SG11201504523UA - Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications - Google Patents

Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Info

Publication number
SG11201504523UA
SG11201504523UA SG11201504523UA SG11201504523UA SG11201504523UA SG 11201504523U A SG11201504523U A SG 11201504523UA SG 11201504523U A SG11201504523U A SG 11201504523UA SG 11201504523U A SG11201504523U A SG 11201504523UA SG 11201504523U A SG11201504523U A SG 11201504523UA
Authority
SG
Singapore
Prior art keywords
optimization
engineering
delivery
compositions
systems
Prior art date
Application number
SG11201504523UA
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=SG11201504523U(A) "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
Publication of SG11201504523UA publication Critical patent/SG11201504523UA/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201—Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8213—Targeted insertion of genes into the plant genome by homologous recombination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/20—Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/30—Detection of binding sites or motifs
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/50—Mutagenesis
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10—Sequence alignment; Homology search
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Environmental Sciences (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hematology (AREA)
  • Rheumatology (AREA)
  • Oncology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Diabetes (AREA)
SG11201504523UA 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications SG11201504523UA (en)

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
US201361791409P 2013-03-15 2013-03-15
US201361802174P 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
US201361836123P 2013-06-17 2013-06-17
US201361835931P 2013-06-17 2013-06-17
US201361847537P 2013-07-17 2013-07-17
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Publications (1)

Publication Number Publication Date
SG11201504523UA true SG11201504523UA (en) 2015-07-30

Family

ID=49883289

Family Applications (3)

Application Number Title Priority Date Filing Date
SG10201912328UA SG10201912328UA (en) 2012-12-12 2013-12-12 Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
SG10201707569YA SG10201707569YA (en) 2012-12-12 2013-12-12 Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
SG11201504523UA SG11201504523UA (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications

Family Applications Before (2)

Application Number Title Priority Date Filing Date
SG10201912328UA SG10201912328UA (en) 2012-12-12 2013-12-12 Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
SG10201707569YA SG10201707569YA (en) 2012-12-12 2013-12-12 Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications

Country Status (17)

Country Link
US (8) US20140179770A1 (en)
EP (3) EP3327127B1 (en)
JP (4) JP2016501531A (en)
KR (1) KR20150105956A (en)
CN (2) CN105164264B (en)
AU (3) AU2013359199C1 (en)
BR (1) BR112015013784A2 (en)
CA (1) CA2894681A1 (en)
DK (2) DK3327127T3 (en)
ES (2) ES2883590T3 (en)
HK (3) HK1215048A1 (en)
IL (2) IL239317B (en)
MX (2) MX2015007550A (en)
RU (1) RU2721275C2 (en)
SG (3) SG10201912328UA (en)
WO (1) WO2014093622A2 (en)
ZA (1) ZA201504132B (en)

