004-01-01 Genzyme Corporation Methods of 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
AU2002336760A1 (en) 2001-09-26 2003-06-10 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
US7539579B2 (en) 2002-04-09 2009-05-26 Beattie Kenneth L Oligonucleotide probes for genosensor chips
WO2003104414A2 (en) 2002-06-11 2003-12-18 The Scripps Research Institute Artificial transcription factors
US7867193B2 (en) 2004-01-29 2011-01-11 The Charles Stark Draper Laboratory, Inc. Drug delivery apparatus
DE60334618D1 (de) 2002-06-28 2010-12-02 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
CA2499188A1 (en) 2002-09-27 2004-04-08 Cold Spring Harbor Laboratory Cell-based rna interference and related methods and compositions
WO2004046321A2 (en) * 2002-11-15 2004-06-03 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
CA2530810A1 (en) 2003-07-03 2005-01-27 The Regents Of The University Of California Genome mapping of functional dna elements and cellular proteins
CN101291653B (zh) 2003-07-16 2012-06-27 普洛体维生物治疗公司 脂质包封的干扰rna
ES2808687T3 (es) 2003-08-08 2021-03-01 Sangamo Therapeutics Inc Métodos y composiciones para escisión dirigida y recombinación
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
KR101164256B1 (ko) 2003-09-15 2012-07-10 프로티바 바이오쎄라퓨틱스, 인코포레이티드 폴리에틸렌글리콜 개질된 지질 화합물 및 그의 용도
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
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
EP1784416B1 (en) 2004-07-16 2011-10-05 GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Vaccines against aids comprising cmv/r nucleic acid constructs
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
US20070081982A1 (en) * 2005-04-28 2007-04-12 Elisabeth Evertsz 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
CA2619833C (en) 2005-08-26 2017-05-09 Danisco A/S Use of crispr associated genes (cas)
WO2007030743A2 (en) 2005-09-08 2007-03-15 Massachusetts Eye & Ear Infirmary Cochlear implants containing biological cells and uses thereof
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
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
EP2194128B1 (en) 2006-05-11 2012-08-01 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. 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
BRPI0718747A2 (pt) 2006-11-14 2013-12-03 Cellectis Variantes meganuclease que clavam uma ou sequência alvo de dna a partir do gene hprt e usos das mesmas.
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
ES2719789T3 (es) 2007-03-02 2019-07-16 Dupont Nutrition Biosci Aps Cultivos con resistencia mejorada a fagos
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
US20100293703A1 (en) 2007-05-29 2010-11-25 Celine Tezartes-Strauss Garment with built-in protecting pads
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
JP5588344B2 (ja) 2007-08-14 2014-09-10 フレッド ハッチンソン キャンサー リサーチ センター 治療薬をデリバリーするための針アレイアセンブリ及び方法
US8404658B2 (en) 2007-12-31 2013-03-26 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
AU2009212135B2 (en) 2008-02-07 2014-08-21 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
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US20090280262A1 (en) 2008-05-08 2009-11-12 Chung Yuan Christian University Method for forming composite membrane with porous coating layer and apparatus thereof
US8431692B2 (en) 2008-06-06 2013-04-30 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
CN102137658A (zh) 2008-06-30 2011-07-27 斯兰斯德有限公司 局部递送药物的方法、组合物和系统
WO2010004594A1 (en) 2008-07-08 2010-01-14 S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. Ophthalmic compositions for treating pathologies of the posterior segment of the eye
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US9255130B2 (en) 2008-07-29 2016-02-09 Academia Sinica Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
CA2736336A1 (en) 2008-09-08 2010-03-11 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof
DK2334794T3 (en) 2008-09-15 2017-02-20 Children's Medical Center Corp MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
SG10201809460SA (en) 2008-10-20 2018-11-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin
CN102264915B (zh) 2008-10-29 2014-04-16 桑格摩生物科学股份有限公司 失活谷氨酰胺合成酶基因表达的方法和组合物
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
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
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
CA2746514C (en) 2008-12-10 2018-11-27 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
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
AU2010226313B2 (en) 2009-03-20 2014-10-09 Sangamo Therapeutics, Inc. Modification of CXCR4 using engineered zinc finger proteins
AR076225A1 (es) 2009-04-07 2011-05-26 Dow Agrosciences Llc Administracion de nanoparticulas revestidas de una secuencia de nucleasa especifica
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
US20120149115A1 (en) 2009-06-11 2012-06-14 Snu R&Db Foundation Targeted genomic rearrangements using site-specific nucleases
WO2010147987A1 (en) 2009-06-15 2010-12-23 Agc Automotive Amerlcas R&D, Inc. Diversity antenna system and method
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
WO2011000108A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
US9234016B2 (en) 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
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
EP3456826B1 (en) 2009-12-10 2023-06-28 Regents of the University of Minnesota Tal effector-mediated dna modification
JP2013515697A (ja) 2009-12-23 2013-05-09 マックスプランク−ゲセルシャフト・ツール・フェーデルング・デル・ヴィッセンシャフテン・エー・ファウ インフルエンザ標的
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, 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
KR101880536B1 (ko) 2010-04-26 2018-07-23 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
BR112012028805A2 (pt) 2010-05-10 2019-09-24 The Regents Of The Univ Of California E Nereus Pharmaceuticals Inc composições de endorribonuclease e métodos de uso das mesmas.
