DK3327127T3 - Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser - Google Patents

Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser Download PDF

Info

Publication number
DK3327127T3
DK3327127T3 DK17199166.4T DK17199166T DK3327127T3 DK 3327127 T3 DK3327127 T3 DK 3327127T3 DK 17199166 T DK17199166 T DK 17199166T DK 3327127 T3 DK3327127 T3 DK 3327127T3
Authority
DK
Denmark
Prior art keywords
optimization
procedures
compositions
modification
systems
Prior art date
Application number
DK17199166.4T
Other languages
Danish (da)
English (en)
Inventor
Feng Zhang
Matthias Heidenreich
Fei Ran
Lukasz Swiech
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Harvard College
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=DK3327127(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Broad Inst Inc, Massachusetts Inst Technology, Harvard College filed Critical Broad Inst Inc
Application granted granted Critical
Publication of DK3327127T3 publication Critical patent/DK3327127T3/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034Isolating an individual clone by screening libraries
    • C12N15/1082Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/82Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8213Targeted insertion of genes into the plant genome by homologous recombination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/20Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/30Detection of binding sites or motifs
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/50Mutagenesis
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10Sequence alignment; Homology search
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/072Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00ICT 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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Medical Informatics (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Theoretical Computer Science (AREA)
  • Evolutionary Biology (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Oncology (AREA)
DK17199166.4T 2012-12-12 2013-12-12 Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser DK3327127T3 (da)

Applications Claiming Priority (14)

Application Number Priority Date Filing Date Title
US201261736527P 2012-12-12 2012-12-12
US201361748427P 2013-01-02 2013-01-02
US201361758468P 2013-01-30 2013-01-30
US201361769046P 2013-02-25 2013-02-25
US201361802174P 2013-03-15 2013-03-15
US201361791409P 2013-03-15 2013-03-15
US201361806375P 2013-03-28 2013-03-28
US201361814263P 2013-04-20 2013-04-20
US201361819803P 2013-05-06 2013-05-06
US201361828130P 2013-05-28 2013-05-28
US201361835931P 2013-06-17 2013-06-17
US201361836123P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
EP13814362.3A EP2931897B1 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Publications (1)

Publication Number Publication Date
DK3327127T3 true DK3327127T3 (da) 2021-06-28

Family

ID=49883289

Family Applications (2)

Application Number Title Priority Date Filing Date
DK17199166.4T DK3327127T3 (da) 2012-12-12 2013-12-12 Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser
DK13814362.3T DK2931897T3 (en) 2012-12-12 2013-12-12 CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK13814362.3T DK2931897T3 (en) 2012-12-12 2013-12-12 CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS

Country Status (15)

Country Link
US (10) US20140179770A1 (enExample)
EP (5) EP3327127B1 (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) ES2658401T3 (enExample)
IL (2) IL293526A (enExample)
MX (2) MX389793B (enExample)
RU (1) RU2721275C2 (enExample)
SG (3) SG10201707569YA (enExample)
WO (1) WO2014093622A2 (enExample)
ZA (1) ZA201504132B (enExample)

Families Citing this family (900)