Families Citing this family (706)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9445581B2 (en) 2012-03-28 2016-09-20 Kymab Limited Animal models and therapeutic molecules
HUE055817T2 (en) 2009-07-08 2021-12-28 Kymab Ltd Animal models and therapeutic molecules
US20120204278A1 (en) 2009-07-08 2012-08-09 Kymab Limited Animal models and therapeutic molecules
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2011140441A2 (en) 2010-05-06 2011-11-10 Children's Hospital Medical Center Methods and systems for converting precursor cells into intestinal tissues through directed differentiation
US9457079B2 (en) 2010-05-12 2016-10-04 The Trustees Of Columbia University In The City Of New York Methods for producing enteroendocrine cells that make and secrete insulin
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
CA2846319A1 (en) 2011-09-19 2013-03-28 Kymab Limited Antibodies, variable domains & chains tailored for human use
WO2013045916A1 (en) 2011-09-26 2013-04-04 Kymab Limited Chimaeric surrogate light chains (slc) comprising human vpreb
GB201117313D0 (en) 2011-10-07 2011-11-16 Gt Biolog Ltd Bacterium for use in medicine
US9253965B2 (en) 2012-03-28 2016-02-09 Kymab Limited Animal models and therapeutic molecules
US11021737B2 (en) 2011-12-22 2021-06-01 President And Fellows Of Harvard College Compositions and methods for analyte detection
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
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
GB2502127A (en) 2012-05-17 2013-11-20 Kymab Ltd Multivalent antibodies and in vivo methods for their production
US10251377B2 (en) 2012-03-28 2019-04-09 Kymab Limited Transgenic non-human vertebrate for the expression of class-switched, fully human, antibodies
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors
WO2013163628A2 (en) 2012-04-27 2013-10-31 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
PT2800811T (en) 2012-05-25 2017-08-17 Univ 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
HUE030799T2 (en) 2012-07-11 2017-06-28 Sangamo Biosciences Inc Methods and compositions for the treatment of lysosomal storage diseases
CN110643600A (en) 2012-10-23 2020-01-03 基因工具股份有限公司 System for cutting target DNA and use thereof
ES2769310T3 (en) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modification and regulation of the genome based on CRISPR
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931892B1 (en) 2012-12-12 2018-09-12 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
MX2015007550A (en) 2012-12-12 2017-02-02 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications.
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201912327SA (en) 2012-12-12 2020-02-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme 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
EP3825401A1 (en) 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
EP3553174A1 (en) * 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
WO2014113493A1 (en) * 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
EP3578666A1 (en) 2013-03-12 2019-12-11 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
ES2901396T3 (en) 2013-03-14 2022-03-22 Caribou Biosciences Inc Nucleic Acid Targeting Nucleic Acid Compositions and Methods
US11332719B2 (en) * 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
CN110540991B (en) * 2013-03-15 2023-10-24 通用医疗公司 Enhancement of specificity of RNA-guided genome editing using truncated guide RNA (tru-gRNA)
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
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
US9788534B2 (en) 2013-03-18 2017-10-17 Kymab Limited Animal models and therapeutic molecules
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
GB201306536D0 (en) 2013-04-10 2013-05-22 Gt Biolog Ltd Polypeptide and immune modulation
JP6411463B2 (en) 2013-04-16 2018-10-24 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Targeted modification of rat genome
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
CN116083487A (en) * 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 Methods and compositions for treating genetic conditions
JP7065564B2 (en) * 2013-05-29 2022-05-12 セレクティス Methods for Providing Accurate DNA Cleavage Using CAS9 Nickase Activity
MY177814A (en) * 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) * 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3011034B1 (en) 2013-06-17 2019-08-07 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN105492611A (en) 2013-06-17 2016-04-13 布罗德研究所有限公司 Optimized CRISPR-CAS double nickase systems, methods and compositions for sequence manipulation
RU2716420C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery and use of systems of crispr-cas, vectors and compositions for targeted action and therapy in liver
JP2016528890A (en) * 2013-07-09 2016-09-23 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 Therapeutic use of genome editing using the CRISPR / Cas system
BR112016000571B1 (en) 2013-07-10 2023-12-26 President And Fellows Of Harvard College IN VITRO METHODS FOR MODULATING EXPRESSION AND FOR ALTERING ONE OR MORE TARGET NUCLEIC ACIDS IN A CELL SIMULTANEOUSLY WITH REGULATING THE EXPRESSION OF ONE OR MORE TARGET NUCLEIC ACIDS IN A CELL AS WELL AS A YEAST OR BACTERIAL CELL COMPRISING NUCLEIC ACIDS
EP3971287A1 (en) * 2013-07-11 2022-03-23 ModernaTX, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
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
MX2016002118A (en) 2013-08-22 2016-06-28 Du Pont Plant genome modification using guide rna/cas endonuclease systems and methods of use.
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
WO2015040402A1 (en) * 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
TW201546284A (en) 2013-10-01 2015-12-16 Kymab Ltd Animal models and therapeutic molecules
WO2015051169A2 (en) 2013-10-02 2015-04-09 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
AU2014346559B2 (en) 2013-11-07 2020-07-09 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
EP3460063B1 (en) 2013-12-11 2024-03-13 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
RU2685914C1 (en) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Methods and compositions for genome targeted modification
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
CN106536729A (en) * 2013-12-12 2017-03-22 布罗德研究所有限公司 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CN105899657A (en) 2013-12-12 2016-08-24 布罗德研究所有限公司 Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
MX2016007328A (en) 2013-12-12 2017-07-19 Broad Inst Inc Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing.
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
KR20160089530A (en) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology 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 (en) * 2014-01-31 2020-10-15 Factor Bioscience Inc Synthetic RNA for use in the treatment of dystrophic epidermolysis bullosa
PL3105328T3 (en) 2014-02-11 2020-10-19 The Regents Of The University Of Colorado, A Body Corporate Crispr enabled multiplexed genome engineering
MX2016010781A (en) 2014-02-18 2017-02-15 Univ Duke Compositions for the inactivation of virus replication and methods of making and using the same.
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
JP2017508457A (en) 2014-02-27 2017-03-30 ザ・ブロード・インスティテュート・インコーポレイテッド T cell balance gene expression, composition and method of use thereof
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015143078A1 (en) 2014-03-18 2015-09-24 University 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
EP3134522B1 (en) 2014-04-25 2021-10-06 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
EP3140403A4 (en) * 2014-05-09 2017-12-20 Université Laval Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
WO2015178343A1 (en) * 2014-05-20 2015-11-26 国立大学法人北海道大学 LIPID MEMBRANE STRUCTURE FOR siRNA INTRACELLULAR DELIVERY
EP3712254A1 (en) 2014-05-28 2020-09-23 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
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
PL3152312T3 (en) 2014-06-06 2020-08-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for modifying a targeted locus
EP3157573A4 (en) 2014-06-19 2018-02-21 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
AU2015280069B2 (en) 2014-06-23 2021-08-12 The General Hospital Corporation Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq)
PT3155099T (en) * 2014-06-23 2018-04-20 Regeneron Pharma Nuclease-mediated dna assembly
WO2015200901A1 (en) 2014-06-26 2015-12-30 The Trustees Of Columbia University In The City Of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
MX2016017317A (en) 2014-06-26 2017-08-24 Regeneron Pharma Methods and compositions for targeted genetic modifications and methods of use.
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
US10081816B1 (en) * 2014-07-03 2018-09-25 Nant Holdings Ip, Llc Mechanical transfection devices and methods
US10760040B1 (en) 2014-07-03 2020-09-01 NanoCav, LLC Mechanical transfection devices and methods
EP2966170A1 (en) * 2014-07-10 2016-01-13 Heinrich-Pette-Institut Leibniz-Institut für experimentelle Virologie-Stiftung bürgerlichen Rechts - HBV inactivation
AU2015288157A1 (en) 2014-07-11 2017-01-19 E. I. Du Pont De Nemours And Company Compositions and methods for producing plants resistant to glyphosate herbicide
CN106795524A (en) * 2014-07-11 2017-05-31 先锋国际良种公司 Change agronomy character and its application method using guide RNA/CAS endonuclease systems
AU2015289656A1 (en) 2014-07-16 2017-02-16 Modernatx, Inc. Circular polynucleotides
US10077453B2 (en) 2014-07-30 2018-09-18 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
WO2016025759A1 (en) * 2014-08-14 2016-02-18 Shen Yuelei Dna knock-in system
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
EP3186376B1 (en) 2014-08-27 2019-03-20 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
CN108064129A (en) 2014-09-12 2018-05-22 纳幕尔杜邦公司 The generation in the site-specific integration site of complex character locus and application method in corn and soybean
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
WO2016049024A2 (en) * 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CN104293831A (en) * 2014-09-28 2015-01-21 上海云舜生物技术有限公司 Method for establishing hypertension mouse model and application of hypertension mouse model
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
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
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
CA2964114A1 (en) * 2014-10-09 2016-04-14 Anthrogenesis Corporation Placenta-derived adherent cell exosomes and uses thereof
US10428310B2 (en) 2014-10-15 2019-10-01 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
CN107208086A (en) 2014-10-17 2017-09-26 霍华德休斯医学研究所 genomic probe
US11584916B2 (en) 2014-10-17 2023-02-21 Children's Hospital Medical Center Method of making in vivo human small intestine organoids from pluripotent stem cells
RU2738421C2 (en) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Versions of recombinant aav and use thereof
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
WO2016069910A1 (en) 2014-10-29 2016-05-06 Massachusetts Eye And Ear Infirmary Methods for efficient delivery of therapeutic molecules in vitro and in vivo
JP6788584B2 (en) * 2014-10-31 2020-11-25 マサチューセッツ インスティテュート オブ テクノロジー Massively Parallel Combinatorial Genetics on CRISPR
RU2020109343A (en) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. AADC POLINUCLEOTIDES FOR TREATMENT OF PARKINSON'S DISEASE
US11680268B2 (en) 2014-11-07 2023-06-20 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