EP2569435A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
WO2011141820A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2575894B1 (en) 2010-05-28 2015-02-25 Oxford Biomedica (UK) Ltd Delivery of lentiviral vectors to the brain
JP2013537410A (ja) 2010-07-23 2013-10-03 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー 標的化エンドヌクレアーゼおよび一本鎖核酸を用いたゲノム編集
WO2012018697A1 (en) 2010-08-02 2012-02-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
CN103270046B (zh) 2010-10-12 2016-05-11 费城儿童医院 治疗b型血友病的方法和组合物
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
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
DK4005603T3 (da) 2011-04-22 2024-11-25 Univ California Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf
WO2012149470A1 (en) 2011-04-27 2012-11-01 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
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
US20130171732A1 (en) 2011-10-06 2013-07-04 Sangamo Biosciences, Inc. Methods and compositions for regulating hiv infection
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
US20130122096A1 (en) 2011-11-14 2013-05-16 Silenseed Ltd. Compositions for drug delivery and methods of manufacturing and using same
US9708414B2 (en) 2011-11-18 2017-07-18 UNIVERSITé LAVAL Methods and products for increasing frataxin levels and uses thereof
HK1202434A1 (en) 2011-11-22 2015-10-02 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
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
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN104284669A (zh) 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
EP2834259A4 (en) 2012-04-02 2016-08-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
PL4289948T3 (pl) * 2012-05-25 2025-06-02 The Regents Of The University Of California Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji
HK1208823A1 (en) 2012-06-01 2016-03-18 德雷塞尔大学 Modulation of hepatitis b virus cccdna transcription
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
AU2013329186B2 (en) 2012-10-10 2019-02-14 Sangamo Therapeutics, 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
PL2784162T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Opracowanie systemów, metod oraz zoptymalizowanych kompozycji przewodnikowych do manipulacji sekwencyjnej
WO2014093694A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN105121648B (zh) 2012-12-12 2021-05-07 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
PT2898075E (pt) * 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CN105658796B (zh) * 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
AU2014207618A1 (en) 2013-01-16 2015-08-06 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
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
IL289396B2 (en) 2013-03-15 2023-12-01 The General Hospital Coporation Using truncated guide rnas (tru-grnas) to increase specificity for rna-guided genome editing
JP6214753B2 (ja) 2013-03-27 2017-10-18 ヴィルコ・アーゲー デバイスをインラインで検査しかつ／または試験する方法、およびそのような方法を遂行するための装置
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
JP7065564B2 (ja) 2013-05-29 2022-05-12 セレクティス Ｃａｓ９ニッカーゼ活性を用いて正確なｄｎａ切断をもたらすための方法
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
MY197877A (en) 2013-06-04 2023-07-22 Harvard College Rna-guided transcriptional regulation
KR102307280B1 (ko) 2013-06-05 2021-10-01 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
KR20240172759A (ko) 2013-06-17 2024-12-10 더 브로드 인스티튜트, 인코퍼레이티드 간의 표적화 및 치료를 위한 ＣＲＩＳＰＲ­Ｃａｓ 시스템, 벡터 및 조성물의 전달 및 용도
EP3597755A1 (en) 2013-06-17 2020-01-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CA2915837A1 (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
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
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
EP3038661B1 (en) 2013-08-29 2023-12-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
WO2015071474A2 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
WO2015089354A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
EP3080260B1 (en) 2013-12-12 2019-03-06 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 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
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
WO2015113063A1 (en) 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
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
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
WO2016022866A1 (en) 2014-08-07 2016-02-11 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
US12180263B2 (en) 2014-11-06 2024-12-31 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
PL3291679T3 (pl) 2015-05-06 2022-04-25 Snipr Technologies Limited Zmiana populacji drobnoustrojowych i modyfikowanie mikrobioty
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
MX392008B (es) 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
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