* 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
PT2564695E (pt) 2009-07-08 2015-06-03 Kymab Ltd Modelos animais e moléculas terapêuticas
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
EP2569430B1 (en) 2010-05-12 2018-10-17 The Trustees of Columbia University in the City of New York Methods for producing enteroendocrine cells that make and secrete insulin
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
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP3839049A3 (en) 2011-09-19 2021-10-20 Kymab Limited Antibodies, variable domains & chains tailored for human use
EP2761008A1 (en) 2011-09-26 2014-08-06 Kymab Limited Chimaeric surrogate light chains (slc) comprising human vpreb
GB201117313D0 (en) 2011-10-07 2011-11-16 Gt Biolog Ltd Bacterium for use in medicine
JP2014531908A (ja) 2011-10-14 2014-12-04 プレジデント アンド フェローズ オブ ハーバード カレッジ 構造アッセンブリによる配列決定
US9253965B2 (en) 2012-03-28 2016-02-09 Kymab Limited Animal models and therapeutic molecules
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
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
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
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
US9834786B2 (en) 2012-04-25 2017-12-05 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
WO2013169382A1 (en) * 2012-05-07 2013-11-14 DePuy Synthes Products, LLC Methods and devices for treating intervertebral disc disease
US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014011237A1 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
CN110669758A (zh) 2012-10-23 2020-01-10 基因工具股份有限公司 用于切割靶dna 的组合物及其用途
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
EP4234696A3 (en) * 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
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
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
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
AU2013359212B2 (en) 2012-12-12 2017-01-19 Massachusetts Institute Of Technology Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2940140B1 (en) 2012-12-12 2019-03-27 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
EP2931891B1 (en) * 2012-12-17 2019-05-15 President and Fellows of Harvard College Rna-guided human genome engineering
CA2898184A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
EP2971184B1 (en) 2013-03-12 2019-04-17 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
DK3620534T3 (da) * 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
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
EP2971165A4 (en) 2013-03-15 2016-11-23 Moderna Therapeutics Inc DISSOLUTION OF DNA FRAGMENTS IN MRNA MANUFACTURING METHODS
KR102271291B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
EP3578652B1 (en) 2013-03-15 2023-07-12 ModernaTX, Inc. Ribonucleic acid purification
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
WO2014165825A2 (en) * 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6576904B2 (ja) * 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法
GB201306536D0 (en) 2013-04-10 2013-05-22 Gt Biolog Ltd Polypeptide and immune modulation
LT2986729T (lt) 2013-04-16 2018-10-25 Regeneron Pharmaceuticals, Inc. Žiurkės genomo tikslinė modifikacija
US9783618B2 (en) 2013-05-01 2017-10-10 Kymab Limited Manipulation of immunoglobulin gene diversity and multi-antibody therapeutics
US11707056B2 (en) 2013-05-02 2023-07-25 Kymab Limited Animals, repertoires and methods
US9783593B2 (en) 2013-05-02 2017-10-10 Kymab Limited Antibodies, variable domains and chains tailored for human use
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝的状態の処置のための方法および組成物
WO2014191518A1 (en) * 2013-05-29 2014-12-04 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
US9267135B2 (en) * 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
AU2014274939B2 (en) 2013-06-04 2020-03-19 President And Fellows Of Harvard College RNA-guideded transcriptional regulation
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204729A1 (en) * 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
BR112015031608A2 (pt) * 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
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
BR112016000571B1 (pt) * 2013-07-10 2023-12-26 President And Fellows Of Harvard College Métodos in vitro para modular a expressão e para alterar um ou mais ácidos nucleicos alvo em uma célula simultaneamente com a regulação da expressão de um ou mais ácidos nucleicos alvo em uma célula, bem como célula de levedura ou bactéria compreendendo ácidos nucleicos
SI3019619T1 (sl) * 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
US9663782B2 (en) * 2013-07-19 2017-05-30 Larix Bioscience Llc Methods and compositions for producing double allele knock outs
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
BR112016003591A8 (pt) 2013-08-22 2018-01-30 Du Pont promotor u6 de polimerase iii de soja e métodos de uso
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
KR20160060659A (ko) * 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
ES3013744T3 (en) * 2013-09-18 2025-04-15 Kymab Ltd Methods, cells and 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
EP3066201B1 (en) 2013-11-07 2018-03-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
CA2930877A1 (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
LT3080279T (lt) 2013-12-11 2018-10-25 Regeneron Pharmaceuticals, Inc. Būdai ir kompozicijos, skirti genomo tikslinei modifikacijai
CN110951779B (zh) 2013-12-11 2024-04-16 瑞泽恩制药公司 用于靶向修饰基因组的方法和组合物
EP3080261B1 (en) 2013-12-12 2019-05-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
MX374529B (es) 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
ES2787198T3 (es) * 2014-01-31 2020-10-15 Factor Bioscience Inc ARN sintético para su uso en el tratamiento de la epidermólisis ampollosa distrófica
DE212015000061U1 (de) 2014-02-11 2017-09-03 The Regents Of The University Of Colorado, A Body Corporate CRISPR-ermöglichtes Multiplex Genom Engineering
CA2940084A1 (en) 2014-02-18 2015-08-27 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
AU2015222944A1 (en) 2014-02-27 2016-09-08 Massachusetts Institute Of Technology T cell balance gene expression, compositions of matters and methods of use thereof
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) * 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
CA2947270A1 (en) * 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic acids targeting mdm2 or mycn
WO2015168800A1 (en) * 2014-05-09 2015-11-12 UNIVERSITé LAVAL Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
EP3147277B1 (en) * 2014-05-20 2023-10-04 National University Corporation Hokkaido University Lipid membrane structure for sirna intracellular delivery
EP3149156B1 (en) 2014-05-28 2021-02-17 Children's Hospital Medical Center Methods and systems for converting precursor cells into gastric tissues through directed differentiation
WO2015184268A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
SG11201609634YA (en) 2014-06-06 2016-12-29 Regeneron Pharma Methods and compositions for modifying a targeted locus
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
US9738897B2 (en) 2014-06-23 2017-08-22 Regeneron Pharmaceuticals, Inc. Nuclease-mediated DNA assembly
JP6784601B2 (ja) 2014-06-23 2020-11-11 ザ ジェネラル ホスピタル コーポレイション シークエンシングによって評価されるゲノムワイドでバイアスのないDSBの同定(GUIDE−Seq)
PL3161128T3 (pl) 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do celowanych modyfikacji genetycznych i sposoby zastosowania
EP3160503B1 (en) 2014-06-26 2021-02-17 The Trustees of Columbia University in the City of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
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
BR112017000621B1 (pt) * 2014-07-11 2024-03-12 Pioneer Hi-Bred International, Inc Método para melhorar um traço agronômico de uma planta de milho ou de soja
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
CA2954626A1 (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
CA2955238A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Circular polynucleotides
WO2016022363A2 (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
WO2016025469A1 (en) * 2014-08-11 2016-02-18 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
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
EP3180426B1 (en) 2014-08-17 2019-12-25 The Broad Institute, Inc. Genome editing using cas9 nickases
US9970030B2 (en) 2014-08-27 2018-05-15 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
US11560568B2 (en) 2014-09-12 2023-01-24 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CN104293831A (zh) * 2014-09-28 2015-01-21 上海云舜生物技术有限公司 建立高血压小鼠模型的方法及其用途
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016057850A1 (en) * 2014-10-09 2016-04-14 Seattle Children' S Hospital (Dba Seattle Children' S Research Institute) Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid
CA2964114A1 (en) 2014-10-09 2016-04-14 Anthrogenesis Corporation Placenta-derived adherent cell exosomes and uses thereof
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
CA2963693A1 (en) 2014-10-10 2016-04-14 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
JP6804438B2 (ja) 2014-10-17 2020-12-23 チルドレンズ ホスピタル メディカル センター 多能性幹細胞を使用するヒト小腸のin vivoモデル、並びにそれを作製、及び使用する方法
EP3207131B1 (en) * 2014-10-17 2022-09-28 Howard Hughes Medical Institute Genomic probes
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
WO2016069912A1 (en) 2014-10-29 2016-05-06 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
TWI716367B (zh) * 2014-10-31 2021-01-21 麻省理工學院 用於常間回文重複序列叢集(crispr)之大量平行組合性基因學
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
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
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
KR20230169197A (ko) 2014-11-14 2023-12-15 보이저 테라퓨틱스, 인크. 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
CA3176380A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
EP3224381B1 (en) 2014-11-25 2019-09-04 The Brigham and Women's Hospital, Inc. Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease
EP3224362B1 (en) 2014-11-26 2024-09-25 The Regents of The University of California Therapeutic compositions comprising transcription factors and methods of making and using the same
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
JP6830437B2 (ja) 2014-12-10 2021-02-17 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 疾患を処置するための遺伝子改変された細胞、組織および臓器
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
EP3653048B9 (en) 2014-12-19 2023-10-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification through single-step multiple targeting
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
WO2016100857A1 (en) 2014-12-19 2016-06-23 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
PT3065748T (pt) 2014-12-23 2018-02-28 4D Pharma Res Ltd Uma estirpe de bacteroides thetaiotaomicron e o seu uso na redução da inflamação
BR112017013274A2 (pt) 2014-12-23 2018-02-06 4D Pharma Research Limited polipeptídeo e imunomodulação
WO2016106236A1 (en) * 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
US11339399B2 (en) 2014-12-31 2022-05-24 Viridos, Inc. Compositions and methods for high efficiency in vivo genome editing
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
RU2713328C2 (ru) 2015-01-28 2020-02-04 Пайонир Хай-Бред Интернэшнл, Инк. Гибридные днк/рнк-полинуклеотиды crispr и способы применения
WO2016123243A1 (en) 2015-01-28 2016-08-04 The Regents Of The University Of California Methods and compositions for labeling a single-stranded target nucleic acid
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
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
US9944912B2 (en) 2015-03-03 2018-04-17 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
WO2016154176A1 (en) * 2015-03-26 2016-09-29 Yale University Methods and compositions for modifying endothelial cells
CN107567499A (zh) 2015-03-27 2018-01-09 纳幕尔杜邦公司 大豆u6核小rna基因启动子及其在植物小rna基因的组成型表达中的用途
EP3300507A4 (en) * 2015-04-02 2019-03-13 Agenovir Corporation GENERIC ADMINISTRATION AND COMPOSITIONS
