CN107787367B - 用于crispr/cas介导的基因调控的化学修饰的引导rna - Google Patents

用于crispr/cas介导的基因调控的化学修饰的引导rna Download PDF

Info

Publication number
CN107787367B
CN107787367B CN201680032647.XA CN201680032647A CN107787367B CN 107787367 B CN107787367 B CN 107787367B CN 201680032647 A CN201680032647 A CN 201680032647A CN 107787367 B CN107787367 B CN 107787367B
Authority
CN
China
Prior art keywords
modified
sgrna
cells
target
nucleotides
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CN201680032647.XA
Other languages
English (en)
Chinese (zh)
Other versions
CN107787367A (zh
Inventor
M·H·波蒂厄斯
A·亨德尔
J·克拉克
R·O·巴克
D·E·赖安
D·J·德林杰
R·凯撒
J·迈尔森
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Agilent Technologies Inc
Leland Stanford Junior University
Original Assignee
Agilent Technologies Inc
Leland Stanford Junior University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=55910340&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN107787367(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Agilent Technologies Inc, Leland Stanford Junior University filed Critical Agilent Technologies Inc
Priority to CN202111196588.9A priority Critical patent/CN114231527A/zh
Publication of CN107787367A publication Critical patent/CN107787367A/zh
Application granted granted Critical
Publication of CN107787367B publication Critical patent/CN107787367B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
    • A61P25/34—Tobacco-abuse
    • 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
    • A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
    • A61P25/36—Opioid-abuse
    • 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
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • A61P7/06—Antianaemics
    • 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
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/63—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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • 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
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Addiction (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
CN201680032647.XA 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna Active CN107787367B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202111196588.9A CN114231527A (zh) 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562143729P 2015-04-06 2015-04-06
US62/143,729 2015-04-06
US201562160545P 2015-05-12 2015-05-12
US62/160,545 2015-05-12
PCT/US2016/026028 WO2016164356A1 (en) 2015-04-06 2016-04-05 Chemically modified guide rnas for crispr/cas-mediated gene regulation

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202111196588.9A Division CN114231527A (zh) 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna

Publications (2)

Publication Number Publication Date
CN107787367A CN107787367A (zh) 2018-03-09
CN107787367B true CN107787367B (zh) 2021-10-26

Family

ID=55910340

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201680032647.XA Active CN107787367B (zh) 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna
CN202111196588.9A Pending CN114231527A (zh) 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202111196588.9A Pending CN114231527A (zh) 2015-04-06 2016-04-05 用于crispr/cas介导的基因调控的化学修饰的引导rna

Country Status (9)

Country Link
US (5) US11306309B2 (enExample)
EP (1) EP3280803B1 (enExample)
JP (1) JP6873911B2 (enExample)
KR (2) KR102888521B1 (enExample)
CN (2) CN107787367B (enExample)
AU (2) AU2016246450B2 (enExample)
CA (1) CA2981715A1 (enExample)
ES (1) ES2884838T3 (enExample)
WO (1) WO2016164356A1 (enExample)