KR20210143718A (ko) 2019-01-17 2021-11-29 조지아 테크 리서치 코포레이션 산화된 콜레스테롤을 함유하는 약물 전달 시스템
US12569438B2 (en) 2019-07-29 2026-03-10 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 (50)

* 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
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
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
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
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
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
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
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
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
US12441995B2 (en) 2013-06-17 2025-10-14 The Broad Institute, Inc. Functional genomics using CRISPR-Cas systems, compositions, methods, screens and applications thereof
US12553065B2 (en) 2013-06-17 2026-02-17 The Broad Institute, Inc. Optimized CRISPR-CAS double nickase systems, methods and compositions for sequence manipulation
US11597949B2 (en) 2013-06-17 2023-03-07 The Broad Institute, Inc. Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation
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
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
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
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
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
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
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
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
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
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
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
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
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
US12571005B2 (en) 2014-12-12 2026-03-10 The Broad Institute, Inc. Protected guide RNAs (pgRNAs)
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
US12123032B2 (en) 2015-06-18 2024-10-22 The Broad Institute, Inc. CRISPR enzyme mutations reducing off-target effects
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
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
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
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
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
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
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
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
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
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
CN105164264B (zh) 2021-05-07
EP2931897A2 (en) 2015-10-21
WO2014093622A2 (en) 2014-06-19
JP2023073245A (ja) 2023-05-25
IL239317A0 (en) 2015-07-30
JP2022040308A (ja) 2022-03-10
US20210269831A1 (en) 2021-09-02
WO2014093622A3 (en) 2014-08-07
JP7013406B2 (ja) 2022-02-15
US20140242699A1 (en) 2014-08-28
EP4549566A3 (en) 2025-07-09
EP3031921A1 (en) 2016-06-15
US11041173B2 (en) 2021-06-22
US20170152528A1 (en) 2017-06-01
AU2013359199C1 (en) 2021-06-17
AU2025203782A1 (en) 2025-06-12
US12252707B2 (en) 2025-03-18
CN110872583A (zh) 2020-03-10
DK2931897T3 (en) 2018-02-05
US20250382642A1 (en) 2025-12-18
US20140179770A1 (en) 2014-06-26
IL293526A (en) 2022-08-01
IL239317B (en) 2022-07-01
EP3327127A1 (en) 2018-05-30
US20170107536A1 (en) 2017-04-20
AU2013359199B2 (en) 2019-10-31
EP3327127B1 (en) 2021-03-24
SG10201912328UA (en) 2020-02-27
EP4299741A3 (en) 2024-02-28
MX2015007550A (es) 2017-02-02
WO2014093622A8 (en) 2014-12-04
KR20150105956A (ko) 2015-09-18
EP2931897B1 (en) 2017-11-01
CA2894681A1 (en) 2014-06-19
MX2022001378A (es) 2022-06-23
SG10201707569YA (en) 2017-10-30
ES2658401T3 (es) 2018-03-09
EP3031921B1 (en) 2025-03-12
AU2013359199A1 (en) 2015-07-30
HK1252619A1 (en) 2019-05-31
SG11201504523UA (en) 2015-07-30
AU2025203782A9 (en) 2026-03-19
AU2019283878A1 (en) 2020-01-23
HK1220725A1 (en) 2017-05-12
CN105164264A (zh) 2015-12-16
HK1215048A1 (en) 2016-08-12
DK3327127T3 (da) 2021-06-28
US20250250589A1 (en) 2025-08-07
JP7228059B2 (ja) 2023-02-22
JP2025181841A (ja) 2025-12-11
JP2019103513A (ja) 2019-06-27
AU2022203759B2 (en) 2025-02-27
JP7736729B2 (ja) 2025-09-09
MX389793B (es) 2025-03-20
ES2883590T3 (es) 2021-12-09
BR112015013784A2 (pt) 2017-07-11
ZA201504132B (en) 2022-03-30
WO2014093622A9 (en) 2014-11-06
RU2015128057A (ru) 2019-03-27
AU2022203759A1 (en) 2022-06-23
EP4549566A2 (en) 2025-05-07
AU2019283878B2 (en) 2022-06-09
RU2721275C2 (ru) 2020-05-18
US20150020223A1 (en) 2015-01-15
US20230399662A1 (en) 2023-12-14
US20240035048A1 (en) 2024-02-01
EP4299741A2 (en) 2024-01-03

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
BR122021007860B1 (pt) Método in vitro ou ex vivo de modificação de uma célula eucariótica isolada por manipulação de uma sequência alvo em um local genômico de interesse
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