EP4335918A3 (en) * 2015-04-03 2024-04-17 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
AU2016246450B2 (en) 2015-04-06 2022-03-17 Agilent Technologies, Inc. Chemically modified guide RNAs for CRISPR/Cas-mediated gene regulation
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
WO2016182893A1 (en) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Functional genomics using crispr-cas systems for saturating mutagenesis of non-coding elements, compositions, methods, libraries and applications thereof
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
EP3294896A1 (en) 2015-05-11 2018-03-21 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
WO2016196282A1 (en) * 2015-05-29 2016-12-08 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
EP3302709B1 (en) 2015-06-01 2021-05-19 Temple University - Of The Commonwealth System of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US10883103B2 (en) 2015-06-02 2021-01-05 Monsanto Technology Llc Compositions and methods for delivery of a polynucleotide into a plant
EP3303634B1 (en) 2015-06-03 2023-08-30 The Regents of The University of California Cas9 variants and methods of use thereof
GB201509578D0 (en) * 2015-06-03 2015-07-15 Univ Singapore Vectors
WO2016196887A1 (en) 2015-06-03 2016-12-08 Board Of Regents Of The University Of Nebraska Dna editing using single-stranded dna
KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
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
SMT201900588T1 (it) 2015-06-15 2019-11-13 4D Pharma Res Limited Blautia stercoris e wexlerae per uso nel trattamento di malattie infiammatorie ed autoimmuni
PT3360559T (pt) 2015-06-15 2020-01-06 4D Pharma Res Ltd Composições compreendendo estirpes bacterianas
PL3307288T3 (pl) 2015-06-15 2019-12-31 4D Pharma Research Limited Kompozycje zawierające szczepy bakteryjne
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
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
US9790490B2 (en) 2015-06-18 2017-10-17 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
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
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
JP6232689B2 (ja) 2015-06-25 2017-11-22 株式会社国際電気通信基礎技術研究所 多器官連関システムを基盤とした予測装置、及び予測プログラム
US20180171302A1 (en) * 2015-06-26 2018-06-21 Domenico ACCILI Genetically Modified IPS Cells That Carry a Marker to Report Expression of Neurogenin3, TPH2, FOXO1 and/or Insulin Genes
EP4545544A3 (en) 2015-06-29 2025-10-08 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
EP4273782A3 (en) * 2015-07-25 2023-12-27 Frost, Habib A system, device and a method for providing a therapy or a cure for cancer and other pathological states
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
JP6937740B2 (ja) * 2015-07-28 2021-09-22 ダニスコ・ユーエス・インク ゲノム編集システムおよび使用方法
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
CA2993431A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Nuclease based knockouts of immunological checkpoint genes in immune cells
EP4512819A3 (en) 2015-08-12 2025-08-06 The General Hospital Corporation Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders
US10709726B2 (en) 2015-08-14 2020-07-14 The University Of Sydney Connexin 45 inhibition for therapy
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
JP6905755B2 (ja) * 2015-08-25 2021-07-21 デューク ユニバーシティ Rnaガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
JP6799586B2 (ja) 2015-08-28 2020-12-16 ザ ジェネラル ホスピタル コーポレイション 遺伝子操作CRISPR−Cas9ヌクレアーゼ
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
KR20180043369A (ko) 2015-09-11 2018-04-27 더 제너럴 하스피탈 코포레이션 뉴클레아제 dsb의 완전한 호출 및 시퀀싱(find-seq)
AU2016324463B2 (en) 2015-09-17 2022-10-27 Modernatx, Inc. Polynucleotides containing a stabilizing tail region
CA2999500A1 (en) 2015-09-24 2017-03-30 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)
US10760081B2 (en) 2015-10-07 2020-09-01 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
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
ES2978086T3 (es) 2015-10-22 2024-09-05 Univ Massachusetts Terapia génica con aspartoacilasa en el tratamiento de enfermedad de Canavan
CA3002980A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
AU2016342038B2 (en) 2015-10-22 2022-09-08 Massachusetts Institute Of Technology Type VI-B CRISPR enzymes and systems
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
EP3368687B1 (en) 2015-10-27 2021-09-29 The Broad Institute, Inc. Compositions and methods for targeting cancer-specific sequence variations
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
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
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
US11092607B2 (en) 2015-10-28 2021-08-17 The Board Institute, Inc. Multiplex analysis of single cell constituents
GB2559526B (en) 2015-11-03 2021-02-17 Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
WO2017083274A1 (en) * 2015-11-09 2017-05-18 Seattle Children's Hospital (dba Seattle Children's Research Institute) Novel rna-based vector system for transient and stable gene expression
EP3374494A4 (en) * 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY
EP3374502B1 (en) 2015-11-13 2021-10-27 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
WO2017087395A1 (en) 2015-11-16 2017-05-26 Research Institute At Nationwide Children's Hospital Materials and methods for treatment of titin-based myopathies and other titinopaties
US11905521B2 (en) 2015-11-17 2024-02-20 The Chinese University Of Hong Kong Methods and systems for targeted gene manipulation
US11001622B2 (en) 2015-11-19 2021-05-11 The Brigham And Women's Hospital, Inc. Method of treating autoimmune disease with lymphocyte antigen CD5-like (CD5L) protein
HUE036362T2 (hu) 2015-11-20 2018-07-30 4D Pharma Res Ltd Baktériumtörzseket tartalmazó készítmények
GB201520497D0 (en) 2015-11-20 2016-01-06 4D Pharma Res Ltd Compositions comprising bacterial strains
SG10202004557QA (en) 2015-11-23 2020-06-29 Univ California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
JP6744917B2 (ja) * 2015-11-25 2020-08-19 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド バリアント検出のための方法
US20210395799A1 (en) * 2015-11-25 2021-12-23 Integrated Dna Technologies, Inc. Methods for variant detection
CN108779466B (zh) 2015-11-30 2024-03-29 杜克大学 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法
NZ782858A (en) 2015-12-04 2025-08-29 Novartis Ag Compositions and methods for immunooncology
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
CN105624196A (zh) * 2015-12-24 2016-06-01 江苏大学 一种建立cyp2c11基因敲除大鼠模型的方法
CN105624191A (zh) * 2015-12-24 2016-06-01 江苏大学 一种建立cyp2d1基因敲除大鼠模型的方法
EP4219689A3 (en) 2015-12-30 2023-12-20 Novartis AG Immune effector cell therapies with enhanced efficacy
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
EA201891614A1 (ru) 2016-01-11 2019-02-28 Те Борд Оф Трастиз Оф Те Лилэнд Стэнфорд Джуниор Юниверсити Химерные белки и способы иммунотерапии
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
US10053693B2 (en) 2016-01-19 2018-08-21 Mubin I. Syed Method for controlling obesity using minimally invasive means
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
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US12122997B2 (en) 2016-02-15 2024-10-22 Temple University—Of the Commonwealth System of Higher Education Excision of retroviral nucleic acid sequences
CN109069544B (zh) 2016-02-18 2023-05-09 宾州研究基金会 脑内生成gaba能神经元
EP3417061B1 (en) 2016-02-18 2022-10-26 The Regents of the University of California Methods and compositions for gene editing in stem cells
WO2017147196A1 (en) 2016-02-22 2017-08-31 Massachusetts Institute Of Technology Methods for identifying and modulating immune phenotypes
US10286073B2 (en) * 2016-02-23 2019-05-14 Ilisa Tech, Inc. Magnetic control of gene delivery in vivo
SG11201807025SA (en) * 2016-02-26 2018-09-27 Lanzatech New Zealand Ltd Crispr/cas systems for c-1 fixing bacteria
GB201612191D0 (en) 2016-07-13 2016-08-24 4D Pharma Plc Compositions comprising bacterial strains
JP2019507610A (ja) 2016-03-04 2019-03-22 インドア バイオテクノロジーズ インコーポレイテッド CRISPR−Casゲノム編集に基づく、Fel d1ノックアウト並びに関連組成物及び方法
PL3313423T3 (pl) 2016-03-04 2019-09-30 4D Pharma Plc Kompozycje zawierające szczepy bakteryjne blautia do leczenia nadwrażliwości trzewnej
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
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
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
US11180539B2 (en) 2016-03-29 2021-11-23 Karydo Therapeutix, Inc. Pharmaceutical composition or food composition, and method for assessing effect of active ingredient in vivo
EP3438282A4 (en) 2016-03-29 2020-05-06 Advanced Telecommunications Research Institute International METHOD FOR SCREENING CANDIDATE SUBSTANCES FOR AN ACTIVE COMPONENT TO PREVENT OR TREAT AT LEAST ONE SELECTED DISEASE IN THE KIDNEY HYPOFUNCTION GROUP, CHRONIC KIDNEY DISEASE AND KIDNEY DEFICIENCY
BR112018069795A2 (pt) * 2016-03-30 2019-01-29 Intellia Therapeutics, Inc. formulações de nanopartículas lipídicas para componentes de crispr/cas
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3443086B1 (en) 2016-04-13 2021-11-24 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CA3026055A1 (en) 2016-04-19 2017-10-26 The Broad Institute, Inc. Novel crispr enzymes and systems
AU2017257624B2 (en) 2016-04-25 2023-05-25 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
US11213594B2 (en) 2016-04-29 2022-01-04 Poseida Therapeutics, Inc. Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
CA3016641A1 (en) 2016-05-05 2017-11-09 Children's Hospital Medical Center Methods for the in vitro manufacture of gastric fundus tissue and compositions related to same
CN117904112A (zh) 2016-05-18 2024-04-19 沃雅戈治疗公司 调节性多核苷酸
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
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
LT3474669T (lt) 2016-06-24 2022-06-10 The Regents Of The University Of Colorado, A Body Corporate Barkodu pažymėtų kombinatorinių bibliotekų generavimo būdai
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
TWI802545B (zh) 2016-07-13 2023-05-21 英商4D製藥有限公司 包含細菌菌株之組合物
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
US11123409B2 (en) * 2016-07-28 2021-09-21 Institute For Basic Science Method of treating or preventing eye disease using Cas9 protein and guide RNA
EP3490373B1 (en) 2016-07-29 2023-10-25 Regeneron Pharmaceuticals, Inc. Mice comprising mutations resulting in expression of c-truncated fibrillin-1
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
RU2767206C2 (ru) * 2016-08-12 2022-03-16 Тулджен Инкорпорейтед Модифицированный иммунорегуляторный элемент и изменение иммунитета посредством этого элемента
EP3500670B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method for selecting target sequences for guide rna of crispr systems
WO2018035364A1 (en) 2016-08-17 2018-02-22 The Broad Institute Inc. Product and methods useful for modulating and evaluating immune responses
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US12431216B2 (en) 2016-08-17 2025-09-30 Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035503A1 (en) * 2016-08-18 2018-02-22 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
WO2019165322A1 (en) * 2018-02-22 2019-08-29 Moore Tara Crispr/cas9 systems, and methods of use thereof
US20220056440A1 (en) * 2016-08-20 2022-02-24 Tara Moore Crispr gene editing for autosomal dominant diseases
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
US11525119B2 (en) 2016-09-06 2022-12-13 The Children's Medical Center Corporation Immune cells derived from induced pluripotent stem cell
US20190262399A1 (en) 2016-09-07 2019-08-29 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
EP3516056B1 (en) * 2016-09-23 2024-11-27 DSM IP Assets B.V. A guide-rna expression system for a host cell
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
AU2017335890B2 (en) 2016-09-30 2024-05-09 The Regents Of The University Of California RNA-guided nucleic acid modifying enzymes and methods of use thereof