Families Citing this family (155)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2015153789A1 (en) * 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10040048B1 (en) 2014-09-25 2018-08-07 Synthego Corporation Automated modular system and method for production of biopolymers
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
EP3234133B1 (en) 2014-12-18 2020-11-11 Integrated DNA Technologies, Inc. Crispr-based compositions and methods of use
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
AU2016225178B2 (en) 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
ES2884838T3 (es) 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
US11279928B2 (en) 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
WO2017077394A2 (en) 2015-11-04 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CA3013179A1 (en) * 2016-01-30 2017-08-03 Bonac Corporation Artificial single guide rna and use thereof
US11845933B2 (en) 2016-02-03 2023-12-19 Massachusetts Institute Of Technology Structure-guided chemical modification of guide RNA and its applications
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
KR101710026B1 (ko) 2016-08-10 2017-02-27 주식회사 무진메디 Cas9 단백질 및 가이드 RNA의 혼성체를 함유하는 나노 리포좀 전달체 조성물
CA3033736C (en) * 2016-08-12 2023-10-24 Toolgen Incorporated Manipulated immunoregulatory element and immunity altered thereby
WO2018035495A1 (en) 2016-08-19 2018-02-22 Whitehead Institute For Biomedical Research Methods of editing dna methylation
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018049075A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
CA3039409A1 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
JP2019535287A (ja) 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Ｃｒｉｓｐｒ／ｃｐｆ１システム及び方法
CN110291198B (zh) 2016-12-08 2024-11-26 因特利亚治疗公司 经修饰的指导rna
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
US20200149039A1 (en) 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
WO2018119060A1 (en) 2016-12-20 2018-06-28 Bristol-Myers Squibb Company Methods for increasing the efficiency of homology directed repair (hdr) in the cellular genome
KR102551664B1 (ko) 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR102758180B1 (ko) * 2016-12-30 2025-01-23 에디타스 메디신, 인코포레이티드 합성 가이드 분자, 그와 관련된 조성물 및 방법
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
FI3565891T3 (fi) 2017-01-09 2023-07-04 Whitehead Inst Biomedical Res Menetelmiä geeniekspression muuttamiseksi häiritsemällä säätelysilmukoita muodostavia transkriptiotekijämultimeerejä
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
AU2018221730B2 (en) * 2017-02-15 2024-06-20 Novo Nordisk A/S Donor repair templates multiplex genome editing
EP3585896A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of merosin-deficient cogenital muscular dystrophy (mdcmd) and other laminin, alpha 2 (lama2) gene related conditions or disorders
EP3585897A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders
BR112019017751A2 (pt) 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
US20200171132A1 (en) * 2017-03-20 2020-06-04 The Research Foundation For The State University Of New York Methods of inhibiting fungal ceramide synthase for treatment of cryptococcus neoformans infection
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018183808A1 (en) * 2017-03-31 2018-10-04 Agenovir Corporation Antiviral therapeutic
WO2018208067A1 (ko) 2017-05-08 2018-11-15 주식회사 툴젠 인위적으로 조작된 조작면역세포
MY201573A (en) 2017-05-12 2024-03-02 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
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
US11166985B2 (en) 2017-05-12 2021-11-09 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
US20190076814A1 (en) 2017-09-11 2019-03-14 Synthego Corporation Biopolymer synthesis system and method
US20200216805A1 (en) * 2017-09-18 2020-07-09 Edigene Inc. Gene editing t cell and use thereof
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
BR112020011255A2 (pt) 2017-12-05 2020-11-24 Vertex Pharmaceuticals Incorporated células-tronco e progenitoras hematopoiéticas cd34+ humanas modificadas por crispr-cas9 e usos das mesmas
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US11542530B2 (en) 2018-01-22 2023-01-03 Industry-Academic Cooperation Foundation Gyeongsang National University Method for increasing efficiency of homologous recombination-based gene editing in plant