MX2017006216A (en) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als).
DK3218386T3 (en) 2014-11-14 2021-06-07 Voyager Therapeutics Inc MODULATORY POLYNUCLEOTID
KR102531016B1 (en) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs
US11168369B2 (en) 2014-11-25 2021-11-09 The Brigham And Women's Hospital, Inc. Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease
US20180057839A1 (en) 2014-11-26 2018-03-01 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
EP3227447B1 (en) 2014-12-03 2024-04-24 Agilent Technologies, Inc. Guide rna with chemical modifications
AU2015360502A1 (en) 2014-12-10 2017-06-29 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3230452A1 (en) 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
RU2707137C2 (en) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Methods and compositions for targeted genetic modification by single-step multiple targeting
WO2016100857A1 (en) 2014-12-19 2016-06-23 Regeneron Pharmaceuticals, Inc. Stem cells for modeling type 2 diabetes
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
EP3400953A1 (en) 2014-12-23 2018-11-14 4D Pharma Research Limited Pirin polypeptide and immune modulation
KR102523805B1 (en) 2014-12-23 2023-04-20 4디 파마 리서치 리미티드 Immune modulation
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology 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
WO2016109840A2 (en) 2014-12-31 2016-07-07 Synthetic Genomics, Inc. Compositions and methods for high efficiency in vivo genome editing
WO2016114972A1 (en) 2015-01-12 2016-07-21 The Regents Of The University Of California Heterodimeric cas9 and methods of use thereof
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
ES2955957T3 (en) 2015-01-28 2023-12-11 Caribou Biosciences Inc CRISPR hybrid DNA/RNA polynucleotides and procedures for use
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
US10584321B2 (en) * 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016138488A2 (en) 2015-02-26 2016-09-01 The Broad Institute Inc. T cell balance gene expression, compositions of matters and methods of use thereof
US11261466B2 (en) 2015-03-02 2022-03-01 Sinai Health System Homologous recombination factors
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CN104673816A (en) * 2015-03-05 2015-06-03 广东医学院 PCr-NHEJ (non-homologous end joining) carrier as well as construction method of pCr-NHEJ carrier and application of pCr-NHEJ carrier in site-specific knockout of bacterial genes
CN106032540B (en) * 2015-03-16 2019-10-25 中国科学院上海生命科学研究院 Gland relevant viral vector building of CRISPR/Cas9 endonuclease enzyme system and application thereof
GB2536650A (en) 2015-03-24 2016-09-28 Augmedics Ltd Method and system for combining video-based and optic-based augmented reality in a near eye display
US20180066253A1 (en) * 2015-03-26 2018-03-08 Yale University Methods and compositions for modifying endothelial cells
EP3553178A1 (en) 2015-03-27 2019-10-16 E. I. du Pont de Nemours and Company Soybean u6 small nuclear rna gene promoters and their use in constitutive expression of small rna genes in plants
WO2016161260A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
ES2959608T3 (en) * 2015-04-03 2024-02-27 Dana Farber Cancer Inst Inc Composition and editing methods of the B cell genome
JP6873911B2 (en) 2015-04-06 2021-05-19 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー In vitro methods to induce gene regulation of target nucleic acids in primary cells
GB201506509D0 (en) 2015-04-16 2015-06-03 Univ Wageningen Nuclease-mediated genome editing
CN107690480B (en) 2015-04-24 2022-03-22 爱迪塔斯医药公司 Evaluation of CAS9 molecule/guide RNA molecule complexes
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
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
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
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
WO2016182959A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016187717A1 (en) * 2015-05-26 2016-12-01 Exerkine Corporation Exosomes useful for genome editing
CA3000155A1 (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
UY36703A (en) 2015-06-02 2016-12-30 Monsanto Technology Llc COMPOSITIONS AND METHODS FOR THE ADMINISTRATION OF A POLINUCLEOTIDE ON A PLANT
CN108368502B (en) 2015-06-03 2022-03-18 内布拉斯加大学董事委员会 DNA editing using single-stranded DNA
GB201509578D0 (en) * 2015-06-03 2015-07-15 Univ Singapore Vectors
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
PT3240554T (en) 2015-06-15 2019-11-04 4D Pharma Res Ltd Blautia stercosis and wexlerae for use in treating inflammatory and autoimmune diseases
MA41060B1 (en) 2015-06-15 2019-11-29 4D Pharma Res Ltd Compositions comprising bacterial strains
DK3206700T3 (en) 2015-06-15 2019-08-05 4D Pharma Res Ltd COMPOSITIONS COMPREHENSIVE BAKERY STUES
PT3307288T (en) 2015-06-15 2019-10-17 4D Pharma Res Ltd Compositions comprising bacterial strains
MA41010B1 (en) 2015-06-15 2020-01-31 4D Pharma Res Ltd Compositions comprising bacterial strains
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
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
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
EP3800255A3 (en) * 2015-06-18 2021-06-23 Robert D. Bowles Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
EP3666895A1 (en) 2015-06-18 2020-06-17 The Broad Institute, Inc. Novel crispr enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
RU2752834C2 (en) 2015-06-18 2021-08-09 Те Брод Инститьют, Инк. Crispr enzyme mutations reducing non-targeted effects
EP3316159A4 (en) 2015-06-25 2019-08-14 Advanced Telecommunications Research Institute International Prediction device based on multiple organ-related system and prediction program
EP3313416A4 (en) * 2015-06-26 2019-02-20 The Trustees of Columbia University in the City of New York Genetically modified ips cells that carry a marker to report expression of neurogenin3, tph2, foxo1 and/or insulin genes
WO2017004261A1 (en) 2015-06-29 2017-01-05 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
US20180360994A1 (en) * 2015-07-25 2018-12-20 Habib Frost System, device and a method for providing a therapy or a cure for cancer and other pathological states
WO2017019867A1 (en) * 2015-07-28 2017-02-02 Danisco Us Inc Genome editing systems and methods of use
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
CN108472314A (en) 2015-07-31 2018-08-31 明尼苏达大学董事会 The cell and therapy of modification
EP3334746B1 (en) 2015-08-14 2021-11-24 The University Of Sydney Connexin 45 inhibition for therapy
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
CA2996001A1 (en) * 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
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
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
IL241462A0 (en) 2015-09-10 2015-11-30 Yeda Res & Dev Heterologous engineering of betalain pigments in plants
AU2016319110B2 (en) 2015-09-11 2022-01-27 The General Hospital Corporation Full interrogation of nuclease DSBs and sequencing (FIND-seq)
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
JP2018529353A (en) 2015-09-30 2018-10-11 ザ ジェネラル ホスピタル コーポレイション Comprehensive in vitro reporting of cleavage events by sequencing (CIRCLE-seq)
WO2017062754A1 (en) * 2015-10-07 2017-04-13 New York University Compositions and methods for enhancing crispr activity by polq inhibition
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3365441A1 (en) 2015-10-22 2018-08-29 The Broad Institute Inc. Type vi-b crispr enzymes and systems
IL294014B1 (en) 2015-10-23 2024-03-01 Harvard College Nucleobase editors and uses thereof
US11492670B2 (en) 2015-10-27 2022-11-08 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
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
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11092607B2 (en) 2015-10-28 2021-08-17 The Board Institute, Inc. Multiplex analysis of single cell constituents
WO2017075294A1 (en) 2015-10-28 2017-05-04 The Board Institute Inc. Assays for massively combinatorial perturbation profiling and cellular circuit reconstruction
AU2016349288A1 (en) 2015-11-03 2018-05-31 President And Fellows Of Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
US20180327781A1 (en) * 2015-11-09 2018-11-15 Seattle Children's Hospital (dba Seattle Children's Research Institute) Novel rna-based vector system for transient and stable gene expression
WO2017083722A1 (en) * 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
EP3374502B1 (en) 2015-11-13 2021-10-27 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
EP3377042A4 (en) 2015-11-16 2019-05-29 The 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
AU2016355178B9 (en) 2015-11-19 2019-05-30 Massachusetts Institute Of Technology Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity
KR101914245B1 (en) 2015-11-20 2018-11-02 4디 파마 리서치 리미티드 Composition Containing Bacterial Strain
GB201520497D0 (en) 2015-11-20 2016-01-06 4D Pharma Res Ltd Compositions comprising bacterial strains
JP2019500899A (en) 2015-11-23 2019-01-17 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Cellular RNA tracking and manipulation through nuclear delivery of CRISPR / Cas9
US20210395799A1 (en) * 2015-11-25 2021-12-23 Integrated Dna Technologies, Inc. Methods for variant detection
US20170145486A1 (en) * 2015-11-25 2017-05-25 Integrated Dna Technologies, Inc. Methods for variant detection
US20190233814A1 (en) 2015-12-18 2019-08-01 The Broad Institute, Inc. Novel crispr enzymes and systems
CN105624191A (en) * 2015-12-24 2016-06-01 江苏大学 Method for establishing CYP2D1 gene knockout rat model
CN105624196A (en) * 2015-12-24 2016-06-01 江苏大学 Method for establishing CYP2C11 gene knockout rat model
CN105548420B (en) * 2015-12-30 2019-08-02 上海泽润生物科技有限公司 The detection method of MOPS residual quantity
ES2944597T3 (en) 2015-12-30 2023-06-22 Novartis Ag Enhanced Efficacy Immune Effector Cell Therapies
US9856497B2 (en) 2016-01-11 2018-01-02 The Board Of Trustee Of The Leland Stanford Junior University Chimeric proteins and methods of regulating gene expression
MX2018008345A (en) 2016-01-11 2018-12-06 Univ Leland Stanford Junior Chimeric proteins and methods of immunotherapy.
US10053693B2 (en) 2016-01-19 2018-08-21 Mubin I. Syed Method for controlling obesity using minimally invasive means
JP6914274B2 (en) 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 Crystal structure of CRISPRCPF1
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
AU2017219605B2 (en) * 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
US20190249172A1 (en) 2016-02-18 2019-08-15 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
CN116763942A (en) 2016-02-18 2023-09-19 宾州研究基金会 Intra-brain generation of GABAergic neurons
US20190144942A1 (en) 2016-02-22 2019-05-16 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
CN114908093A (en) * 2016-02-26 2022-08-16 朗泽科技新西兰有限公司 CRISPR/CAS system for C1 immobilized bacteria
MA42560B1 (en) 2016-03-04 2019-07-31 4D Pharma Plc Compositions comprising bacterial strains of blautia for the treatment of visceral hypersensitivity
GB201612191D0 (en) 2016-07-13 2016-08-24 4D Pharma Plc Compositions comprising bacterial strains
EP3429567B1 (en) 2016-03-16 2024-01-10 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
US11427861B2 (en) 2016-03-17 2022-08-30 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
EP3433363A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017170803A1 (en) * 2016-03-29 2017-10-05 株式会社国際電気通信基礎技術研究所 Pharmaceutical composition or food composition, and method for assessing effect of active ingredient in vivo