AU2017335883B2 (en) 2016-09-30 2024-06-13 The Regents Of The University Of California RNA-guided nucleic acid modifying enzymes and methods of use thereof
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
CN118726313A (zh) 2016-10-07 2024-10-01 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
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
EP3529347A1 (en) 2016-10-19 2019-08-28 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
CN110382012B (zh) 2016-11-04 2023-08-25 儿童医院医学中心 肝类器官疾病模型以及其制备和使用方法
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
CN110062764B (zh) 2016-12-05 2024-07-02 儿童医院医学中心 结肠类器官及其制备和使用方法
KR102898130B1 (ko) 2016-12-09 2025-12-10 더 브로드 인스티튜트, 인코퍼레이티드 Crispr 이펙터 시스템 기반 진단
WO2018107103A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr-systems for modifying a trait of interest in a plant
GB201621123D0 (en) 2016-12-12 2017-01-25 4D Pharma Plc Compositions comprising bacterial strains
RU2019121992A (ru) 2016-12-14 2021-01-15 Лигандал, Инк. Способы и композиции для доставки полезной нагрузки в виде нуклеиновых кислот и белков
EP3555297A1 (en) 2016-12-19 2019-10-23 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
AU2017382323B2 (en) * 2016-12-23 2024-06-13 President And Fellows Of Harvard College Gene editing of PCSK9
EP3564386B1 (en) * 2016-12-27 2021-12-08 Chugai Seiyaku Kabushiki Kaisha Method for evaluating immunogenicity of test substance
EP3565907B1 (en) 2017-01-06 2022-05-04 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
WO2018136702A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11591621B2 (en) 2017-02-07 2023-02-28 Modalis Therapeutics Corporation Method of treating diseases associated with elevated KRAS expression using CRISPR-GNDM system
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
CN118416088A (zh) * 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
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
EP3596218B1 (en) 2017-03-15 2023-08-23 The Broad Institute, Inc. Crispr effector system based diagnostics for virus detection
US11174515B2 (en) 2017-03-15 2021-11-16 The Broad Institute, Inc. CRISPR effector system based diagnostics
EP3596207B1 (en) 2017-03-15 2023-12-20 The Broad Institute, Inc. Novel cas13b orthologues crispr enzymes and systems
WO2018170515A1 (en) 2017-03-17 2018-09-20 The Broad Institute, Inc. Methods for identifying and modulating co-occurant cellular phenotypes
CA3057330A1 (en) * 2017-03-21 2018-09-27 Anthony P. Shuber Treating cancer with cas endonuclease complexes
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
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
US11963966B2 (en) 2017-03-31 2024-04-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
WO2018183921A1 (en) 2017-04-01 2018-10-04 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
US11618928B2 (en) 2017-04-12 2023-04-04 The Broad Institute, Inc. CRISPR effector system based diagnostics for malaria detection
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
US12331319B2 (en) 2017-04-12 2025-06-17 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
EP3609997A4 (en) 2017-04-14 2021-03-03 Children's Hospital Medical Center COMPOSITIONS OF STEM CELLS FROM MULTIPLE DONOR CELLS AND THEIR PREPARATION PROCEDURES
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
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.
EP3612023A4 (en) 2017-04-20 2021-05-12 Egenesis, Inc. Methods for generating genetically modified animals
AU2018254619B2 (en) 2017-04-21 2022-07-21 The General Hospital Corporation Variants of Cpf1 (CAS12a) with altered PAM specificity
EP3612232A1 (en) 2017-04-21 2020-02-26 The Broad Institute, Inc. Targeted delivery to beta cells
US11696954B2 (en) * 2017-04-28 2023-07-11 Exicure Operating Company 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
WO2018204777A2 (en) 2017-05-05 2018-11-08 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)
KR20180123445A (ko) 2017-05-08 2018-11-16 주식회사 툴젠 인위적으로 조작된 조작면역세포
WO2018208910A1 (en) 2017-05-09 2018-11-15 The Broad Institute Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症(als)を処置する方法
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
EP3622092A4 (en) 2017-05-11 2021-06-23 The Broad Institute, Inc. METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF
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
CA3063405A1 (en) * 2017-05-12 2018-11-15 President And Fellows Of Harvard College Universal early cancer diagnostics
EP3624942A1 (en) * 2017-05-16 2020-03-25 Wisconsin Alumni Research Foundation System and method for en masse patterning of molecule structures
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3063739A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP7212945B2 (ja) 2017-05-22 2023-01-26 フォーディー ファーマ リサーチ リミテッド 細菌株を含む組成物
MA41708A (fr) 2017-05-24 2020-04-08 4D Pharma Res Ltd Compositions comprenant des souches bactériennes
WO2018218166A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
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
CA3065813A1 (en) 2017-06-09 2018-12-13 Editas Medicine, Inc. Engineered cas9 nucleases
HUE059249T2 (hu) 2017-06-14 2022-11-28 4D Pharma Res Limited Baktériumtörzseket tartalmazó készítmények
AU2018285445B2 (en) 2017-06-14 2020-03-26 Cj Bioscience, Inc. Compositions comprising bacterial strains
PT3638271T (pt) 2017-06-14 2021-01-05 4D Pharma Res Ltd Composições contendo estirpes bacterianas
US11897953B2 (en) 2017-06-14 2024-02-13 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
WO2018237107A1 (en) 2017-06-23 2018-12-27 University Of Kentucky Research Foundation Method
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
JP7454494B2 (ja) 2017-06-26 2024-03-22 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crispr/cas-アデニンデアミナーゼ系の組成物、系及び方法
CA3068304A1 (en) * 2017-06-28 2019-01-03 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
EP3645721A1 (en) 2017-06-30 2020-05-06 Novartis AG Methods for the treatment of disease with gene editing systems
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
EP3648781A4 (en) 2017-07-07 2021-05-19 The Broad Institute, Inc. CRISPR SYSTEM BASED ANTIVIRAL THERAPY
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
WO2019018440A1 (en) 2017-07-17 2019-01-24 The Broad Institute, Inc. HUMAN COLON CELL ATLAS IN GOOD HEALTH AND WITH HEMORRHAGIC RECTO-COLITIS
WO2019017321A1 (ja) * 2017-07-18 2019-01-24 国立大学法人京都大学 遺伝子変異導入方法
WO2019018694A1 (en) 2017-07-19 2019-01-24 Dana-Farber Cancer Institute, Inc. DIAGNOSIS AND TREATMENT OF CANCER
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
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
CN115074343A (zh) * 2017-07-31 2022-09-20 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
BR112019027673A2 (pt) 2017-07-31 2020-09-15 Regeneron Pharmaceuticals, Inc. animal não humano, e, métodos para testar a recombinação induzida por crispr/cas e para otimizar a capacidade de crispr/cas
WO2019028023A2 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR EVALUATING CRISPR / CAS MEDIATED DISRUPTION OR EXCISION AND CRISPR / CAS INDUCED RECOMBINATION USING IN VIVO EXOGENIC DONOR NUCLEIC ACID
CN111278450B (zh) 2017-08-23 2024-04-09 通用医疗公司 具有改变的PAM特异性的工程化的CRISPR-Cas9核酸酶
US11319532B2 (en) 2017-08-30 2022-05-03 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
WO2019060746A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. SYSTEMS, METHODS, AND COMPOSITIONS FOR THE TARGETED EDITING OF NUCLEIC ACIDS
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof
MX2020003019A (es) 2017-09-27 2020-08-03 Pioneer Hi Bred Int Aplicacion en suelo de agentes protectores de cultivos.
KR102215475B1 (ko) * 2017-09-28 2021-02-15 주식회사 탠덤 암 치료용 신규 재조합 원형질막-기반 소포체
AU2018338787C1 (en) 2017-09-29 2025-10-23 Intellia Therapeutics, Inc. Compositions and methods for TTR gene editing and treating ATTR amyloidosis
AU2018338790B2 (en) 2017-09-29 2022-09-15 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus and methods of use
WO2019070755A1 (en) 2017-10-02 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR DETECTING AND MODULATING A GENETIC SIGNATURE OF IMMUNOTHERAPY RESISTANCE IN CANCER
CA3076518A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. Crispr effector system based diagnostics
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
KR20250068785A (ko) 2017-10-04 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
EP3694603A4 (en) 2017-10-10 2021-07-14 Children's Hospital Medical Center ESOPHAGUS TISSUE AND / OR ORGANOID COMPOSITIONS AND METHOD FOR MANUFACTURING THEREOF
US11725228B2 (en) 2017-10-11 2023-08-15 The General Hospital Corporation Methods for detecting site-specific and spurious genomic deamination induced by base editing technologies
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
AU2018351072B9 (en) 2017-10-20 2024-09-05 Fred Hutchinson Cancer Center Systems and methods to produce B cells genetically modified to express selected antibodies
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US12227753B2 (en) 2017-11-01 2025-02-18 The Regents Of The University Of California CasY compositions and methods of use
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
WO2019090175A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
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
CN120310773A (zh) 2017-11-16 2025-07-15 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
US12521201B2 (en) 2017-12-07 2026-01-13 Augmedics Ltd. Spinous process clamp
WO2019113506A1 (en) 2017-12-07 2019-06-13 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
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3727394A4 (en) 2017-12-21 2021-09-08 Children's Hospital Medical Center DIGITALIZED HUMAN ORGANOIDS AND METHOD OF USING THEREOF
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
BR112020014168A2 (pt) 2018-01-12 2020-12-08 Basf Se Proteína, ácido nucleico isolado, gene recombinante, vetor, célula hospedeira, planta, parte de planta ou semente de trigo, métodos para produzir, produto de trigo, farinha, farelo integral, amido, grânulos de amido ou farelo de trigo e métodos para identificar e/ou selecionar uma planta de trigo
WO2019143678A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
EP3740483B1 (en) 2018-01-17 2023-01-11 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
CN111741955B (zh) 2018-01-17 2024-02-23 沃泰克斯药物股份有限公司 Dna-pk抑制剂
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
CA3087362A1 (en) 2018-01-29 2019-08-01 The Broad Institute, Inc. Crispr effector system based diagnostics
KR20250099758A (ko) 2018-01-31 2025-07-02 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
CN112203676A (zh) * 2018-02-02 2021-01-08 宾州研究基金会 治疗脑损伤的方法和材料
EP3735248A4 (en) * 2018-02-09 2021-11-17 Empirico Inc. PROCESS FOR INHIBITED OR ELIMINATING EOSINOPHILIC DISEASES OF THE RESPIRATORY TRACT AND ASSOCIATED CONDITIONS
US20220010293A1 (en) 2018-02-23 2022-01-13 Pioneer Hi-Bred International, Inc. Novel cas9 orthologs
US20210054370A1 (en) * 2018-02-27 2021-02-25 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
KR101889683B1 (ko) * 2018-03-14 2018-08-17 유병탁 철골 구조 시공 방법
PT3765615T (pt) 2018-03-14 2023-08-28 Arbor Biotechnologies Inc Novas enzimas e sistemas de direcionamento de dna crispr
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
FI3765616T3 (fi) 2018-03-14 2023-08-29 Arbor Biotechnologies Inc Uusia CRISPR-DNA- ja RNA-kohdennusentsyymejä ja -järjestelmiä
BR112020018658A2 (pt) 2018-03-15 2020-12-29 KSQ Therapeutics, Inc. Composições de regulação gênica e métodos para imu-noterapia aprimorada
EP3592140A1 (en) * 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
EP3768327A4 (en) * 2018-03-23 2022-04-13 The Trustees of Columbia University in the City of New York GENE EDITING FOR AUTOSOMAL DOMINANT DISEASES
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
CA3097044A1 (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
WO2019204585A1 (en) 2018-04-19 2019-10-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded dna