US20190233816A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN110229814B (zh) * 2018-03-06 2025-11-14 中国科学院动物研究所 改进的向导rna
US20210071202A1 (en) * 2018-03-29 2021-03-11 Jichi Medical University Genome editing method, composition, cell, cell preparation, and method for producing cell preparation
EP3781678A4 (en) * 2018-04-18 2022-06-01 Yale University Compositions and methods for multiplexed tumor vaccination with endogenous gene activation
JP7542441B2 (ja) 2018-05-11 2024-08-30 クリスパー セラピューティクス アクチェンゲゼルシャフト 癌を治療するための方法及び組成物
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
CN108624592B (zh) * 2018-05-24 2021-08-17 苏州大学张家港工业技术研究院 针对DJ-1基因编辑的sgRNA筛选及其载体与应用
EP3802818A4 (en) * 2018-05-25 2022-03-02 New York Institute of Technology Method and system for use in direct sequencing of rna
EP3802779A1 (en) * 2018-06-01 2021-04-14 Avectas Limited Cell engineering platform
EP3802828A4 (en) * 2018-06-08 2022-10-26 Intellia Therapeutics, Inc. RNA MODIFIED GUIDES FOR GENE EDITING
CN110616233B (zh) * 2018-06-20 2022-02-22 西安桑尼赛尔生物医药有限公司 CRISPR-Cas9高效敲除原代T细胞基因的方法及其应用
CN110616187B (zh) * 2018-06-20 2021-12-03 西安桑尼赛尔生物医药有限公司 CRISPR-Cas9高效敲入嵌合抗原受体基因到T细胞特定基因组位点的方法及应用
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
SG11202101994XA (en) 2018-08-28 2021-03-30 Vor Biopharma Inc Genetically engineered hematopoietic stem cells and uses thereof
AU2019342197B2 (en) 2018-09-21 2025-10-16 President And Fellows Of Harvard College Methods and compositions for treating diabetes, and methods for enriching mRNA coding for secreted proteins
WO2020068643A1 (en) * 2018-09-25 2020-04-02 Excision Biotherapeutics, Inc. CRISPRs WITH IMPROVED SPECIFICITY
KR102126573B1 (ko) 2018-10-18 2020-06-26 대한민국 CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도
CN111088253A (zh) * 2018-10-24 2020-05-01 广州鼓润医疗科技有限公司 针对hbb-28地中海贫血基因的crispr单碱基供体修复体系
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN111254164A (zh) * 2018-11-30 2020-06-09 中国科学院大连化学物理研究所 一种快速建立crispr基因编辑肝癌细胞株的方法及细胞株
CN111321171A (zh) * 2018-12-14 2020-06-23 江苏集萃药康生物科技有限公司 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法
CN109652440A (zh) * 2018-12-28 2019-04-19 北京市农林科学院 VQRn-Cas9&PmCDA1&UGI碱基编辑系统在植物基因编辑中的应用
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN110157695A (zh) * 2019-01-25 2019-08-23 南昌大学 一种植物RNA m5C甲基化修饰的系统及方法
DE112020001306T5 (de) 2019-03-19 2022-01-27 Massachusetts Institute Of Technology Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220249558A1 (en) 2019-04-30 2022-08-11 Crispr Therapeutics Ag Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
AU2020280103A1 (en) 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
CN110283838B (zh) * 2019-06-27 2022-03-25 安徽省农业科学院水稻研究所 一种高剪切效率ScCas9基因及其应用
WO2021041001A2 (en) * 2019-08-09 2021-03-04 Regents Of The University Of Minnesota AUGMENTED sgRNAS AND METHODS FOR THEIR USE TO ENHANCE SOMATIC AND GERMLINE PLANT GENOME ENGINEERING
CA3151669A1 (en) 2019-08-28 2021-03-04 Vor Biopharma Inc. Compositions and methods for cd123 modification
US20220290160A1 (en) 2019-08-28 2022-09-15 Vor Biopharma Inc. Compositions and methods for cll1 modification
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
EP4038190A1 (en) 2019-10-03 2022-08-10 Artisan Development Labs, Inc. Crispr systems with engineered dual guide nucleic acids
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20230028445A1 (en) * 2019-10-11 2023-01-26 Black Hawk Genomics, Llc Identification of genomic structural variants using long-read sequencing
AU2020369599A1 (en) 2019-10-23 2022-05-12 Monsanto Technology Llc Compositions and methods for RNA-templated editing in plants
IL292434A (en) * 2019-11-05 2022-06-01 Pairwise Plants Services Inc Compositions and methods for rna-encoded dna-replacement of alleles
CN115176001A (zh) * 2019-12-11 2022-10-11 因特利亚治疗公司 用于基因编辑的修饰的引导rna
IL271656A (en) 2019-12-22 2021-06-30 Yeda Res & Dev System and methods for identifying cells that have undergone genome editing
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
CA3190447A1 (en) * 2020-08-03 2022-02-10 The Board Of Trustees Of The Leland Stanford Junior University Gene correction for scid-x1 in long-term hematopoietic stem cells
CN112111507B (zh) * 2020-08-10 2022-08-23 江苏大学 一种灰树花CRISPR-Cas9基因编辑系统、方法及应用
CN112266911B (zh) * 2020-08-27 2022-07-22 清华大学 核酸分子