JP7245651B2 (en) * 2016-03-30 2023-03-24 インテリア セラピューティクス，インコーポレイテッド Lipid Nanoparticle Formulations for CRISPR/CAS Components
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
KR102670601B1 (en) * 2016-04-19 2024-05-29 더 브로드 인스티튜트, 인코퍼레이티드 The novel CRISPR enzyme and system
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
WO2017189308A1 (en) 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017189525A1 (en) 2016-04-25 2017-11-02 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
WO2017190091A1 (en) 2016-04-29 2017-11-02 Vindico Nanobiotechnology, Llc Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
EP4177335A1 (en) 2016-05-05 2023-05-10 Children's Hospital Medical Center Methods for the in vitro manufacture of gastric fundus tissue and compositions related to same
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc Modulatory polynucleotides
EP3457840B1 (en) 2016-05-20 2024-04-10 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US11788083B2 (en) 2016-06-17 2023-10-17 The Broad Institute, Inc. Type VI CRISPR orthologs and systems
AU2017280353B2 (en) 2016-06-24 2021-11-11 Inscripta, Inc. Methods for generating barcoded combinatorial libraries
EP3474849A4 (en) 2016-06-27 2020-07-29 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
TW201821093A (en) 2016-07-13 2018-06-16 英商４Ｄ製藥有限公司 Compositions comprising bacterial strains
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
WO2018021855A1 (en) * 2016-07-28 2018-02-01 기초과학연구원 Pharmaceutical composition for treating ocular diseases, containing 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
AU2017305404B2 (en) 2016-08-02 2023-11-30 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
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
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
RU2022103870A (en) * 2016-08-12 2022-03-04 Тулджен Инкорпорейтед MODIFIED IMMUNOREGULATORY ELEMENT AND CHANGE OF IMMUNE THROUGH THIS ELEMENT
US20200283743A1 (en) * 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11630103B2 (en) 2016-08-17 2023-04-18 The Broad Institute, Inc. Product and methods useful for modulating and evaluating immune responses
WO2018035250A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Methods for identifying class 2 crispr-cas systems
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and 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
WO2018039145A1 (en) 2016-08-20 2018-03-01 Avellino Lab Usa, Inc. Single guide rna, 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
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
IL247752A0 (en) 2016-09-11 2016-11-30 Yeda Res & Dev Compositions and methods for regulating gene expression for targeted mutagenesis
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CN109715804A (en) * 2016-09-23 2019-05-03 帝斯曼知识产权资产管理有限公司 Guide RNA expression system for host cell
US20190225974A1 (en) 2016-09-23 2019-07-25 BASF Agricultural Solutions Seed US LLC Targeted genome optimization in plants
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
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
CN110462034A (en) * 2016-10-07 2019-11-15 综合Dna技术公司 Streptococcus pyogenes CAS9 mutated gene and polypeptide encoded by it
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
KR20190075964A (en) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 AAV capsid design
CN110214180A (en) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 The AAV of nucleobase editing machine is delivered
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
AU2017346885B2 (en) 2016-10-18 2021-10-21 Intima Bioscience, Inc. Tumor infiltrating lymphocytes and methods of therapy
CA3041517A1 (en) 2016-10-19 2018-04-26 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
AU2017358122B2 (en) * 2016-11-14 2022-12-22 Toolgen Incorporated Artificially engineered SC function control system
SG10202105768WA (en) 2016-12-05 2021-06-29 Childrens Hospital Med Ct Colonic organoids and methods of making and using same
PT3551753T (en) 2016-12-09 2022-09-02 Harvard College Crispr effector system based diagnostics
GB201621123D0 (en) 2016-12-12 2017-01-25 4D Pharma Plc Compositions comprising bacterial strains
KR20210035022A (en) 2016-12-14 2021-03-31 리간달 인코포레이티드 Compositions and methods for delivery of nucleic acid and/or protein loads
GB2605925B (en) * 2016-12-23 2023-02-22 Harvard College Gene editing of PCSK9
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11733235B2 (en) 2016-12-27 2023-08-22 Chugai Seiyaku Kabushiki Kaisha Method for evaluating immunogenicity of test substance
CN106632693B (en) * 2017-01-19 2021-05-25 上海科技大学 SpyCas9 protein with multiple nuclear localization sequences and application thereof
KR102619197B1 (en) 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 HSD17B13 variant and its uses
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Compositions and methods for the treatment of hemoglobinopathies
WO2018147343A1 (en) * 2017-02-07 2018-08-16 Edigene Corporation Method of treating diseases associated with elevated kras expression using crispr-gndm system
WO2018152418A1 (en) * 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
EP3589751A4 (en) * 2017-03-03 2021-11-17 The Regents of The University of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (en) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ Base-editing factor from cytosine to guanine
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3596207B1 (en) 2017-03-15 2023-12-20 The Broad Institute, Inc. Novel cas13b orthologues crispr enzymes and systems
US11104937B2 (en) 2017-03-15 2021-08-31 The Broad Institute, Inc. CRISPR effector system based diagnostics
US11174515B2 (en) 2017-03-15 2021-11-16 The Broad Institute, Inc. CRISPR effector system based diagnostics
CN111108220A (en) 2017-03-15 2020-05-05 博德研究所 CRISPR-Effector System-based diagnostics for Virus detection
US20200115753A1 (en) 2017-03-17 2020-04-16 Massachusetts Institute Of Technology 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
MX2019011272A (en) 2017-03-22 2019-10-24 Novartis Ag Compositions and methods for immunooncology.
KR20190130613A (en) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Nucleobase edits comprising nucleic acid programmable DNA binding proteins
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
WO2018183921A1 (en) 2017-04-01 2018-10-04 The Broad Institute, Inc. Methods and compositions for detecting and modulating an immunotherapy resistance gene signature in cancer
WO2018191520A1 (en) 2017-04-12 2018-10-18 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
US20200071773A1 (en) 2017-04-12 2020-03-05 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
US11618928B2 (en) 2017-04-12 2023-04-04 The Broad Institute, Inc. CRISPR effector system based diagnostics for malaria detection
KR20200006054A (en) 2017-04-12 2020-01-17 더 브로드 인스티튜트, 인코퍼레이티드 New Type VI CRISPR Orthologs and Systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel 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
EP3612629A1 (en) 2017-04-18 2020-02-26 The Broad Institute, Inc. Compositions for detecting secretion and methods of use
EP3612551A4 (en) 2017-04-21 2020-12-30 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
US20200384115A1 (en) 2017-04-21 2020-12-10 The Broad Institute , Inc. Targeted delivery to beta cells
WO2018201086A1 (en) 2017-04-28 2018-11-01 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
US11696954B2 (en) * 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
US11072816B2 (en) 2017-05-03 2021-07-27 The Broad Institute, Inc. Single-cell proteomic assay using aptamers
CN110913866A (en) 2017-05-05 2020-03-24 沃雅戈治疗公司 Compositions and methods for treating Amyotrophic Lateral Sclerosis (ALS)
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
US20210278416A1 (en) 2017-05-09 2021-09-09 The Broad Institute, Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
AU2018265022A1 (en) 2017-05-10 2019-11-21 The Regents Of The University Of California Directed editing of cellular RNA via nuclear delivery of CRISPR/Cas9
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
KR102607330B1 (en) * 2017-05-16 2023-11-28 위스콘신 얼럼나이 리서어치 화운데이션 Systems and methods for batch patterning of molecular structures
JP7364472B2 (en) 2017-05-18 2023-10-18 ザ・ブロード・インスティテュート・インコーポレイテッド Systems, methods, and compositions for targeted nucleic acid editing
TW201907928A (en) 2017-05-22 2019-03-01 英商4D製藥研究有限公司 a composition comprising a bacterial strain
MA41708A (en) 2017-05-24 2020-04-08 4D Pharma Res Ltd COMPOSITIONS CONTAINING BACTERIAL STRAINS
JP2020521446A (en) 2017-05-25 2020-07-27 ザ ジェネラル ホスピタル コーポレイション Bipartite base editor (BBE) structure and type II-C-CAS9 zinc finger editing
WO2018226762A1 (en) 2017-06-05 2018-12-13 Fred Hutchinson Cancer Research Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
AU2018279829B2 (en) 2017-06-09 2024-01-04 Editas Medicine, Inc. Engineered Cas9 nucleases
US11897953B2 (en) 2017-06-14 2024-02-13 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
ES2841902T3 (en) 2017-06-14 2021-07-12 4D Pharma Res Ltd Compositions comprising bacterial strains
MA49425A (en) 2017-06-14 2020-04-22 4D Pharma Res Ltd COMPOSITIONS CONTAINING BACTERIAL STRAINS
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
WO2018237107A1 (en) 2017-06-23 2018-12-27 University Of Kentucky Research Foundation Method
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
CN110869031A (en) * 2017-06-28 2020-03-06 南佛罗里达大学 Modified UBE3A gene for use in methods of gene therapy for Angel syndrome
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. Adeno-associated viral vectors for gene therapy
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP7210028B2 (en) * 2017-07-18 2023-01-23 国立大学法人京都大学 Gene mutation introduction method
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
BR102017016440A2 (en) 2017-07-31 2019-03-19 Universidade Federal Do Rio Grande Do Sul COMPOSITION FOR CENTRAL NERVOUS SYSTEM GENE THERAPY, OBTAINING AND USE PROCESS
BR112019027673A2 (en) 2017-07-31 2020-09-15 Regeneron Pharmaceuticals, Inc. non-human animal, and, methods to test the recombination induced by crispr / cas and to optimize the ability of crispr / cas
JP2020533957A (en) 2017-07-31 2020-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． CRISPR Reporter Non-Human Animals and Their Use
KR20200032693A (en) * 2017-07-31 2020-03-26 리제너론 파마슈티칼스 인코포레이티드 Cas-transformed mouse embryonic stem cells and mice and uses thereof
WO2019040650A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
EP3681501A1 (en) * 2017-09-15 2020-07-22 The Board of Trustees of the Leland Stanford Junior University Mitochondrial aldehyde dehydrogenase-2 modulators for protecting, expanding and increasing the potency of hematopoietic stem cells
CA3073848A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP7397488B2 (en) 2017-09-22 2023-12-13 ユニバーシティ オブ マサチューセッツ SOD1 dual expression vector and its use
CA3077307A1 (en) 2017-09-27 2019-04-04 Pioneer Hi-Bred International, Inc. Soil application of crop protection agents
WO2019066535A1 (en) * 2017-09-28 2019-04-04 주식회사 탠덤 Novel recombinant plasma membrane-based vesicle, for treating cancer
AU2018338790B2 (en) 2017-09-29 2022-09-15 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus and methods of use
KR20200058509A (en) 2017-09-29 2020-05-27 인텔리아 테라퓨틱스, 인크. Composition and method for TTR gene editing and treatment of ATTR amyloidosis
AU2018346530A1 (en) 2017-10-04 2020-04-30 Massachusetts Institute Of Technology CRISPR effector system based diagnostics