ES2922902T3 (es) 2018-04-24 2022-09-21 Kws Saat Se & Co Kgaa Plantas con digestibilidad mejorada y haplotipos marcadores
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
EP3787693A4 (en) * 2018-04-30 2022-03-09 Oregon Health & Science University GENE THERAPY METHODS
US11980507B2 (en) 2018-05-02 2024-05-14 Augmedics Ltd. Registration of a fiducial marker for an augmented reality system
WO2019213626A1 (en) * 2018-05-03 2019-11-07 President And Fellows Of Harvard College In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells
US20210386829A1 (en) 2018-05-04 2021-12-16 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
CN112534054B (zh) 2018-05-11 2025-10-28 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
US12522812B2 (en) 2018-05-16 2026-01-13 Arbor Biotechnologies, Inc. CRISPR-associated systems and components
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
EP3797160A1 (en) 2018-05-23 2021-03-31 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
US20210371932A1 (en) 2018-06-01 2021-12-02 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
AU2019294630A1 (en) 2018-06-26 2020-12-17 Massachusetts Institute Of Technology Crispr effector system based amplification methods, systems, and diagnostics
WO2020006049A1 (en) 2018-06-26 2020-01-02 The Broad Institute, Inc. Crispr/cas and transposase based amplification compositions, systems and methods
US20210207203A1 (en) 2018-06-26 2021-07-08 The Broad Institute, Inc. Crispr double nickase based amplification compositions, systems, and methods
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
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
WO2020014261A1 (en) 2018-07-09 2020-01-16 The Broad Institute, Inc. Rna programmable epigenetic rna modifiers and uses thereof
EP3821012A4 (en) 2018-07-13 2022-04-20 The Regents of The University of California Retrotransposon-based delivery vehicle and methods of use thereof
US20210284981A1 (en) * 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
JP7506657B2 (ja) 2018-07-26 2024-06-26 チルドレンズ ホスピタル メディカル センター 肝胆膵組織およびその作製方法
AU2019314433A1 (en) 2018-07-31 2021-03-25 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
GB201812603D0 (en) 2018-08-02 2018-09-19 British American Tobacco Investments Ltd Method
US20210163944A1 (en) 2018-08-07 2021-06-03 The Broad Institute, Inc. Novel cas12b enzymes and systems
KR102158428B1 (ko) 2018-08-16 2020-09-22 (주)라트바이오 인위적 뉴클레아제를 생산하는 형질전환 배아 및 형질전환 동물의 제작 방법
KR20210058816A (ko) 2018-08-18 2021-05-24 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 원 위치에 유전자 편집
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020068304A2 (en) 2018-08-20 2020-04-02 The Broad Institute, Inc. Inhibitors of rna-guided nuclease target binding and uses thereof
WO2020041387A1 (en) 2018-08-20 2020-02-27 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
US12460244B2 (en) 2018-08-20 2025-11-04 The Broad Institute, Inc. Inhibitors of RNA-guided nuclease activity and uses thereof
WO2020041456A1 (en) 2018-08-22 2020-02-27 The Regents Of The University Of California Variant type v crispr/cas effector polypeptides and methods of use thereof
US20210317479A1 (en) 2018-09-06 2021-10-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
CN112823012A (zh) 2018-09-12 2021-05-18 儿童医院医学中心 用于产生造血干细胞及其衍生物的类器官组合物
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
AU2019343045A1 (en) 2018-09-18 2021-05-13 Vnv Newco Inc. ARC-based capsids and uses thereof
US12467925B2 (en) 2018-10-03 2025-11-11 The Broad Institute, Inc. CRISPR effector system based diagnostics for hemorrhagic fever detection
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
WO2020077135A1 (en) 2018-10-10 2020-04-16 Dana-Farber Cancer Institute, Inc. Modulating resistance to bcl-2 inhibitors
US20220411783A1 (en) 2018-10-12 2022-12-29 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
US20210379057A1 (en) 2018-10-16 2021-12-09 Massachusetts Institute Of Technology Nutlin-3a for use in treating a mycobacterium tuberculosis infection
CN109295081B (zh) * 2018-10-17 2020-06-12 中国人民解放军第四军医大学 一种LDLR-Lamp2b融合基因、表达载体及其应用
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020092057A1 (en) 2018-10-30 2020-05-07 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
BR112021008411A2 (pt) 2018-11-01 2021-09-14 E.I. Du Pont De Nemours And Company Sistemas e métodos para fabricação de núcleos agrícolas de liberação controlada
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
GB201817971D0 (en) 2018-11-02 2018-12-19 British American Tobacco Investments Ltd Method
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
US12460189B2 (en) 2018-11-09 2025-11-04 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
CN114045308B (zh) * 2018-11-13 2024-11-05 四川横竖生物科技股份有限公司 基于hNPY与hAgRP的基因过表达嵌合动物模型、工程猴模型及应用
CN113302312A (zh) 2018-11-14 2021-08-24 博德研究所 使用sherlock检测方法对高度进化的病毒变体进行多重化
JP2022513602A (ja) 2018-11-14 2022-02-09 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系に基づく液滴診断システム及び方法
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
CN119193710A (zh) * 2018-12-05 2024-12-27 弗莱德哈钦森癌症中心 遗传修饰细胞的减少和最少的操作制造
UA129215C2 (uk) 2018-12-05 2025-02-12 Інтелліа Терапьютікс, Інк. Модифіковані амінові ліпіди
US12258594B2 (en) 2018-12-05 2025-03-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
US20250043366A1 (en) 2018-12-13 2025-02-06 The Broad Institute, Inc. Tiled assays using crispr-cas based detection
BR112021011372A2 (pt) 2018-12-14 2021-08-31 Pioneer Hi-Bred International, Inc. Novos sistemas crispr-cas para edição de genoma
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
US11384344B2 (en) 2018-12-17 2022-07-12 The Broad Institute, Inc. CRISPR-associated transposase systems and methods of use thereof
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
SG11202105189RA (en) 2018-12-20 2021-06-29 Regeneron Pharma Nuclease-mediated repeat expansion
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US20220064635A1 (en) * 2019-01-07 2022-03-03 Emendobio Inc. Crispr compositions and methods for promoting gene editing of adenosine deaminase 2 (ada2)
EP3911632A1 (en) 2019-01-15 2021-11-24 Empirico Inc. Prodrugs of alox-15 inhibitors and methods of using the same
GB201900940D0 (en) 2019-01-23 2019-03-13 British American Tobacco Investments Ltd Method
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160044A1 (en) 2019-01-28 2020-08-06 The Broad Institute, Inc. In-situ spatial transcriptomics
US11965159B2 (en) 2019-01-29 2024-04-23 The Broad Institute, Inc. Compositions and methods for regulating proteins and nucleic acids activities
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020163856A1 (en) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Modified mitochondrion and methods of use thereof
AU2020223060B2 (en) 2019-02-13 2023-04-13 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
US20220098593A1 (en) * 2019-02-13 2022-03-31 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
US20230053540A1 (en) 2019-02-19 2023-02-23 Massachusetts Institute Of Technology Treatment of liver injury
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
CA3130789A1 (en) 2019-03-07 2020-09-10 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
CA3129744A1 (en) 2019-03-12 2020-09-17 Emmanuelle CHARPENTIER Cas9 variants with enhanced specificity
WO2020186101A1 (en) 2019-03-12 2020-09-17 The Broad Institute, Inc. Detection means, compositions and methods for modulating synovial sarcoma cells
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
US20220220546A1 (en) 2019-03-14 2022-07-14 The Broad Institute, Inc. Sherlock assays for tick-borne diseases
WO2020186213A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2020186231A2 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Crispr effector system based multiplex diagnostics
EP3937969A1 (en) 2019-03-14 2022-01-19 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate intestinal innate lymphoid cells
WO2020191069A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Modulation of type 2 immunity by targeting clec-2 signaling
US20220177863A1 (en) 2019-03-18 2022-06-09 The Broad Institute, Inc. Type vii crispr proteins and systems
ES2969026T3 (es) 2019-03-18 2024-05-16 Regeneron Pharma Plataforma de detección de abandonos CRISPR/CAS para revelar vulnerabilidades genéticas asociadas con la agregación de tau
EP3942023A1 (en) 2019-03-18 2022-01-26 The Broad Institute, Inc. Compositions and methods for modulating metabolic regulators of t cell pathogenicity
CN120648640A (zh) 2019-03-18 2025-09-16 瑞泽恩制药公司 用于鉴定tau接种或聚集的基因修饰因子的CRISPR/Cas筛选平台
BR112021018607A2 (pt) 2019-03-19 2021-11-23 Massachusetts Inst Technology Métodos e composições para editar sequências de nucleotídeos
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
US20220195514A1 (en) 2019-03-29 2022-06-23 The Broad Institute, Inc. Construct for continuous monitoring of live cells
WO2020206046A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Methods and compositions for cell therapy
WO2020206036A1 (en) 2019-04-01 2020-10-08 The Broad Institute, Inc. Novel nucleic acid modifier
CN118064502A (zh) 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
KR102661779B1 (ko) 2019-04-04 2024-04-30 리제너론 파마슈티칼스 인코포레이티드 인간화 응고 인자 12 좌위를 포함하는 비-인간 동물
CN113795588B (zh) 2019-04-04 2025-02-25 瑞泽恩制药公司 用于在靶向性载体中无瘢痕引入靶向修饰的方法
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020223539A1 (en) 2019-04-30 2020-11-05 The Broad Institute, Inc. Methods and compositions for barcoding nucleic acid libraries and cell populations
EP3963062B1 (en) * 2019-05-03 2026-01-07 Specific Biologics Inc. Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing
WO2020231702A1 (en) * 2019-05-11 2020-11-19 Youngsuk Yi Compositions and methods containing exosomes
CN114096684B (zh) 2019-05-13 2024-11-19 科沃施种子欧洲股份两合公司 玉米的耐旱性
GB201906768D0 (en) 2019-05-14 2019-06-26 British American Tobacco Investments Ltd Method
WO2020232271A1 (en) 2019-05-14 2020-11-19 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
US20220220469A1 (en) 2019-05-20 2022-07-14 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
US20220226464A1 (en) 2019-05-28 2022-07-21 Massachusetts Institute Of Technology Methods and compositions for modulating immune responses
WO2020243661A1 (en) 2019-05-31 2020-12-03 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
US12497597B2 (en) 2019-05-31 2025-12-16 Children's Hospital Medical Center Methods of generating and expanding hematopoietic stem cells
KR20220016869A (ko) 2019-06-04 2022-02-10 리제너론 파마슈티칼스 인코포레이티드 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
US12188045B2 (en) 2019-06-07 2025-01-07 KSQ Therapeutics, Inc. Guide RNA combinations and methods of use
EP3813522B1 (en) 2019-06-14 2025-05-07 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
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
GB201909562D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
GB201909563D0 (en) 2019-07-03 2019-08-14 British American Tobacco Investments Ltd Method
US11980506B2 (en) 2019-07-29 2024-05-14 Augmedics Ltd. Fiducial marker
US12178666B2 (en) 2019-07-29 2024-12-31 Augmedics Ltd. Fiducial marker
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021030627A1 (en) 2019-08-13 2021-02-18 The General Hospital Corporation Methods for predicting outcomes of checkpoint inhibition and treatment thereof
US12421557B2 (en) 2019-08-16 2025-09-23 The Broad Institute, Inc. Methods for predicting outcomes and treating colorectal cancer using a cell atlas
EP4017871B1 (en) 2019-08-21 2024-01-17 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
WO2021046257A1 (en) 2019-09-03 2021-03-11 The Broad Institute, Inc. Crispr effector system based multiplex cancer diagnostics
CN114729384A (zh) 2019-09-12 2022-07-08 博德研究所 工程化腺相关病毒衣壳
AU2020346056A1 (en) 2019-09-13 2022-03-31 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
CN114616336B (zh) 2019-09-20 2025-11-14 博德研究所 用于将货物递送至靶细胞的组合物和方法