US20240110189A1 (en) 2020-08-28 2024-04-04 Vor Biopharma Inc. Compositions and methods for cll1 modification
EP4204564A1 (en) 2020-08-28 2023-07-05 Vor Biopharma Inc. Compositions and methods for cd123 modification
JP2023541457A (ja) 2020-09-14 2023-10-02 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ３８修飾のための化合物および方法
WO2022056459A1 (en) 2020-09-14 2022-03-17 Vor Biopharma, Inc. Compositions and methods for cd5 modification
EP4214318A1 (en) 2020-09-18 2023-07-26 Vor Biopharma Inc. Compositions and methods for cd7 modification
US20230364233A1 (en) 2020-09-28 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd6 modification
US20230364146A1 (en) 2020-09-30 2023-11-16 Vor Biopharma Inc. Compositions and methods for cd30 gene modification
CN112126661B (zh) * 2020-09-30 2022-07-22 上海中医药大学 一种高效敲除nk细胞中tigit基因的方法
US20230357798A1 (en) * 2020-10-12 2023-11-09 The Board Of Trustees Of The Leland Stanford Junior University Gene correction for x-cgd in hematopoietic stem and progenitor cells
KR20230097089A (ko) 2020-10-27 2023-06-30 보르 바이오파마 인크. 조혈 악성종양을 치료하기 위한 조성물 및 방법
WO2022094245A1 (en) 2020-10-30 2022-05-05 Vor Biopharma, Inc. Compositions and methods for bcma modification
CN112553195B (zh) * 2020-11-05 2022-04-05 南方医科大学 一种用于CRISPR-Cas9定点突变编辑DNMT1基因的试剂及其应用
US20230414755A1 (en) 2020-11-13 2023-12-28 Vor Biopharma Inc. Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors
CA3202219A1 (en) 2020-12-31 2022-07-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
AU2022227650A1 (en) 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
US20240200059A1 (en) 2021-04-09 2024-06-20 Vor Biopharma Inc. Photocleavable guide rnas and methods of use thereof
WO2022256448A2 (en) 2021-06-01 2022-12-08 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
EP4355879A4 (en) 2021-06-14 2025-07-23 Kamau Therapeutics Inc METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS
WO2022266538A2 (en) 2021-06-18 2022-12-22 Artisan Development Labs, Inc. Compositions and methods for targeting, editing or modifying human genes
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
US20250122534A1 (en) 2021-08-02 2025-04-17 Vor Biopharma Inc. Compositions and methods for gene modification
US11884915B2 (en) * 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
AU2022346785A1 (en) * 2021-09-14 2024-04-18 Agilent Technologies, Inc. Methods for using guide rnas with chemical modifications
US20240417755A1 (en) 2021-09-27 2024-12-19 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
EP4413146A2 (en) * 2021-10-05 2024-08-14 The Board of Trustees of the Leland Stanford Junior University Treatment of polycythemia vera via crispr/aav6 genome editing
AU2022387087A1 (en) 2021-11-09 2024-05-02 Vor Biopharma Inc. Compositions and methods for erm2 modification
WO2023155924A1 (en) * 2022-02-21 2023-08-24 Huidagene Therapeutics Co., Ltd. Guide rna and uses thereof
US20250179531A1 (en) 2022-02-25 2025-06-05 Vor Biopharma Inc. Compositions and methods for homology-directed repair gene modification
WO2023167882A1 (en) 2022-03-01 2023-09-07 Artisan Development Labs, Inc. Composition and methods for transgene insertion
EP4499829A2 (en) 2022-03-29 2025-02-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
US20250295695A1 (en) 2022-04-04 2025-09-25 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
WO2023225410A2 (en) 2022-05-20 2023-11-23 Artisan Development Labs, Inc. Systems and methods for assessing risk of genome editing events
EP4555091A2 (en) 2022-07-13 2025-05-21 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
EP4590820A2 (en) 2022-09-22 2025-07-30 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
WO2024062138A1 (en) 2022-09-23 2024-03-28 Mnemo Therapeutics Immune cells comprising a modified suv39h1 gene
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2024137533A1 (en) 2022-12-19 2024-06-27 Graphite Bio, Inc. Improved peptide inhibitors of p53 binding protein 53bp1
WO2024168312A1 (en) 2023-02-09 2024-08-15 Vor Biopharma Inc. Methods for treating hematopoietic malignancy
WO2024193704A1 (en) * 2023-03-22 2024-09-26 Huidagene Therapeutics Co., Ltd. Guide nucleic acids targeting dmd and uses thereof
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025177303A1 (en) * 2024-02-19 2025-08-28 Micro Crispr Pvt Ltd. Crispr-mediated gene editing to induce fetal hemoglobin