EP3692152A4 (en) 2017-10-04 2021-12-01 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US11725228B2 (en) 2017-10-11 2023-08-15 The General Hospital Corporation Methods for detecting site-specific and spurious genomic deamination induced by base editing technologies
US11680296B2 (en) 2017-10-16 2023-06-20 Massachusetts Institute Of Technology Mycobacterium tuberculosis host-pathogen interaction
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
EP3697895A4 (en) 2017-10-20 2021-12-08 Fred Hutchinson Cancer Research Center Systems and methods to produce b cells genetically modified to express selected antibodies
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US20210180053A1 (en) 2017-11-01 2021-06-17 Novartis Ag Synthetic rnas and methods of use
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
WO2019099943A1 (en) 2017-11-16 2019-05-23 Astrazeneca Ab Compositions and methods for improving the efficacy of cas9-based knock-in strategies
US11332736B2 (en) 2017-12-07 2022-05-17 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
EP3725092A4 (en) 2017-12-14 2021-09-22 FloDesign Sonics, Inc. Acoustic transducer drive and controller
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 (en) 2018-01-12 2020-12-08 Basf Se PROTEIN, ISOLATED NUCLEIC ACID, RECOMBINANT GENE, VECTOR, HOSTING CELL, PLANT, PART OF PLANT OR WHEAT SEED, METHODS OF PRODUCTION, WHEAT PRODUCT, FLOUR, WHOLE MUSHROOM, STARCH, MUSHROOMS AND MUSHROOMS. AND / OR SELECTING A WHEAT PLANT
WO2019143675A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
BR112020013626A2 (en) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated quinoxalinone compounds, compositions, methods and kits to increase genome editing efficiency
EA202091708A1 (en) 2018-01-17 2020-11-10 Вертекс Фармасьютикалз Инкорпорейтед DNA PC INHIBITORS
BR112020015093A2 (en) 2018-01-29 2020-12-08 The Broad Institute, Inc. DIAGNOSIS BASED ON THE CRISPR EFFECTOR SYSTEM
CN112203676A (en) * 2018-02-02 2021-01-08 宾州研究基金会 Methods and materials for treating brain injury
EP3735248A4 (en) * 2018-02-09 2021-11-17 Empirico Inc. Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions
US20210054370A1 (en) * 2018-02-27 2021-02-25 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
AU2019236210A1 (en) 2018-03-14 2020-09-10 Arbor Biotechnologies, Inc. Novel CRISPR DNA targeting enzymes and systems
KR101889683B1 (en) * 2018-03-14 2018-08-17 유병탁 Constructing method for steel frame structure
WO2019178428A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna and rna targeting enzymes and systems
US20190284553A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
KR102647714B1 (en) 2018-03-19 2024-03-18 리제너론 파마슈티칼스 인코포레이티드 Transcriptional regulation in animals using the CRISPR/Cas system
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
WO2019204378A1 (en) 2018-04-17 2019-10-24 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
US20210071240A1 (en) 2018-04-19 2021-03-11 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded dna
HUE059223T2 (en) 2018-04-24 2022-10-28 Kws Saat Se & Co Kgaa Plants with improved digestibility and marker haplotypes
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
US20210147831A1 (en) 2018-04-27 2021-05-20 The Broad Institute, Inc. Sequencing-based proteomics
EP3787693A4 (en) * 2018-04-30 2022-03-09 Oregon Health & Science University Methods of gene therapy
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
WO2019213660A2 (en) 2018-05-04 2019-11-07 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
EP3794130A4 (en) 2018-05-16 2022-07-27 Synthego Corporation Methods and systems for guide rna design and use
US20210198664A1 (en) 2018-05-16 2021-07-01 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
CN108707628B (en) * 2018-05-28 2021-11-23 上海海洋大学 Preparation method of zebra fish notch2 gene mutant
WO2019232542A2 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
US20210147915A1 (en) 2018-06-26 2021-05-20 The Broad Institute, Inc. Crispr/cas and transposase based amplification compositions, systems and methods
SG11202012786RA (en) 2018-06-26 2021-01-28 Massachusetts Inst Technology Crispr effector system based amplification methods, systems, and diagnostics
AU2019291827A1 (en) 2018-06-26 2020-12-24 Massachusetts Institute Of Technology Crispr double nickase based amplification compositions, systems, and methods
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
US20210285009A1 (en) 2018-07-13 2021-09-16 The Regents Of The University Of California Retrotransposon-based delivery vehicle and methods of use thereof
US20210284981A1 (en) * 2018-07-24 2021-09-16 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
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
EP3833761A1 (en) 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems
KR102103103B1 (en) * 2018-08-16 2020-04-21 (주)라트바이오 Transgenic animals and transgenic embryos producing an engineered nuclease
WO2020068304A2 (en) 2018-08-20 2020-04-02 The Broad Institute, Inc. Inhibitors of rna-guided nuclease target binding and uses thereof
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US20210355522A1 (en) 2018-08-20 2021-11-18 The Broad Institute, Inc. Inhibitors of rna-guided nuclease activity and uses thereof
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
EP3847650A1 (en) 2018-09-06 2021-07-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP4268831A3 (en) 2018-09-12 2024-05-22 Fred Hutchinson Cancer Center Reducing cd33 expression to selectively protect therapeutic cells
CA3113095A1 (en) 2018-09-18 2020-03-26 Vnv Newco Inc. Arc-based capsids and uses thereof
US20210396756A1 (en) 2018-10-03 2021-12-23 The Broad Institute, Inc. Crispr effector system based diagnostics for hemorrhagic fever detection
WO2020077135A1 (en) 2018-10-10 2020-04-16 Dana-Farber Cancer Institute, Inc. Modulating resistance to bcl-2 inhibitors
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
WO2020081730A2 (en) 2018-10-16 2020-04-23 Massachusetts Institute Of Technology Methods and compositions for modulating microenvironment
CN109295081B (en) * 2018-10-17 2020-06-12 中国人民解放军第四军医大学 LDLR-Lamp2b fusion gene, expression vector and application thereof
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US20210388389A1 (en) 2018-10-30 2021-12-16 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
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
CN113832185A (en) * 2018-11-13 2021-12-24 四川横竖生物科技股份有限公司 Vector and reagent for constructing hNPY and hAGRP gene overexpression chimeric animal model and application
KR20210104698A (en) 2018-11-14 2021-08-25 더 브로드 인스티튜트, 인코퍼레이티드 Droplet diagnosis system and method based on CRISPR system
BR112021009441A2 (en) 2018-11-14 2021-11-23 Massachusetts Inst Technology Multiplexing highly evolving viral variants with Sherlock
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
WO2020118110A1 (en) * 2018-12-05 2020-06-11 Fred Hutchinson Cancer Research Center Reduced and minimal manipulation manufacturing of genetically-modified cells
KR20200071198A (en) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Development of new adoptive T cell immunotherapy by modification of Nrf2 expression
WO2020124050A1 (en) 2018-12-13 2020-06-18 The Broad Institute, Inc. Tiled assays using crispr-cas based detection
WO2020123887A2 (en) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
JP7449291B2 (en) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Nuclease-mediated repeat expansion
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US20220064635A1 (en) * 2019-01-07 2022-03-03 Emendobio Inc. Crispr compositions and methods for promoting gene editing of adenosine deaminase 2 (ada2)
US11116778B2 (en) 2019-01-15 2021-09-14 Empirico Inc. Prodrugs of ALOX-15 inhibitors and methods of using the same
GB201900940D0 (en) 2019-01-23 2019-03-13 British American Tobacco Investments Ltd Method
US20220119871A1 (en) 2019-01-28 2022-04-21 The Broad Institute, Inc. In-situ spatial transcriptomics
US11965159B2 (en) 2019-01-29 2024-04-23 The Broad Institute, Inc. Compositions and methods for regulating proteins and nucleic acids activities
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
US20220145330A1 (en) 2019-02-10 2022-05-12 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glads Modified mitochondrion and methods of use thereof
WO2020168075A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
CN116497067A (en) 2019-02-13 2023-07-28 比姆医疗股份有限公司 Compositions and methods for treating heme lesions
WO2020172343A2 (en) 2019-02-19 2020-08-27 Massachusetts Institute Of Technology Methods for treating injuries
WO2020181101A1 (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
US20220154282A1 (en) 2019-03-12 2022-05-19 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
WO2020186235A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate intestinal innate lymphoid cells
US20220154258A1 (en) 2019-03-14 2022-05-19 The Broad Institute, Inc. Crispr effector system based multiplex diagnostics
WO2020186223A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Sherlock assays for tick-borne diseases
WO2020186213A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2020190932A1 (en) 2019-03-18 2020-09-24 Regeneron Pharmaceuticals, Inc. Crispr/cas screening platform to identify genetic modifiers of tau seeding or aggregation
EP3942023A1 (en) 2019-03-18 2022-01-26 The Broad Institute, Inc. Compositions and methods for modulating metabolic regulators of t cell pathogenicity
ES2969026T3 (en) 2019-03-18 2024-05-16 Regeneron Pharma CRISPR/CAS dropout detection platform to reveal genetic vulnerabilities associated with tau aggregation
WO2020191069A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Modulation of type 2 immunity by targeting clec-2 signaling
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN111733225A (en) * 2019-03-25 2020-10-02 北京大学第一医院 PRKG1 gene SNP marker for assisting accurate medication of clopidogrel
WO2020205681A1 (en) 2019-03-29 2020-10-08 Massachusetts Institute Of Technology Constructs for continuous monitoring of live cells
US20220175848A1 (en) 2019-04-01 2022-06-09 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
US20200318136A1 (en) 2019-04-03 2020-10-08 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
CN113795588A (en) 2019-04-04 2021-12-14 瑞泽恩制药公司 Methods for scar-free introduction of targeted modifications in targeting vectors
SG11202108454RA (en) 2019-04-04 2021-09-29 Regeneron Pharma Non-human animals comprising a humanized coagulation factor 12 locus
US20220213469A1 (en) 2019-04-30 2022-07-07 The Broad Institute, Inc. Methods and compositions for barcoding nucleic acid libraries and cell populations
MX2021013400A (en) * 2019-05-03 2022-04-20 Specific Biologics Inc Lipid-encapsulated dual-cleaving endonuclease for dna and gene editing.
US20230190819A1 (en) * 2019-05-11 2023-06-22 Youngsuk Yi Compositions and methods containing exosomes
US20220243287A1 (en) 2019-05-13 2022-08-04 KWS SAAT SE & Co. KGaA Drought tolerance in corn
GB201906768D0 (en) 2019-05-14 2019-06-26 British American Tobacco Investments Ltd Method
US20220249701A1 (en) 2019-05-14 2022-08-11 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
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
JP2022534202A (en) * 2019-05-21 2022-07-28 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル Expression vectors for cholesterol 24-hydrolase in the treatment of Rett syndrome
AR118995A1 (en) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa HAPLOID INDUCTION ENHANCER