CA3151563A1 (en) 2019-09-20 2021-03-25 Feng Zhang 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
WO2021074367A1 (en) 2019-10-17 2021-04-22 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
EP4054651A1 (en) 2019-11-08 2022-09-14 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
AU2020388387A1 (en) 2019-11-22 2022-06-02 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
KR20220130686A (ko) * 2019-12-23 2022-09-27 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Crispr-cas 이펙터 폴리펩티드 및 이의 사용 방법
WO2021146641A1 (en) 2020-01-17 2021-07-22 The Broad Institute, Inc. Small type ii-d cas proteins and methods of use thereof
US12165747B2 (en) 2020-01-23 2024-12-10 The Broad Institute, Inc. Molecular spatial mapping of metastatic tumor microenvironment
CA3165346A1 (en) 2020-01-23 2021-07-29 George Q. Daley Stroma-free t cell differentiation from human pluripotent stem cells
US12312613B2 (en) 2020-01-24 2025-05-27 The General Hospital Corporation Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
US12264341B2 (en) 2020-01-24 2025-04-01 The General Hospital Corporation CRISPR-Cas enzymes with enhanced on-target activity
CN113281130B (zh) * 2020-02-20 2024-08-23 中国科学院上海高等研究院 一种用于大尺寸脑组织的高效的高尔基染色方法
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
IL295935A (en) * 2020-02-28 2022-10-01 Huigene Therapeutics Co Ltd Type vi-e and type vi-f crispr-cas system and uses thereof
AU2021232598A1 (en) 2020-03-04 2022-09-08 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
CN111321150B (zh) * 2020-03-13 2020-12-04 广西壮族自治区水产科学研究院 一种LvCTL4基因及编码的蛋白质、蛋白质的获取方法、表达载体、重组菌和应用
WO2021188734A1 (en) 2020-03-17 2021-09-23 The Broad Institute, Inc. Crispr system high throughput diagnostic systems and methods
US11851702B2 (en) 2020-03-23 2023-12-26 The Broad Institute, Inc. Rapid diagnostics
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
MX2022012189A (es) 2020-03-31 2023-01-05 Arbor Biotechnologies Inc Composiciones que comprenden una variante del polipéptido cas12i2 y usos de las mismas.
WO2021202938A1 (en) 2020-04-03 2021-10-07 Creyon Bio, Inc. Oligonucleotide-based machine learning
JP2023521055A (ja) 2020-04-09 2023-05-23 アール・ジエイ・レイノルズ・タバコ・カンパニー ニコチアナ・タバカムにおけるニコチンレベルの調整方法
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
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2021231575A1 (en) * 2020-05-12 2021-11-18 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
AR122206A1 (es) 2020-05-29 2022-08-24 Kws Saat Se & Co Kgaa Inducción de haploides en plantas
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
JP2023537053A (ja) 2020-08-07 2023-08-30 ザ・ブロード・インスティテュート・インコーポレイテッド Xpr1:kidins220タンパク質複合体を介したがんにおけるリン酸塩調節不全の治療的標的化
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
CN112159809B (zh) * 2020-09-22 2021-06-22 广州瑞风生物科技有限公司 靶向CTGF基因的gRNA及其应用
WO2022188039A1 (en) 2021-03-09 2022-09-15 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
WO2022068912A1 (en) 2020-09-30 2022-04-07 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
IL302046A (en) 2020-10-15 2023-06-01 Hoffmann La Roche Nucleic acid structures 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
CN114796493A (zh) * 2021-01-28 2022-07-29 中国科学技术大学 血管源性bace1作为脑小血管损伤相关疾病的治疗靶点的用途
AU2022220332A1 (en) 2021-02-11 2023-08-24 Arbor Biotechnologies, Inc. Compositions comprising a variant cas12i4 polypeptide and uses thereof
CN112997966A (zh) * 2021-03-08 2021-06-22 国家卫生健康委科学技术研究所 一种基于CRISPR/Cas9技术敲入miRNA-125a的小鼠模型及构建方法
CN115427561B (zh) 2021-03-09 2024-06-04 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
WO2022204543A1 (en) * 2021-03-25 2022-09-29 The Regents Of The University Of California Methods and materials for treating huntington's disease
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
CN117337326A (zh) 2021-05-27 2024-01-02 中国科学院动物研究所 工程化的Cas12i核酸酶、效应蛋白及其用途
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4347826A1 (en) 2021-06-02 2024-04-10 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4355351A1 (en) * 2021-06-18 2024-04-24 Innovative Cellular Therapeutics Holdings, Ltd. Modified cells targeting tumor ecm and therapy thereof
US11896445B2 (en) 2021-07-07 2024-02-13 Augmedics Ltd. Iliac pin and adapter
EP4373837A2 (en) 2021-07-20 2024-05-29 The Broad Institute, Inc. Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
US20230048564A1 (en) 2021-07-22 2023-02-16 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
CN118368974A (zh) 2021-07-30 2024-07-19 科沃施种子欧洲股份两合公司 具有提高的消化率和标记单倍型的植物
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
JP7125727B1 (ja) 2021-09-07 2022-08-25 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法
CA3230927A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
EP4408995A4 (en) 2021-09-28 2025-08-13 Acrigen Biosciences COMPOSITIONS AND METHODS FOR NUCLEIC ACID MODIFICATIONS
EP4412587A1 (en) 2021-10-08 2024-08-14 President And Fellows Of Harvard College Ionic liquids for drug delivery
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
WO2023064924A1 (en) 2021-10-14 2023-04-20 Codiak Biosciences, Inc. Modified producer cells for extracellular vesicle production
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
US20240415980A1 (en) 2021-10-28 2024-12-19 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
AU2022375820A1 (en) 2021-11-01 2024-06-13 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
CN114015674B (zh) 2021-11-02 2024-08-30 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
US20240408236A1 (en) 2021-11-26 2024-12-12 Epigenic Therapeutics, Inc. Method of modulating pcsk9 and uses thereof
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
CN114107231B (zh) * 2021-12-13 2023-08-18 重庆大学 实现全脑突触后神经元胞体标记的重组腺相关病毒及其应用
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
GB202118058D0 (en) 2021-12-14 2022-01-26 Univ Warwick Methods to increase yields in crops
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
EP4448775A1 (en) 2021-12-17 2024-10-23 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
WO2023115039A2 (en) 2021-12-17 2023-06-22 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
JP2025501221A (ja) 2021-12-29 2025-01-17 ブリストル-マイヤーズ スクイブ カンパニー ランディングパッド細胞株の生成
WO2023133595A2 (en) 2022-01-10 2023-07-13 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
US20250152709A1 (en) 2022-02-01 2025-05-15 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
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
US20250032642A1 (en) 2022-02-02 2025-01-30 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
US20250163392A1 (en) 2022-02-02 2025-05-22 Inscripta, Inc. Nucleic acid-guided nickase fusion proteins
AU2023215810A1 (en) 2022-02-03 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
US20250137006A1 (en) 2022-02-03 2025-05-01 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
AU2023215671A1 (en) 2022-02-04 2024-08-15 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
EP4487875A4 (en) 2022-02-28 2025-07-30 National Univ Corporation Tokai National Higher Education And Research System MEDICINE FOR A DISEASE CAUSED BY A FRAME-SHIFT MUTATION
CN114480497B (zh) * 2022-02-28 2023-09-15 湖南师范大学 一种ep400基因敲除斑马鱼心力衰竭模型的构建及其应用的方法
CA3255225A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California COMPOSITIONS AND METHODS OF GENETIC COMPLEMENTATION
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205148D0 (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
JP2025514304A (ja) 2022-04-29 2025-05-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
JP2025516527A (ja) 2022-05-09 2025-05-30 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 インビボ抗体産生のためのベクター及び方法
EP4526324A1 (en) 2022-05-18 2025-03-26 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
AU2023273885A1 (en) 2022-05-19 2024-12-05 Lyell Immunopharma, Inc. Polynucleotides targeting nr4a3 and uses thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
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
KR20250051026A (ko) 2022-07-14 2025-04-16 더 브로드 인스티튜트, 인코퍼레이티드 트랜스페린 수용체와의 상호작용을 통해 cns 전체 유전자 전달을 가능하게 하는 aav 캡시드
AU2023311964A1 (en) 2022-07-18 2025-01-30 Renagade Therapeutics Management Inc. Gene editing components, systems, and methods of use
WO2024020557A1 (en) 2022-07-22 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising a variant nuclease and uses thereof
EP4558626A1 (en) 2022-07-22 2025-05-28 Arbor Biotechnologies, Inc. Compositions comprising a variant polypeptide and uses thereof
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
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
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
KR20250044256A (ko) 2022-08-05 2025-03-31 리제너론 파마슈티칼스 인코포레이티드 Tdp-43의 응집 저항성 변이체
CN115247149B (zh) * 2022-08-22 2023-06-16 华域生物科技(天津)有限公司 适用于nk细胞的培养基组合物及培养方法
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
CA3265393A1 (en) 2022-08-26 2024-02-29 KWS SAAT SE & Co. KGaA USE OF PAIRED GENES FOR HYBRID SELECTION
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
IL319122A (en) 2022-09-28 2025-04-01 Regeneron Pharma Different antibody-resistant receptors improve 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
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
US20240173426A1 (en) 2022-11-14 2024-05-30 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
JP2025542309A (ja) 2022-12-20 2025-12-25 ナノセル セラピューティクス ホールディングス ビー.ブイ. 組み込み型ウイルスベクターおよび自己不活性化ウイルスベクターならびにその構築物および使用
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
EP4652288A2 (en) * 2023-01-18 2025-11-26 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
IL321908A (en) 2023-01-27 2025-09-01 Regeneron Pharma Different rhabdomyovirus glycoproteins and their use
WO2024163842A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
GB202303077D0 (en) 2023-03-02 2023-04-19 Univ Oslo Brassica plants with improved seed retention
WO2024192141A1 (en) 2023-03-13 2024-09-19 Dana-Farber Cancer Institute, Inc. Treatment of cancers having a drug-resistant mesenchymal cell state
WO2024197065A2 (en) * 2023-03-20 2024-09-26 Verve Therapeutics, Inc. In vivo nickase-based editing of the lpa gene for treatment of cardiovascular disease
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
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
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
WO2024227131A1 (en) * 2023-04-27 2024-10-31 Rensselaer Polytechnic Institute Recombinant enzyme for the accurate insertion of dna sequences in eukaryotic cells
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2024238817A2 (en) * 2023-05-16 2024-11-21 Fallon Jim Deceased Systems and methods for an intranasal drug delivery system
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
WO2024238977A2 (en) 2023-05-18 2024-11-21 Children's Hospital Medical Center Liver organoids with intrahepatic sympathetic nerves, and methods of use thereof
WO2024243340A1 (en) 2023-05-23 2024-11-28 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2024259309A1 (en) 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders
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
WO2025006963A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods and compositions for increasing homology-directed repair
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
TW202519551A (zh) 2023-07-28 2025-05-16 美商雷傑納榮製藥公司 用於治療酸性神經髓磷脂酶缺乏症之抗TfR:酸性神經髓磷脂酶
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
WO2025049788A1 (en) 2023-08-29 2025-03-06 The Broad Institute, Inc. Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish
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
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
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
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
WO2025217202A1 (en) 2024-04-08 2025-10-16 Children's Hospital Medical Center Bile duct organoid
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium
EP4677108A1 (en) 2024-04-22 2026-01-14 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
WO2025265017A1 (en) 2024-06-20 2025-12-26 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