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103614415A (zh) * 2013-11-27 2014-03-05 苏州同善生物科技有限公司 一种基于crispr基因敲除技术建立肥胖症大鼠动物模型的方法
WO2014093712A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014093635A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
WO2015006747A2 (en) * 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use.
WO2015026885A1 (en) * 2013-08-22 2015-02-26 Pioneer Hi-Bred International, Inc. Genome modification using guide polynucleotide/cas endonuclease systems and methods of use

Family Cites Families (157)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5032401A (en) 1989-06-15 1991-07-16 Alpha Beta Technology Glucan drug delivery system and adjuvant
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
US7371580B2 (en) 2001-08-24 2008-05-13 Agilent Technologies, Inc. Use of unstructured nucleic acids in assaying nucleic acid molecules
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
WO2005065419A2 (en) 2003-12-29 2005-07-21 Am Biosolutions Method of culturing cells
US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
WO2008141248A2 (en) 2007-05-10 2008-11-20 Agilent Technologies, Inc. Thiocarbon-protecting groups for rna synthesis
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
KR101606943B1 (ko) 2008-06-27 2016-03-28 고쿠리츠 다이가쿠 호진 교토 다이가쿠 유도된 다능성 줄기 세포의 효율적인 확립 방법
US20100076183A1 (en) 2008-09-22 2010-03-25 Dellinger Douglas J Protected monomer and method of final deprotection for rna synthesis
WO2011016588A1 (en) 2009-08-07 2011-02-10 Kyoto University Method of efficiently establishing induced pluripotent stem cells
WO2011027908A1 (en) 2009-09-02 2011-03-10 Kyoto University Method of selecting safe pluripotent stem cells
US9585920B2 (en) 2011-02-04 2017-03-07 Katherine Rose Kovarik Method and system for treating cancer cachexia
US9528124B2 (en) 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
CN104284669A (zh) 2012-02-24 2015-01-14 弗雷德哈钦森癌症研究中心 治疗血红蛋白病的组合物和方法
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
EP3747891A3 (en) 2012-05-21 2021-03-24 Agilent Technologies, Inc. Compositions and methods for conjugating oligonucleotides
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
EP2880171B1 (en) 2012-08-03 2018-10-03 The Regents of The University of California Methods and compositions for controlling gene expression by rna processing
UA118090C2 (uk) 2012-09-07 2018-11-26 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив
US20140075593A1 (en) 2012-09-07 2014-03-13 Dow Agrosciences Llc Fluorescence activated cell sorting (facs) enrichment to generate plants
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
CA2884162C (en) 2012-09-07 2020-12-29 Dow Agrosciences Llc Fad3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
KR101656236B1 (ko) 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
WO2014071235A1 (en) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
BR122019025681B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
EP3401388B1 (en) 2012-12-06 2019-07-10 Synthetic Genomics, Inc. Algal mutants having a locked-in high light acclimated phenotype
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
WO2014093688A1 (en) 2012-12-12 2014-06-19 1Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
WO2014159719A1 (en) 2013-03-14 2014-10-02 Scrips Health Methods of isolating nucleic acids
US20160138027A1 (en) 2013-03-14 2016-05-19 The Board Of Trustees Of The Leland Stanford Junior University Treatment of diseases and conditions associated with dysregulation of mammalian target of rapamycin complex 1 (mtorc1)
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
EP2971006A4 (en) 2013-03-15 2017-02-08 Transposagen Biopharmaceuticals, Inc. Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
US11274305B2 (en) 2013-04-04 2022-03-15 Trustees Of Dartmouth College Compositions and methods for in vivo excision of HIV-1 proviral DNA
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2014172458A1 (en) 2013-04-16 2014-10-23 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
DK3004337T3 (da) 2013-05-29 2017-11-13 Cellectis Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
EP3004349B1 (en) 2013-05-29 2018-03-28 Cellectis S.A. A method for producing precise dna cleavage using cas9 nickase activity
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
RU2690935C2 (ru) 2013-06-04 2019-06-06 Президент Энд Фэллоуз Оф Харвард Коллидж Направляемая рнк регуляция транскрипции
US9982277B2 (en) 2013-06-11 2018-05-29 The Regents Of The University Of California Methods and compositions for target DNA modification
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
EP3011035B1 (en) 2013-06-17 2020-05-13 The Broad Institute, Inc. Assay for quantitative evaluation of target site cleavage by one or more crispr-cas guide sequences
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
CA2917638C (en) 2013-07-09 2024-09-10 Harvard College RNA MULTIPLEX GUIDED GENOMIC ENGINEERING
SG10201800213VA (en) 2013-07-10 2018-02-27 Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
EP3019005B1 (en) 2013-07-10 2019-02-20 EffStock, LLC Mrap2 knockouts
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
AU2014315335B2 (en) 2013-09-04 2017-08-24 Corteva Agriscience Llc Rapid targeting analysis in crops for determining donor insertion
WO2015035034A1 (en) 2013-09-04 2015-03-12 Mice With Horns, Llc Materials and methods for correcting recessive mutations in animals