US20220226464A1 (en) 2019-05-28 2022-07-21 Massachusetts Institute Of Technology Methods and compositions for modulating immune responses
US20220243178A1 (en) 2019-05-31 2022-08-04 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
CN112005960B (en) * 2019-05-31 2022-10-21 武汉科技大学 Automatic pregnant mother mouse breeding method
KR20220016869A (en) 2019-06-04 2022-02-10 리제너론 파마슈티칼스 인코포레이티드 Non-Human Animals Comprising a Humanized TTR Locus with Beta-Slip Mutations and Methods of Use
MX2021015122A (en) 2019-06-07 2022-04-06 Regeneron Pharma Non-human animals comprising a humanized albumin locus.
JP2022536606A (en) 2019-06-14 2022-08-18 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 Model of tauopathy
WO2020257514A1 (en) * 2019-06-21 2020-12-24 Children's Medical Center Corporation Methods and compositions for allele specific gene editing
AU2020298572A1 (en) 2019-07-02 2021-11-18 Fred Hutchinson Cancer Center Recombinant Ad35 vectors and related gene therapy improvements
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
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
WO2021041922A1 (en) 2019-08-30 2021-03-04 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
JP2022547570A (en) 2019-09-12 2022-11-14 ザ・ブロード・インスティテュート・インコーポレイテッド Engineered adeno-associated virus capsid
CA3153980A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
EP4031660A1 (en) 2019-09-20 2022-07-27 The Broad Institute, Inc. Novel type vi crispr enzymes and systems
US20220389451A1 (en) 2019-09-20 2022-12-08 The Broad Institute, Inc. Compositions and methods for delivering cargo to a target cell
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector
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
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
BR112022008861A2 (en) 2019-11-08 2022-08-23 Regeneron Pharma NUCLEIC ACID CONSTRUCTION, VECTOR, LIPID NANO PARTICLE, CELL, COMPOSITION FOR USE IN EXPRESSING RETINOSCHISIN, METHOD FOR INTEGRATING A CODING SEQUENCE, AND, METHOD FOR MODIFYING AN RS1 GENE
CN110684801A (en) * 2019-11-13 2020-01-14 武汉华美生物工程有限公司 Method for gene editing by transducing cas9 gene into mammalian cell
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
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
JP2023508362A (en) * 2019-12-23 2023-03-02 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア CRISPR-CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
IL294715A (en) 2020-01-23 2022-09-01 Childrens Medical Ct Corp Stroma-free t cell differentiation from human pluripotent stem cells
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
KR20240035924A (en) * 2020-02-28 2024-03-18 후이진 테라퓨틱스 씨오., 엘티디. Type vi-e and type vi-f crispr-cas system and uses thereof
CN115485385A (en) 2020-03-04 2022-12-16 瑞泽恩制药公司 Methods and compositions for sensitizing tumor cells to immunotherapy
CN111321150B (en) * 2020-03-13 2020-12-04 广西壮族自治区水产科学研究院 LvCTL4 gene, encoded protein, protein acquisition method, expression vector, recombinant bacterium and application
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US11851702B2 (en) 2020-03-23 2023-12-26 The Broad Institute, Inc. Rapid diagnostics
BR112022019713A2 (en) 2020-03-31 2022-12-20 Arbor Biotechnologies Inc COMPOSITIONS COMPRISING A CAS12I2 VARIANT POLYPEPTIDE AND USES THEREOF
WO2021202938A1 (en) 2020-04-03 2021-10-07 Creyon Bio, Inc. Oligonucleotide-based machine learning
WO2021205000A2 (en) 2020-04-09 2021-10-14 R.J. Reynolds Tobacco Company Method
WO2021211734A1 (en) * 2020-04-15 2021-10-21 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
WO2021216623A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
WO2021224633A1 (en) 2020-05-06 2021-11-11 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
MX2022014008A (en) 2020-05-08 2023-02-09 Broad Inst Inc Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence.
CN111521826A (en) * 2020-05-21 2020-08-11 江海松 Application of Notch3 protein in peripheral blood exosome as molecular marker and detection kit
CN116782762A (en) 2020-05-29 2023-09-19 科沃施种子欧洲股份两合公司 Plant haploid induction
WO2022010286A1 (en) * 2020-07-08 2022-01-13 경상국립대학교산학협력단 Donor nucleic acid used for gene correction through microhomology-mediated end joining and uses thereof
US20230321278A1 (en) * 2020-07-10 2023-10-12 President And Fellows Of Harvard College Aav vector delivery systems
CN111979241B (en) * 2020-07-23 2021-10-01 上海市第一人民医院 Method for preparing non-human mammal model of retinitis pigmentosa
WO2022018667A1 (en) 2020-07-24 2022-01-27 Pfizer Inc. Combination therapies using cdk2 and cdc25a inhibitors
AU2021338707A1 (en) 2020-09-10 2023-04-06 Monsanto Technology Llc Increasing gene editing and site-directed integration events utilizing meiotic and germline promoters
CN112159809B (en) * 2020-09-22 2021-06-22 广州瑞风生物科技有限公司 gRNA of target CTGF gene and application thereof
WO2022068912A1 (en) 2020-09-30 2022-04-07 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
WO2022188039A1 (en) 2021-03-09 2022-09-15 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
RU2749307C1 (en) * 2020-10-30 2021-06-08 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) New compact type ii cas9 nuclease from 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
IL305030A (en) 2021-02-11 2023-10-01 Arbor Biotechnologies Inc Compositions comprising a variant cas12i4 polypeptide and uses thereof
CN112997966A (en) * 2021-03-08 2021-06-22 国家卫生健康委科学技术研究所 Mouse model knocking-in miRNA-125a based on CRISPR/Cas9 technology and construction method
WO2022188797A1 (en) 2021-03-09 2022-09-15 Huigene Therapeutics Co., Ltd. Engineered crispr/cas13 system and uses thereof
WO2022204543A1 (en) * 2021-03-25 2022-09-29 The Regents Of The University Of California Methods and materials for treating huntington's disease
WO2022240846A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
CN113180015A (en) * 2021-05-25 2021-07-30 四川省农业机械研究设计院 Silkworm rearing room and three-section type assembly line silkworm rearing method
EP4349979A1 (en) 2021-05-27 2024-04-10 Institute Of Zoology, Chinese Academy Of Sciences Engineered cas12i nuclease, effector protein and use thereof
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
CA3225121A1 (en) 2021-07-20 2023-01-26 Benjamin DEVERMAN Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
US20230048564A1 (en) 2021-07-22 2023-02-16 Arbor Biotechnologies, Inc. Crispr-associated transposon systems and methods of using same
CA3221517A1 (en) 2021-07-30 2023-02-02 Monika KLOIBER-MAITZ Plants with improved digestibility and marker haplotypes
CN113607963B (en) * 2021-08-11 2024-05-24 河南省人民医院 Protein marker for predicting retinal aging process, screening method and application thereof
EP4384608A1 (en) 2021-08-12 2024-06-19 Arbor Biotechnologies, Inc. Compositions comprising a variant cas12i3 polypeptide and uses thereof
CN113373178A (en) * 2021-08-13 2021-09-10 上海南方模式生物科技股份有限公司 Construction method and application of TLR8 gene humanized animal model
JP7125727B1 (en) 2021-09-07 2022-08-25 国立大学法人千葉大学 Compositions for modifying nucleic acid sequences and methods for modifying target sites in nucleic acid sequences
CA3230927A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
WO2023059846A1 (en) 2021-10-08 2023-04-13 President And Fellows Of Harvard College Ionic liquids for drug delivery
IL311962A (en) 2021-10-14 2024-06-01 Lonza Sales Ag Modified producer cells for extracellular vesicle production
CN113913428A (en) * 2021-10-14 2022-01-11 南通大学 Small interfering RNA of targeting Tap 2a gene and application thereof in preparation of medicine for treating chronic pain
CN118251491A (en) 2021-10-28 2024-06-25 瑞泽恩制药公司 CRISPR/Cas related methods and compositions for knockout of C5
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
CN114015674A (en) 2021-11-02 2022-02-08 辉二(上海)生物科技有限公司 Novel CRISPR-Cas12i system
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023093862A1 (en) 2021-11-26 2023-06-01 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
CA3238939A1 (en) 2021-12-08 2023-06-15 Gaurang Patel Mutant myocilin disease model and uses thereof
CN114107231B (en) * 2021-12-13 2023-08-18 重庆大学 Recombinant adeno-associated virus for realizing whole brain postsynaptic neuron cell body marking and application thereof
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
WO2023115039A2 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
TW202342757A (en) 2021-12-17 2023-11-01 美商薩那生物科技公司 Modified paramyxoviridae attachment glycoproteins
WO2023122764A1 (en) 2021-12-22 2023-06-29 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
CN114280300B (en) * 2021-12-28 2023-04-25 四川大学华西医院 Application of urine protein in diagnosis of metabolic liver disease
WO2023129974A1 (en) 2021-12-29 2023-07-06 Bristol-Myers Squibb Company Generation of landing pad cell lines
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
WO2023150518A1 (en) 2022-02-01 2023-08-10 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
TW202332767A (en) 2022-02-02 2023-08-16 美商雷傑納榮製藥公司 Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023150637A1 (en) 2022-02-02 2023-08-10 Inscripta, Inc. Nucleic acid-guided nickase fusion proteins
WO2023148478A1 (en) 2022-02-03 2023-08-10 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
WO2023148476A1 (en) 2022-02-03 2023-08-10 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
WO2023148475A1 (en) 2022-02-04 2023-08-10 Nicoventures Trading Limited Method of modulating alkaloid content in tobacco plants
CN114480497B (en) * 2022-02-28 2023-09-15 湖南师范大学 Construction and application method of ep400 gene knockout zebra fish heart failure model
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
GB202205148D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
GB202205149D0 (en) 2022-04-07 2022-05-25 Nicoventures Trading Ltd Method
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
GB202206109D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
GB202206107D0 (en) 2022-04-27 2022-06-08 Nicoventures Trading Ltd Method
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023225564A1 (en) 2022-05-18 2023-11-23 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
WO2023225665A1 (en) 2022-05-19 2023-11-23 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
CN115053861A (en) * 2022-06-30 2022-09-16 南方医科大学南方医院 Construction method and application of animal model of schizophrenia based on immune activation
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
WO2024016003A2 (en) 2022-07-14 2024-01-18 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
WO2024020346A2 (en) 2022-07-18 2024-01-25 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
WO2024020567A1 (en) 2022-07-22 2024-01-25 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 (en) * 2022-07-26 2022-09-27 华中科技大学同济医学院附属协和医院 Fusion plasmid of small molecular peptide and application of fusion plasmid in antitumor drugs
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
WO2024031053A1 (en) 2022-08-05 2024-02-08 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43
CN115247149B (en) * 2022-08-22 2023-06-16 华域生物科技(天津)有限公司 Culture medium composition suitable for NK cells and culture method
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
WO2024042199A1 (en) 2022-08-26 2024-02-29 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
WO2024064838A1 (en) 2022-09-21 2024-03-28 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024124044A1 (en) 2022-12-07 2024-06-13 The Brigham And Women’S Hospital, Inc. Compositions and methods targeting sat1 for enhancing anti¬ tumor immunity during tumor progression

Family Cites Families (239)

* 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
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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
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
EP0264166B1 (en) 1986-04-09 1996-08-21 Genzyme Corporation Transgenic animals secreting desired proteins into milk
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4783989A (en) 1987-03-05 1988-11-15 Atlantic Richfield Company Vapor pressure measurement apparatus and method
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
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
WO1996039154A1 (en) 1995-06-06 1996-12-12 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
WO1997000321A1 (en) 1995-06-14 1997-01-03 Commonwealth Scientific And Industrial Research Organisation Immune response modulators and uses therefor
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
JP4303418B2 (en) 1997-10-24 2009-07-29 ライフ テクノロジーズ コーポレーション Recombination cloning using nucleic acids with 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 (en) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7033744B2 (en) 2001-03-16 2006-04-25 Naoya Kobayashi Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof
US8318173B2 (en) 2001-04-05 2012-11-27 The John Hopkins University Chimeric vaccines
ES2566561T3 (en) 2001-07-12 2016-04-13 University Of Massachusetts In vivo production of small interfering RNAs that mediate gene silencing
JP2005509409A (en) 2001-08-08 2005-04-14 ジェンザイム・コーポレーション Methods for treating diabetes and other glycemic 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 (en) 2001-12-21 2005-05-25 牛津生物医学（英国）有限公司 Transgenic organism
US7206639B2 (en) 2002-03-15 2007-04-17 Sarcos Investments Lc Cochlear drug delivery system and method
US7539579B2 (en) 2002-04-09 2009-05-26 Beattie Kenneth L Oligonucleotide probes for genosensor chips
WO2003104414A2 (en) 2002-06-11 2003-12-18 The Scripps Research Institute Artificial transcription factors
WO2004002453A1 (en) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab 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
EP1648519B1 (en) 2003-07-16 2014-10-08 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
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 (en) 2003-11-21 2006-02-10 Pasteur Institut GENOME OF LEGIONELLA PNEUMOPHILA SOUCHE PARIS- DIAGNOSTIC AND EPIDEMIOLOGICAL APPLICATIONS
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
WO2005072793A1 (en) 2004-01-29 2005-08-11 The Charles Stark Draper Laboratory, Inc. Implantable drug delivery apparatus
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
CA2569664C (en) 2004-06-07 2013-07-16 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 (en) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse NON-INTERACTIVE AND NON-REPLICATIVE LENTIVIRUS, PREPARATION AND USES
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
CA2606002A1 (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
WO2007014275A2 (en) * 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US10066233B2 (en) * 2005-08-26 2018-09-04 Dupont Nutrition Biosciences Aps Method of modulating cell resistance
US9265933B2 (en) 2005-09-08 2016-02-23 Massachusetts Eye And Ear Infirmary Cochlear implants containing biological cells and uses thereof
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
WO2007091269A2 (en) 2006-02-08 2007-08-16 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
US9677123B2 (en) 2006-03-15 2017-06-13 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
JP5570806B2 (en) 2006-05-11 2014-08-13 アルナイラム ファーマシューティカルズ， インコーポレイテッド Compositions and methods for inhibiting the expression of the PCSK9 gene
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
US20100146651A1 (en) 2006-11-14 2010-06-10 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
RU2531343C2 (en) 2007-03-02 2014-10-20 ДюПон Ньютришн Байосайенсиз АпС, Method of generating starter culture, starter culture and fermentation method with its application
PE20090064A1 (en) 2007-03-26 2009-03-02 Novartis Ag DOUBLE-CHAIN RIBONUCLEIC ACID TO INHIBIT THE EXPRESSION OF THE HUMAN E6AP GENE AND THE PHARMACEUTICAL COMPOSITION THAT INCLUDES IT
CN101815446A (en) 2007-05-29 2010-08-25 施莲·特萨特斯-斯特劳斯 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
CA2941187C (en) 2007-08-14 2021-03-02 Fred Hutchinson Cancer Research Center Needle array assembly and method for delivering therapeutic agents
WO2009088786A1 (en) 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. Rna interference for the treatment of heart failure
JP2011511806A (en) 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 Compounds that enhance Atoh1 expression
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
CN102119217B (en) 2008-04-15 2015-06-03 普洛体维生物治疗公司 Novel lipid formulations for nucleic acid delivery
US20090280262A1 (en) 2008-05-08 2009-11-12 Chung Yuan Christian University Method for forming composite membrane with porous coating layer and apparatus thereof
WO2009147684A2 (en) 2008-06-06 2009-12-10 Quark Pharmaceuticals, Inc. Compositions and methods for treatment of ear disorders
JP2011526619A (en) 2008-06-30 2011-10-13 サイレンシード リミテッド Topical drug delivery system, method and composition thereof
US20110117189A1 (en) 2008-07-08 2011-05-19 S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. Ophthalmic compositions for treating pathologies of the posterior segment of the eye
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
US9255130B2 (en) 2008-07-29 2016-02-09 Academia Sinica Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
EP2352821B1 (en) 2008-09-08 2016-11-23 Cellectis Meganuclease variants cleaving a dna target sequence from a glutamine synthetase gene and uses thereof
PT2334794T (en) 2008-09-15 2017-02-15 Harvard College Modulation of bcl11a for treatment of hemoglobinopathies
US20100076057A1 (en) * 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
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
CN104910025B (en) 2008-11-07 2019-07-16 麻省理工学院 Alkamine lipid and its purposes
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
KR101803737B1 (en) * 2008-12-04 2017-12-01 상가모 테라퓨틱스, 인코포레이티드 Genome editing in rats using zinc-finger nucleases
US20110023146A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110023153A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20120159653A1 (en) 2008-12-04 2012-06-21 Sigma-Aldrich Co. Genomic editing of genes involved in macular degeneration
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
AU2009324534B2 (en) 2008-12-10 2015-07-30 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
JP5932632B2 (en) 2009-03-20 2016-06-15 サンガモ バイオサイエンシーズ， インコーポレイテッド Modification of CXCR4 using modified zinc finger protein
WO2010129602A2 (en) 2009-05-04 2010-11-11 Fred Hutchinson Cancer Research Center Cocal vesiculovirus envelope pseudotyped retroviral vectors
WO2011036510A1 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
WO2010143917A2 (en) 2009-06-11 2010-12-16 Toolgen Incorporation 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
IL292615B2 (en) 2009-07-01 2023-11-01 Protiva Biotherapeutics Inc Nucleic acid-lipid particles, compositions comprising the same and uses thereof
AU2010275432A1 (en) * 2009-07-24 2012-02-02 Sigma-Aldrich Co. Llc. Method for genome editing
US9234016B2 (en) 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US8927807B2 (en) 2009-09-03 2015-01-06 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011064736A1 (en) * 2009-11-27 2011-06-03 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
AU2010334799A1 (en) 2009-12-23 2012-07-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Influenza targets
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
CN102939377B (en) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 Use Zinc finger nuclease to carry out genome editor to Rosa site
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
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2569435A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
AU2011256838B2 (en) * 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
DK2575894T3 (en) 2010-05-28 2015-05-26 Oxford Biomedica Ltd FEED lentiviral vectors FOR BRAIN
BR112013001685B1 (en) 2010-07-23 2021-10-13 Sigma-Aldrich Co. Llc METHOD FOR EDITING AT LEAST ONE ENDOGENOUS CHROMOSOMIC SEQUENCE IN A CELL BY INSERTING A SEQUENCE INTO THE CHROMOSOMIC SEQUENCE
US9081737B2 (en) 2010-08-02 2015-07-14 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US9549901B2 (en) 2010-09-03 2017-01-24 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
AU2011316575B2 (en) 2010-10-12 2015-10-29 Sangamo Therapeutics, 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
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
KR20220056884A (en) 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Adeno-associated virus virions with variant capsid and methods of use thereof
EP2702160B1 (en) 2011-04-27 2020-05-27 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
EP3498833B1 (en) 2011-09-21 2023-08-16 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
JP2014530603A (en) 2011-10-06 2014-11-20 サンガモ バイオサイエンシーズ， インコーポレイテッド Methods and compositions for controlling HIV infection
EP2771457B1 (en) 2011-10-27 2017-11-22 Sangamo Therapeutics, 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
WO2013071440A1 (en) 2011-11-18 2013-05-23 UNIVERSITé LAVAL Methods and products for increasing frataxin levels and uses thereof
CA2856137A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US20130137173A1 (en) 2011-11-30 2013-05-30 Feng Zhang Nucleotide-specific recognition sequences for designer tal effectors
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
KR20140102759A (en) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 Modified nucleoside, nucleotide, and nucleic acid compositions
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CN108285491B (en) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 Methods and compositions for treating huntington's disease
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
PT2800811T (en) * 2012-05-25 2017-08-17 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN104507488A (en) 2012-06-01 2015-04-08 德雷塞尔大学 Modulation of hepatitis b virus cccdna transcription
FR2993379B1 (en) 2012-07-10 2015-09-04 Morpho SECURE COMPARATIVE PROCESSING METHOD
FR2995325B1 (en) 2012-09-11 2015-03-13 Ct Tech De L Ind Des Papiers Cartons Et Celluloses PROCESS FOR PRODUCING A MULTILAYER FIBROUS MEDIUM BY FRACTIONATION AND STRATIFICATION
EP2906684B8 (en) 2012-10-10 2020-09-02 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
CN110643600A (en) 2012-10-23 2020-01-03 基因工具股份有限公司 System for cutting target DNA and use thereof
ES2769310T3 (en) 2012-12-06 2020-06-25 Sigma Aldrich Co Llc Modification and regulation of the genome based on CRISPR
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP3825401A1 (en) * 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
SG10201912327SA (en) * 2012-12-12 2020-02-27 Broad Inst Inc Engineering and Optimization of Improved Systems, Methods and Enzyme 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
EP2931892B1 (en) 2012-12-12 2018-09-12 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US20140310830A1 (en) * 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP4279588A3 (en) 2012-12-12 2024-01-17 The Broad Institute, Inc. Engineering of systems, methods and optimized guide 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
MX2015007550A (en) 2012-12-12 2017-02-02 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications.
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
WO2014113493A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
WO2014124226A1 (en) 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
US11135273B2 (en) 2013-02-07 2021-10-05 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
ES2901396T3 (en) 2013-03-14 2022-03-22 Caribou Biosciences Inc Nucleic Acid Targeting Nucleic Acid Compositions and Methods
CN110540991B (en) 2013-03-15 2023-10-24 通用医疗公司 Enhancement of specificity of RNA-guided genome editing using truncated guide RNA (tru-gRNA)
AU2013384372B2 (en) 2013-03-27 2018-03-22 Wilco Ag Method of inline inspecting and/or testing devices and apparatus to perform such method
WO2014165349A1 (en) 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
CN116083487A (en) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 Methods and compositions for treating genetic conditions
JP7065564B2 (en) 2013-05-29 2022-05-12 セレクティス Methods for Providing Accurate DNA Cleavage Using CAS9 Nickase Activity
MY177814A (en) 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
KR20230136697A (en) 2013-06-05 2023-09-26 듀크 유니버시티 Rna-guided gene editing and gene regulation
RU2716420C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery and use of systems of crispr-cas, vectors and compositions for targeted action and therapy in liver
WO2014204728A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011034B1 (en) 2013-06-17 2019-08-07 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CN105492611A (en) 2013-06-17 2016-04-13 布罗德研究所有限公司 Optimized CRISPR-CAS double nickase systems, methods and compositions for sequence manipulation
EP3971287A1 (en) 2013-07-11 2022-03-23 ModernaTX, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
CN103388006B (en) 2013-07-26 2015-10-28 华东师范大学 A kind of construction process of site-directed point mutation
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US20160237455A1 (en) 2013-09-27 2016-08-18 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
AU2014346559B2 (en) 2013-11-07 2020-07-09 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
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology 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
CN105899657A (en) 2013-12-12 2016-08-24 布罗德研究所有限公司 Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
CN106536729A (en) 2013-12-12 2017-03-22 布罗德研究所有限公司 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
US10354746B2 (en) 2014-01-27 2019-07-16 Georgia Tech Research Corporation Methods and systems for identifying CRISPR/Cas off-target sites
CN103779358A (en) * 2014-01-27 2014-05-07 京东方科技集团股份有限公司 Array substrate, manufacturing method of array substrate and display device
JP6323228B2 (en) 2014-07-18 2018-05-16 富士電機株式会社 Power converter
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
ES2905525T3 (en) 2015-05-06 2022-04-11 Snipr Tech Ltd Alteration of microbial populations and modification of the microbiota
RU2752834C2 (en) 2015-06-18 2021-08-09 Те Брод Инститьют, Инк. Crispr enzyme mutations reducing non-targeted effects
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
JP6186470B2 (en) 2016-04-20 2017-08-23 パイオニア株式会社 Acoustic device, volume control method, volume control program, and recording medium
KR102085165B1 (en) 2017-11-10 2020-03-05 주식회사 엘지화학 Organic metal compound and organic light emitting device comprising the same

Also Published As

Publication number Publication date
AU2019283878B2 (en) 2022-06-09
US20170107536A1 (en) 2017-04-20
EP3327127B1 (en) 2021-03-24
BR112015013784A2 (en) 2017-07-11
JP7228059B2 (en) 2023-02-22
EP3327127A1 (en) 2018-05-30
HK1215048A1 (en) 2016-08-12
ES2658401T3 (en) 2018-03-09
US11041173B2 (en) 2021-06-22
AU2019283878A1 (en) 2020-01-23
ZA201504132B (en) 2022-03-30
EP2931897A2 (en) 2015-10-21
JP2022040308A (en) 2022-03-10
HK1252619A1 (en) 2019-05-31
CN105164264A (en) 2015-12-16
CN105164264B (en) 2021-05-07
WO2014093622A9 (en) 2014-11-06
HK1220725A1 (en) 2017-05-12
JP2016501531A (en) 2016-01-21
WO2014093622A8 (en) 2014-12-04
SG10201912328UA (en) 2020-02-27
WO2014093622A2 (en) 2014-06-19
JP2023073245A (en) 2023-05-25
IL239317B (en) 2022-07-01
IL293526A (en) 2022-08-01
AU2013359199B2 (en) 2019-10-31
US20140242699A1 (en) 2014-08-28
JP2019103513A (en) 2019-06-27
MX2015007550A (en) 2017-02-02
KR20150105956A (en) 2015-09-18
US20210269831A1 (en) 2021-09-02
EP4299741A3 (en) 2024-02-28
JP7013406B2 (en) 2022-02-15
MX2022001378A (en) 2022-06-23
AU2013359199C1 (en) 2021-06-17
US20170152528A1 (en) 2017-06-01
ES2883590T3 (en) 2021-12-09
AU2022203759A1 (en) 2022-06-23
US20140179770A1 (en) 2014-06-26
AU2013359199A1 (en) 2015-07-30
CA2894681A1 (en) 2014-06-19
WO2014093622A3 (en) 2014-08-07
CN110872583A (en) 2020-03-10
DK2931897T3 (en) 2018-02-05
RU2721275C2 (en) 2020-05-18
RU2015128057A (en) 2019-03-27
DK3327127T3 (en) 2021-06-28
US20150020223A1 (en) 2015-01-15
EP2931897B1 (en) 2017-11-01
EP4299741A2 (en) 2024-01-03
US20230399662A1 (en) 2023-12-14
US20240035048A1 (en) 2024-02-01
IL239317A0 (en) 2015-07-30
SG10201707569YA (en) 2017-10-30

Similar Documents

Publication Publication Date Title
HK1252619A1 (en) Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
IL289736A (en) Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
HK1223646A1 (en) Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
IL274182B (en) Delivery methods and compositions for nuclease-mediated genome engineering
HK1216759A1 (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
HK1247961A1 (en) Crispr-cas component systems, methods and compositions for sequence manipulation
HK1207119A1 (en) Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
PL2784162T3 (en) Engineering of systems, methods and optimized guide compositions for sequence manipulation
HK1208056A1 (en) Crispr-cas component systems, methods and compositions for sequence manipulation crispr-cas
PL2931898T3 (en) Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2994531A4 (en) Delivery methods and compositions for nuclease-mediated genome engineering
EP3858420C0 (en) Tailless needleless valve system
IL243995B (en) Continuous release compositions made from hyaluronic acid, and therapeutic applications of same
EP2827882A4 (en) Systems, compositions, and methods for transplantation