Family Cites Families (262)

* 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
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
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
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
DE122007000007I2 (de) 1986-04-09 2010-12-30 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
CA2307016A1 (en) 1997-10-24 1999-05-06 Life Technologies, Inc. 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
JP3454818B1 (ja) * 2001-03-16 2003-10-06 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
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
ES2566561T3 (es) 2001-07-12 2016-04-13 University Of Massachusetts Producción in vivo de ARN pequeños de interferencia que median el silenciamiento génico
EP1572885A2 (en) 2001-08-08 2005-09-14 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
US7776321B2 (en) 2001-09-26 2010-08-17 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
CN1620508A (zh) 2001-12-21 2005-05-25 牛津生物医学(英国)有限公司 转基因生物
US7206639B2 (en) 2002-03-15 2007-04-17 Sarcos Investments Lc Cochlear drug delivery system and method
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
AU2003274404A1 (en) 2002-06-11 2003-12-22 The Scripps Research Institute Artificial transcription factors
US7867193B2 (en) 2004-01-29 2011-01-11 The Charles Stark Draper Laboratory, Inc. Drug delivery apparatus
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
AU2003283976B2 (en) 2002-09-27 2009-12-10 Cold Spring Harbor Laboratory Cell-based RNA interference and related methods and compositions
AU2003290937A1 (en) * 2002-11-15 2004-06-15 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
NZ545000A (en) 2003-07-03 2008-08-29 Univ California Genome mapping of functional DNA elements and cellular proteins
KR101168440B1 (ko) 2003-07-16 2012-07-27 프로티바 바이오쎄라퓨틱스, 인코포레이티드 지질 캡슐화된 간섭 rna
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP3222715A1 (en) 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
US8673634B2 (en) 2003-11-13 2014-03-18 Massachusetts Eye & Ear Infirmary Method for the treatment of hearing loss
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
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
US7799565B2 (en) 2004-06-07 2010-09-21 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
WO2006020071A2 (en) 2004-07-16 2006-02-23 The Government Of The United States Of America As Represented By The Secretary Of The 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
AU2006239169A1 (en) * 2005-04-28 2006-11-02 Benitec, Limited. Multiple-RNAi expression cassettes for simultaneous delivery of RNAi agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
CN101273141B (zh) * 2005-07-26 2013-03-27 桑格摩生物科学股份有限公司 外源核酸序列的靶向整合和表达
DK2325332T3 (da) 2005-08-26 2013-01-28 Dupont Nutrition Biosci Aps Anvendelse af CRISPR-associerede gener (CAS)
WO2007030743A2 (en) 2005-09-08 2007-03-15 Massachusetts Eye & Ear Infirmary Cochlear implants containing biological cells and uses thereof
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
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
WO2007106690A2 (en) 2006-03-15 2007-09-20 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
ES2874149T3 (es) 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
CA2669313A1 (en) 2006-11-14 2008-05-22 Cellectis Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof
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
AU2008223544B2 (en) 2007-03-02 2014-06-05 Dupont Nutrition Biosciences Aps Cultures with improved phage resistance
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
ES2868136T3 (es) 2007-08-14 2021-10-21 Hutchinson Fred Cancer Res Disposición de matriz de agujas y método para administrar agentes terapéuticos
JP2011512326A (ja) 2007-12-31 2011-04-21 ナノコア セラピューティクス,インコーポレイテッド 心不全の治療用のrna干渉
JP2011511806A (ja) 2008-02-07 2011-04-14 マサチューセッツ・アイ・アンド・イア・インファーマリー Atoh1発現を増強する化合物
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
JP5475753B2 (ja) 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
US20090280262A1 (en) 2008-05-08 2009-11-12 Chung Yuan Christian University Method for forming composite membrane with porous coating layer and apparatus thereof
US8431692B2 (en) 2008-06-06 2013-04-30 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
BRPI0910198A2 (pt) 2008-06-30 2016-01-12 Silenseed Ltd composição para administração a partir de um sítio local de um agente baseado em nucleotídeos e método para tratar um sujeito sofrendo de uma doença requerendo tratamento local com um agente baseado em nucleotídeo.
EP2309980A1 (en) 2008-07-08 2011-04-20 S.I.F.I. Societa' Industria Farmaceutica Italiana Ophthalmic compositions for treating pathologies of the posterior segment of the eye
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
WO2010014218A2 (en) 2008-07-29 2010-02-04 Academia Sinica Puf-a and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
CA2736336A1 (en) 2008-09-08 2010-03-11 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof
ES2615829T3 (es) 2008-09-15 2017-06-08 The Children's Medical Center Corporation Modulación de BCL11A para el tratamiento de hemoglobinopatías
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
CA2739895C (en) 2008-10-20 2018-09-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
WO2010053518A2 (en) 2008-10-29 2010-05-14 Sangamo Biosciences, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
US20110023153A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
CA2755192C (en) 2009-03-20 2018-09-11 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
AU2010234539B2 (en) 2009-04-07 2015-04-09 Corteva Agriscience Llc Nanoparticle mediated delivery of sequence specific nucleases
US20120164118A1 (en) 2009-05-04 2012-06-28 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
CA2767129C (en) 2009-07-01 2015-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
KR20120097483A (ko) * 2009-07-24 2012-09-04 시그마-알드리치 컴퍼니., 엘엘씨 게놈 편집을 위한 방법
JP5866283B2 (ja) 2009-07-28 2016-02-17 サンガモ バイオサイエンシーズ, インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物
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
CA2782014C (en) * 2009-11-27 2021-08-31 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
DK2510096T4 (en) 2009-12-10 2018-05-14 Univ Iowa State Res Found Inc NUMBER EFFECTOR-MEDIATED DNA MODIFICATION
US8933046B2 (en) 2009-12-23 2015-01-13 Max-Planck-Gesellschaft Zur Foerderung Der Wissenschaften E.V. Influenza targets
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
SG185367A1 (en) 2010-04-26 2012-12-28 Sangamo Biosciences Inc Genome editing of a rosa locus using zinc-finger nucleases
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CA2798703A1 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
EP2569435A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
KR101953237B1 (ko) * 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 신규 dna 결합 단백질 및 이의 용도
ES2536791T3 (es) 2010-05-28 2015-05-28 Oxford Biomedica (Uk) Ltd Administración de vectores lentivirales al cerebro
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
DK2601611T3 (da) 2010-08-02 2021-02-01 Integrated Dna Tech Inc Fremgangsmåder til forudsigelse af stabilitet og smeltetemperaturer for nukleinsyreduplekser
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
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
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
HRP20220036T1 (hr) 2011-04-22 2022-04-01 The Regents Of The University Of California Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe
SG194089A1 (en) 2011-04-27 2013-11-29 Amyris Inc Methods for genomic modification
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
WO2012164565A1 (en) * 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
AU2012318562A1 (en) 2011-10-06 2014-04-10 Sangamo Therapeutics, Inc. Methods and compositions for regulating HIV infection
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
JP6259766B2 (ja) 2011-11-18 2018-01-10 ユニヴェルシテ ラヴァルUniversite Laval フラタキシンレベルを増加させる方法および生成物ならびにその使用
WO2013078400A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
US20130137173A1 (en) 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
HRP20220717T1 (hr) 2011-12-16 2022-07-22 Modernatx, Inc. Modificirani pripravci mrna
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP3272356A1 (en) 2012-02-24 2018-01-24 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
RU2639277C2 (ru) 2012-02-29 2017-12-20 Сангамо Байосайенсиз, Инк. Способы и составы лечения болезни хантингтона
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
JP6189415B2 (ja) 2012-04-02 2017-08-30 モデルナティエックス インコーポレイテッドModernaTX,Inc. 細胞質および細胞骨格タンパク質の産生のための修飾ポリヌクレオチド
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US10266850B2 (en) * 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CA2874828A1 (en) 2012-06-01 2013-12-05 Drexel University 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
JP6401704B2 (ja) 2012-10-10 2018-10-10 サンガモ セラピューティクス, インコーポレイテッド T細胞を修飾する化合物およびその使用
CN110669758A (zh) 2012-10-23 2020-01-10 基因工具股份有限公司 用于切割靶dna 的组合物及其用途
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
RU2721275C2 (ru) * 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
AU2013359212B2 (en) * 2012-12-12 2017-01-19 Massachusetts Institute Of Technology Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
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
EP2940140B1 (en) 2012-12-12 2019-03-27 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
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
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
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931891B1 (en) 2012-12-17 2019-05-15 President and Fellows of Harvard College Rna-guided human genome engineering
CA2898184A1 (en) 2013-01-16 2014-07-24 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
DK3620534T3 (da) 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
KR102271291B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
KR102109069B1 (ko) 2013-03-27 2020-05-12 윌코아게 디바이스들을 인라인 검사 및/또는 테스트하는 방법 및 그와 같은 방법을 수행하는 장치
JP6576904B2 (ja) 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP2016521975A (ja) 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝的状態の処置のための方法および組成物
WO2014191518A1 (en) 2013-05-29 2014-12-04 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
AU2014274939B2 (en) 2013-06-04 2020-03-19 President And Fellows Of Harvard College RNA-guideded transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
CN105658805B (zh) 2013-06-05 2021-12-31 杜克大学 Rna指导的基因编辑和基因调节
AU2014281030B2 (en) 2013-06-17 2020-07-09 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
WO2014204729A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
KR20160060659A (ko) 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
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
EP3066201B1 (en) 2013-11-07 2018-03-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
CA2930877A1 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
CN106536729A (zh) 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
CN103668472B (zh) 2013-12-31 2014-12-24 北京大学 利用CRISPR/Cas9系统构建真核基因敲除文库的方法
CN103779358A (zh) * 2014-01-27 2014-05-07 京东方科技集团股份有限公司 一种阵列基板及其制作方法、显示装置
US10354746B2 (en) 2014-01-27 2019-07-16 Georgia Tech Research Corporation Methods and systems for identifying CRISPR/Cas off-target sites
WO2015191693A2 (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
EP3180426B1 (en) 2014-08-17 2019-12-25 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
US9944912B2 (en) 2015-03-03 2018-04-17 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
ES2905525T3 (es) 2015-05-06 2022-04-11 Snipr Tech Ltd Alteración de poblaciones microbianas y modificación de la microbiota
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
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
US11559588B2 (en) 2017-02-22 2023-01-24 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
CA3123886A1 (en) 2019-01-17 2020-07-23 James DAHLMAN Drug delivery systems containing oxidized cholesterols
JP7600208B2 (ja) 2019-07-29 2024-12-16 ジョージア テック リサーチ コーポレイション 拘束された脂質を含むナノ材料およびその使用

Also Published As

Publication number Publication date
JP7013406B2 (ja) 2022-02-15
WO2014093622A3 (en) 2014-08-07
US12252707B2 (en) 2025-03-18
EP2931897B1 (en) 2017-11-01
US20140242699A1 (en) 2014-08-28
EP3327127B1 (en) 2021-03-24
EP4299741A3 (en) 2024-02-28
WO2014093622A9 (en) 2014-11-06
US20250382642A1 (en) 2025-12-18
HK1252619A1 (en) 2019-05-31
JP2019103513A (ja) 2019-06-27
ZA201504132B (en) 2022-03-30
EP3031921B1 (en) 2025-03-12
SG10201707569YA (en) 2017-10-30
AU2022203759A1 (en) 2022-06-23
CA2894681A1 (en) 2014-06-19
AU2013359199C1 (en) 2021-06-17
CN110872583A (zh) 2020-03-10
CN105164264B (zh) 2021-05-07
KR20150105956A (ko) 2015-09-18
AU2019283878A1 (en) 2020-01-23
US20170152528A1 (en) 2017-06-01
MX2015007550A (es) 2017-02-02
JP2023073245A (ja) 2023-05-25
JP2025181841A (ja) 2025-12-11
DK2931897T3 (en) 2018-02-05
AU2019283878B2 (en) 2022-06-09
IL239317B (en) 2022-07-01
IL293526A (en) 2022-08-01
JP2016501531A (ja) 2016-01-21
HK1215048A1 (en) 2016-08-12
US20230399662A1 (en) 2023-12-14
US20240035048A1 (en) 2024-02-01
AU2022203759B2 (en) 2025-02-27
WO2014093622A2 (en) 2014-06-19
RU2721275C2 (ru) 2020-05-18
US20140179770A1 (en) 2014-06-26
WO2014093622A8 (en) 2014-12-04
EP4299741A2 (en) 2024-01-03
JP7736729B2 (ja) 2025-09-09
EP3327127A1 (en) 2018-05-30
AU2013359199A1 (en) 2015-07-30
MX2022001378A (es) 2022-06-23
EP3031921A1 (en) 2016-06-15
EP2931897A2 (en) 2015-10-21
US20170107536A1 (en) 2017-04-20
EP4549566A2 (en) 2025-05-07
RU2015128057A (ru) 2019-03-27
AU2013359199B2 (en) 2019-10-31
IL239317A0 (en) 2015-07-30
SG11201504523UA (en) 2015-07-30
US11041173B2 (en) 2021-06-22
AU2025203782A1 (en) 2025-06-12
JP7228059B2 (ja) 2023-02-22
MX389793B (es) 2025-03-20
HK1220725A1 (en) 2017-05-12
US20150020223A1 (en) 2015-01-15
ES2658401T3 (es) 2018-03-09
CN105164264A (zh) 2015-12-16
JP2022040308A (ja) 2022-03-10
EP4549566A3 (en) 2025-07-09
SG10201912328UA (en) 2020-02-27
BR112015013784A2 (pt) 2017-07-11
US20210269831A1 (en) 2021-09-02
ES2883590T3 (es) 2021-12-09
US20250250589A1 (en) 2025-08-07

Similar Documents

Publication Publication Date Title
DK3327127T3 (da) Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til sekvensmanipulation og terapeutiske anvendelser
DK3064585T3 (da) Konstruering og optimering af forbedrede systemer, fremgangsmåder og enzymsammensætninger til sekvensmanipulation
DK3252160T3 (da) Systemer, fremgangsmåder og sammensætninger med crispr-cas-bestanddele til sekvensmanipulation
DK3011029T3 (da) Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering
DK3011031T3 (da) Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
DK3556869T3 (da) Sammensætninger og fremgangsmåder til sekventering af polynukleotider
DK2970872T3 (da) Mikroorganismer og fremgangsmåder til fremstilling af sialylerede og N-acetylglucosamin-holdige oligosaccharider
DK3611262T3 (da) Fremgangsmåder til sekvensering af immunrepertoire
DK3597749T5 (da) Fremgangsmåder og sammensætninger til rna-dirigeret mål-dna-modificering og til rna-dirigeret modulering af transkription
DK3656557T3 (da) System og arbejdsstation til design, fabrikation og fremstilling af biomaterielle konstruktioner
DK2729151T3 (da) Farmaceutisk sammensætning, fremgangsmåder til behandling og anvendelser deraf
DK2776567T3 (da) Sammensætninger og fremgangsmåder til behandling af cytomegalovirus.
DK2804587T3 (da) Formulering og fremgangsmåde til øgning af lægemidlers biotilgængelighed
DK2852391T3 (da) Systemer til behandling af lungeinfektioner
DK2971167T3 (da) Sammensætninger og fremgangsmåder til targeting af nucleinsyre med nucleinsyrer
DK2954051T3 (da) Modificeret kapsid til genoverførsel til behandling af nethinden
DK3594348T3 (da) Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder
DK3019503T3 (da) Polycykliske-carbamoylpyridon-forbindelser og anvendelse deraf til behandling af hiv-infektioner
DK2970456T3 (da) Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
DK2897620T3 (da) Fremgangsmåde til behandling af cancer
DK2981255T3 (da) Terapeutiske anvendelser af empagliflozin
DK3025586T3 (da) Sammensætning til bekæmpelse af plantesygdomme og anvendelse deraf
EP2827882A4 (en) SYSTEMS, COMPOSITIONS AND METHODS FOR TRANSPLANTATIONS
DK2967049T3 (da) Metoder, forbindelser og sammensætninger til behandlingen af angiotensin relaterede sygdomme
DK3049073T3 (da) Sammensætninger og fremgangsmåder til destabilisering, ændring og dispergering af biofilm