WO2015033293A1 (en) 2013-09-04 2015-03-12 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US20150071946A1 (en) 2013-09-06 2015-03-12 The Johns Hopkins University Tumor-specific retrotransposon insertions
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
EP3988649B1 (en) 2013-09-18 2024-11-27 Kymab Limited Methods, cells and organisms
AU2014331939A1 (en) 2013-10-08 2016-04-28 Amelia HENRY Drought-resistant cereal grasses and related materials and methods
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
WO2015066638A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal maize loci
US10233465B2 (en) 2013-11-04 2019-03-19 Dow Agrosciences Llc Optimal soybean loci
BR102014027442B1 (pt) 2013-11-04 2022-09-27 Dow Agrosciences Llc Molécula de ácido nucleico recombinante, uso de uma planta de milho, parte de planta de milho ou célula de planta de milho compreendendo a mesma e método para produzir uma célula vegetal transgênica compreendendo um dna de interesse
AU2014341928B2 (en) 2013-11-04 2017-11-30 Corteva Agriscience Llc A universal donor system for gene targeting
EP3862434A1 (en) 2013-11-04 2021-08-11 Dow AgroSciences LLC Optimal soybean loci
WO2015070062A1 (en) 2013-11-07 2015-05-14 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
WO2015075056A1 (en) 2013-11-19 2015-05-28 Thermo Fisher Scientific Baltics Uab Programmable enzymes for isolation of specific dna fragments
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
BR112016013201B1 (pt) 2013-12-12 2023-01-31 The Broad Institute, Inc. Uso de uma composição compreendendo um sistema crispr-cas no tratamento de uma doença genética ocular
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3080266B1 (en) 2013-12-12 2021-02-03 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
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
EP3079726B1 (en) 2013-12-12 2018-12-05 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
EP3540051B1 (en) 2013-12-12 2022-08-17 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hsv and viral diseases and disorders.
JP2017503485A (ja) 2013-12-12 2017-02-02 ザ・ブロード・インスティテュート・インコーポレイテッド 遺伝子産物の発現、構造情報、及び誘導性モジュラーｃａｓ酵素を変更するためのｃｒｉｓｐｒ−ｃａｓ系並びに方法
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3553176A1 (en) * 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3122880B1 (en) * 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US20160060655A1 (en) 2014-05-30 2016-03-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
US20150344836A1 (en) 2014-05-30 2015-12-03 Ohio State Innovation Foundation Agrobacterium Strains for Plant Transformation and Related Materials and Methods
WO2015191693A2 (en) * 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
IL286474B2 (en) 2014-06-23 2023-11-01 Massachusetts Gen Hospital Genome-wide random identification of DSBS assessed by sequencing (guide-sequence)
MX384887B (es) 2014-06-23 2025-03-14 Regeneron Pharma Ensamblaje de adn mediado por nucleasa.
WO2015200725A1 (en) 2014-06-25 2015-12-30 Cold Spring Harbor Laboratory Methods and compositions for inhibiting growth and epithelial to mesenchymal transition (emt) in cancer cells
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US9970030B2 (en) 2014-08-27 2018-05-15 Caribou Biosciences, Inc. Methods for increasing CAS9-mediated engineering efficiency
CN113584015B (zh) 2014-08-27 2025-05-02 新英格兰生物实验室公司 合成子的形成
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
US11015209B2 (en) 2014-09-26 2021-05-25 Pioneer Hi-Bred International, Inc. Wheat MS1 polynucleotides, polypeptides, and methods of use
BR112017003842B1 (pt) 2014-09-26 2024-02-20 Philip Morris Products S.A. Método para produção de um produto de tabaco
WO2016073079A2 (en) 2014-09-26 2016-05-12 Yale University Compositions and methods for biocontainment of microorganisms
CN107002140A (zh) 2014-09-26 2017-08-01 双孔人公司 通过合成探针的纳米孔隙探测的靶序列检测
EP3204513A2 (en) 2014-10-09 2017-08-16 Life Technologies Corporation Crispr oligonucleotides and gene editing
CN107106615A (zh) 2014-10-09 2017-08-29 人类起源公司 胎盘来源的贴壁细胞外泌体及其用途
EP4427809A3 (en) 2014-10-31 2024-12-04 The Trustees of The University of Pennsylvania Altering gene expression in car-t cells and uses thereof
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
JP6788584B2 (ja) 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 Ｃｒｉｓｐｒについての超並列コンビナトリアル遺伝学
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
EP3234133B1 (en) * 2014-12-18 2020-11-11 Integrated DNA Technologies, Inc. Crispr-based compositions and methods of use
ES2884838T3 (es) * 2015-04-06 2021-12-13 Univ Leland Stanford Junior ARN guía químicamente modificados para la regulación génica mediada por CRISPR/CAS
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
FI3390632T3 (fi) 2015-12-18 2025-11-25 Danisco Us Inc Menetelmät ja koostumukset polymeraasi ii (pol-ii) -pohjaisen opas-rna:n ilmentämiseen
WO2017106569A1 (en) 2015-12-18 2017-06-22 The Regents Of The University Of California Modified site-directed modifying polypeptides and methods of use thereof
CN109072218B (zh) 2015-12-18 2023-04-18 国立研究开发法人科学技术振兴机构 基因修饰非人生物、卵细胞、受精卵以及目的基因的修饰方法
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014093712A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
WO2014093635A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
WO2015006747A2 (en) * 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use.
WO2015026885A1 (en) * 2013-08-22 2015-02-26 Pioneer Hi-Bred International, Inc. Genome modification using guide polynucleotide/cas endonuclease systems and methods of use
CN103614415A (zh) * 2013-11-27 2014-03-05 苏州同善生物科技有限公司 一种基于crispr基因敲除技术建立肥胖症大鼠动物模型的方法

Also Published As

Publication number Publication date
AU2022204254B2 (en) 2024-06-27
KR102648489B1 (ko) 2024-03-15
US11851652B2 (en) 2023-12-26
JP6873911B2 (ja) 2021-05-19
US11535846B2 (en) 2022-12-27
US11306309B2 (en) 2022-04-19
CA2981715A1 (en) 2016-10-13
CN114231527A (zh) 2022-03-25
US20220195425A1 (en) 2022-06-23
US20220195426A1 (en) 2022-06-23
AU2022204254A1 (en) 2022-07-07
WO2016164356A1 (en) 2016-10-13
AU2016246450A1 (en) 2017-11-23
US20240401034A1 (en) 2024-12-05
KR20170134649A (ko) 2017-12-06
ES2884838T3 (es) 2021-12-13
KR20240038141A (ko) 2024-03-22
KR102888521B1 (ko) 2025-11-19
US20180119140A1 (en) 2018-05-03
CN107787367A (zh) 2018-03-09
EP3280803B1 (en) 2021-05-26
AU2016246450B2 (en) 2022-03-17
EP3280803A1 (en) 2018-02-14
US20220195427A1 (en) 2022-06-23
JP2018513682A (ja) 2018-05-31

Similar Documents

Publication Publication Date Title
AU2022204254B2 (en) Chemically modified guide rnas for crispr/cas-mediated gene regulation
US20230250423A1 (en) Genome editing of human neural stem cells using nucleases
US11884915B2 (en) Guide RNAs with chemical modification for prime editing
US20230340468A1 (en) Methods for using guide rnas with chemical modifications
CN118043465A (zh) 用于使用具有化学修饰的指导rna的方法
CA3230928A1 (en) Methods for using guide rnas with chemical modifications
HK40003944B (en) Genome editing of human neural stem cells using nucleases
HK40003944A (en) Genome editing of human neural stem cells using nucleases

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant