KR102888521B1 - Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna - Google Patents

Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna

Info

Publication number
KR102888521B1
KR102888521B1 KR1020247008357A KR20247008357A KR102888521B1 KR 102888521 B1 KR102888521 B1 KR 102888521B1 KR 1020247008357 A KR1020247008357 A KR 1020247008357A KR 20247008357 A KR20247008357 A KR 20247008357A KR 102888521 B1 KR102888521 B1 KR 102888521B1
Authority
KR
South Korea
Prior art keywords
sgrna
cells
modified
target
sequence
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
KR1020247008357A
Other languages
English (en)
Korean (ko)
Other versions
KR20240038141A (ko
Inventor
매튜 에이치 폴테우스
아얄 헨델
조 클라크
라스무스 오 백
다니엘 이 라이언
더글라스 제이 델린저
로버트 카이저
조엘 마이어슨
Original Assignee
더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
애질런트 테크놀로지스, 인크.
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=KR102888521(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티, 애질런트 테크놀로지스, 인크. filed Critical 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티
Publication of KR20240038141A publication Critical patent/KR20240038141A/ko
Application granted granted Critical
Publication of KR102888521B1 publication Critical patent/KR102888521B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • C12N15/11—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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Addiction (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
KR1020247008357A 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna Active KR102888521B1 (ko)

Applications Claiming Priority (6)

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
KR1020177031965A KR102648489B1 (ko) 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020177031965A Division KR102648489B1 (ko) 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna

Publications (2)

Publication Number Publication Date
KR20240038141A KR20240038141A (ko) 2024-03-22
KR102888521B1 true KR102888521B1 (ko) 2025-11-19

Family

ID=55910340

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020177031965A Active KR102648489B1 (ko) 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
KR1020247008357A Active KR102888521B1 (ko) 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna

Family Applications Before (1)

Application Number Title Priority Date Filing Date
KR1020177031965A Active KR102648489B1 (ko) 2015-04-06 2016-04-05 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna

Country Status (9)

Country Link
US (5) US11306309B2 (enExample)
EP (1) EP3280803B1 (enExample)
JP (1) JP6873911B2 (enExample)
KR (2) KR102648489B1 (enExample)
CN (2) CN114231527A (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
EP3613852A3 (en) 2011-07-22 2020-04-22 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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3126497B1 (en) * 2014-04-01 2018-12-12 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
AU2015355546B2 (en) * 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
CN119320775A (zh) 2014-12-18 2025-01-17 综合基因技术公司 基于crispr的组合物和使用方法
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US20180030438A1 (en) 2015-02-23 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2017004279A2 (en) 2015-06-29 2017-01-05 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
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
US11518994B2 (en) 2016-01-30 2022-12-06 Bonac Corporation Artificial single guide RNA and use thereof
WO2017136794A1 (en) 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
KR101710026B1 (ko) 2016-08-10 2017-02-27 주식회사 무진메디 Cas9 단백질 및 가이드 RNA의 혼성체를 함유하는 나노 리포좀 전달체 조성물
SG11201901184QA (en) * 2016-08-12 2019-03-28 Toolgen Inc Manipulated immunoregulatory element and immunity altered thereby
CA3034369A1 (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
EP3510152A4 (en) 2016-09-07 2020-04-29 Flagship Pioneering, Inc. METHODS AND COMPOSITIONS FOR MODULATING GENE EXPRESSION
KR102606680B1 (ko) 2016-10-07 2023-11-27 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Ｓ. 피오게네스 ｃａｓ９ 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
GB2573062A (en) 2016-10-14 2019-10-23 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
SG10202106058WA (en) * 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
US11293022B2 (en) 2016-12-12 2022-04-05 Integrated Dna Technologies, Inc. Genome editing enhancement
WO2018111944A1 (en) 2016-12-12 2018-06-21 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
CN110300803B (zh) 2016-12-20 2024-05-14 百时美施贵宝公司 提高细胞基因组中同源定向修复(hdr)效率的方法
TW202509233A (zh) * 2016-12-22 2025-03-01 美商英特利亞醫療公司 用於治療α-1抗胰蛋白酶缺乏症之組合物及方法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3565895A1 (en) * 2016-12-30 2019-11-13 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
US11873496B2 (en) 2017-01-09 2024-01-16 Whitehead Institute For Biomedical Research Methods of altering gene expression by perturbing transcription factor multimers that structure regulatory loops
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
ES2969213T3 (es) * 2017-02-15 2024-05-17 2Seventy Bio Inc Plantillas de reparación de donantes para edición multiplex del genoma
WO2018154412A1 (en) * 2017-02-22 2018-08-30 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
AU2018229351B2 (en) 2017-02-28 2024-01-04 Vor Biopharma, Inc. Compositions and methods for inhibition of lineage specific proteins
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
WO2018175480A1 (en) * 2017-03-20 2018-09-27 The Research Foundation For The State University Of New York Methods of inhibiting fungal ceramide synthase for treatment of cryptococcus neoformans infection
US11268082B2 (en) 2017-03-23 2022-03-08 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
US12275963B2 (en) 2017-05-08 2025-04-15 Toolgen Incorporated Artificially manipulated immune cell
JP7356354B2 (ja) 2017-05-12 2023-10-04 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 細胞の操作のための材料及び方法並びに免疫腫瘍学におけるその使用
US11166985B2 (en) 2017-05-12 2021-11-09 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
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
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
SG11202005147WA (en) 2017-12-05 2020-06-29 Vertex Pharma Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019143077A1 (ko) * 2018-01-22 2019-07-25 경상대학교산학협력단 식물체에서 상동재조합 기반의 유전자 편집 효율을 증가시키는 방법
US20190233816A1 (en) * 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749767A1 (en) 2018-02-05 2020-12-16 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
CN112534051B (zh) * 2018-03-29 2025-07-29 学校法人自治医科大学 基因组编辑方法、组合物、细胞、细胞制剂、以及细胞制剂的制造方法
CA3096708A1 (en) * 2018-04-18 2019-10-24 Yale University Compositions and methods for multiplexed tumor vaccination with endogenous gene activation
SG11202011080QA (en) 2018-05-11 2020-12-30 Crispr Therapeutics Ag Methods and compositions for treating cancer
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 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
US20210217494A1 (en) * 2018-05-25 2021-07-15 New York Institute Of Technology Method and system for use in direct sequencing of rna
CN112513243A (zh) * 2018-06-01 2021-03-16 阿维塔斯有限公司 细胞工程平台
EP3802828A4 (en) * 2018-06-08 2022-10-26 Intellia Therapeutics, Inc. RNA MODIFIED GUIDES FOR GENE EDITING
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
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
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
WO2020047164A1 (en) 2018-08-28 2020-03-05 Vor Biopharma, Inc Genetically engineered hematopoietic stem cells and uses thereof
CA3113676A1 (en) 2018-09-21 2020-03-26 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
WO2020092453A1 (en) 2018-10-29 2020-05-07 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
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022531185A (ja) 2019-04-30 2022-07-06 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 遺伝子改変ｔ細胞を標的化するｃｄ１９を使用するｂ細胞悪性病変の同種細胞療法
CN114423865A (zh) 2019-05-23 2022-04-29 Vor生物制药股份有限公司 Cd33修饰的组合物和方法
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
US20220315938A1 (en) * 2019-08-09 2022-10-06 Regents Of The University Of Minnesota AUGMENTED sgRNAS AND METHODS FOR THEIR USE TO ENHANCE SOMATIC AND GERMLINE PLANT GENOME ENGINEERING
MX2022002462A (es) 2019-08-28 2022-06-02 Vor Biopharma Inc Composiciones y métodos para la modificación de cll1.
KR20220047381A (ko) 2019-08-28 2022-04-15 보르 바이오파마 인크. Cd123 변형을 위한 조성물 및 방법
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
CA3153197A1 (en) 2019-10-03 2021-04-08 Ryan T. Gill 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
JP2022551551A (ja) * 2019-10-11 2022-12-12 ブラックホーク ジェノミクス エルエルシー ロングリードシークエンシングを用いたゲノム構造バリアントの同定
CN115279898A (zh) 2019-10-23 2022-11-01 成对植物服务股份有限公司 用于植物中rna模板化编辑的组合物和方法
KR20220110748A (ko) * 2019-11-05 2022-08-09 페어와이즈 플랜츠 서비시즈, 인크. 대립유전자의 rna-코딩된 dna-대체를 위한 조성물 및 방법
JP2023506482A (ja) * 2019-12-11 2023-02-16 インテリア セラピューティクス，インコーポレイテッド 遺伝子編集のための修飾されたガイドｒｎａ
IL271656A (en) 2019-12-22 2021-06-30 Yeda Res & Dev System and methods for identifying cells that have undergone genome editing
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
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
US20240238344A1 (en) 2020-08-28 2024-07-18 Vor Biopharma Inc. Compositions and methods for cd123 modification
JP2023540276A (ja) 2020-08-28 2023-09-22 ブイオーアール バイオファーマ インコーポレーテッド Ｃｌｌ１改変のための組成物および方法
EP4211244A1 (en) 2020-09-14 2023-07-19 Vor Biopharma, Inc. Compositions and methods for cd38 modification
US20240041932A1 (en) 2020-09-14 2024-02-08 Vor Biopharma Inc. Compositions and methods for cd5 modification
US20240033290A1 (en) 2020-09-18 2024-02-01 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
WO2022081585A1 (en) * 2020-10-12 2022-04-21 The Board Of Trustees Of The Leland Stanford Junior University Gene correction for x-cgd in hematopoietic stem and progenitor cells
WO2022093983A1 (en) 2020-10-27 2022-05-05 Vor Biopharma, Inc. Compositions and methods for treating hematopoietic malignancy
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
KR20230107610A (ko) 2020-11-13 2023-07-17 보르 바이오파마 인크. 키메라 항원 수용체를 발현하는 유전적으로 조작된 세포와 관련된 방법 및 조성물
US20240366675A1 (en) 2020-12-31 2024-11-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CA3209863A1 (en) 2021-02-25 2022-09-01 Andrea BARGHETTI 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
EP4370676A2 (en) 2021-06-18 2024-05-22 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
EP4381062A1 (en) 2021-08-02 2024-06-12 Vor Biopharma Inc. Compositions and methods for gene modification
WO2023039586A1 (en) * 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
EP4402266A4 (en) * 2021-09-14 2025-11-05 Agilent Technologies Inc GUIDELINES FOR USING RNA WITH CHEMICAL MODIFICATIONS
JP2024535920A (ja) 2021-09-27 2024-10-02 ブイオーアール バイオファーマ インコーポレーテッド 遺伝子編集のための融合ポリペプチド及びその使用方法
US20240382528A1 (en) * 2021-10-05 2024-11-21 The Board Of Trustees Of The Leland Stanford Junior University Treatment of polycythemia vera via crispr/aav6 genome editing
JP2024543369A (ja) 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Ｅｍｒ２修飾のための化合物及び方法
WO2023155924A1 (en) * 2022-02-21 2023-08-24 Huidagene Therapeutics Co., Ltd. Guide rna and uses thereof
EP4482960A1 (en) 2022-02-25 2025-01-01 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
CN118974256A (zh) 2022-03-29 2024-11-15 锐正基因(苏州)有限公司 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
EP4504771A1 (en) 2022-04-04 2025-02-12 Vor Biopharma Inc. Compositions and methods for mediating epitope engineering
US20250308637A1 (en) 2022-05-20 2025-10-02 Celyntra Therapeutics Sa 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
CN119948162A (zh) 2022-09-22 2025-05-06 锐正基因(苏州)有限公司 用于治疗高胆固醇血症和/或心血管疾病的组合物和方法
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
EP4638477A1 (en) 2022-12-19 2025-10-29 Kamau Therapeutics, 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 (3)

* 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
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 (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5132418A (en) 1980-02-29 1992-07-21 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
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 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
HUE028389T2 (en) 2007-05-10 2016-12-28 Agilent Technologies Inc For the synthesis of thiocarbons protecting groups RNSS
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
JP5340265B2 (ja) 2008-06-27 2013-11-13 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
US20100076183A1 (en) 2008-09-22 2010-03-25 Dellinger Douglas J Protected monomer and method of final deprotection for rna synthesis
AU2010279913B2 (en) 2009-08-07 2016-04-28 Kyoto University Method of efficiently establishing induced pluripotent stem cells
JP5682043B2 (ja) 2009-09-02 2015-03-11 国立大学法人京都大学 安全な多能性幹細胞の選択方法
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
BR112014020625A2 (pt) 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
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
CN104321332A (zh) 2012-05-21 2015-01-28 安捷伦科技有限公司 组合物和缀合寡核苷酸的方法
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
HK1207111A1 (en) 2012-08-03 2016-01-22 加利福尼亚大学董事会 Methods and compositions for controlling gene expression by rna processing
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
US20140075593A1 (en) 2012-09-07 2014-03-13 Dow Agrosciences Llc Fluorescence activated cell sorting (facs) enrichment to generate plants
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
AU2013312538B2 (en) 2012-09-07 2019-01-24 Corteva Agriscience Llc FAD3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
JP6517143B2 (ja) 2012-10-23 2019-05-22 ツールゲン インコーポレイテッド 標的ｄｎａに特異的なガイドｒｎａおよびｃａｓタンパク質コード核酸またはｃａｓタンパク質を含む、標的ｄｎａを切断するための組成物、ならびにその使用
KR102315098B1 (ko) 2012-11-01 2021-10-21 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
WO2014071235A1 (en) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
MX358837B (es) 2012-12-06 2018-09-05 Synthetic Genomics Inc Mutantes de algas que tienen un fenotipo aclimatado a alta incidencia de luz, bloqueado.
US9447422B2 (en) 2012-12-06 2016-09-20 Synthetic Genomics, Inc. Autonomous replication sequences and episomal DNA molecules
JP2016505256A (ja) 2012-12-12 2016-02-25 ザ・ブロード・インスティテュート・インコーポレイテッ 配列操作のためのＣＲＩＳＰＲ−Ｃａｓ成分系、方法および組成物
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
PL2896697T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Projektowanie systemów, sposoby i optymalizowane kompozycje kierujące do manipulacji sekwencją
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
US9970002B2 (en) 2012-12-12 2018-05-15 Massachusetts Institute Of Technology Compositions and methods for functional nucleic acid delivery
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
JP6700788B2 (ja) 2012-12-17 2020-05-27 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｒｎａ誘導性ヒトゲノム改変
WO2014159719A1 (en) 2013-03-14 2014-10-02 Scrips Health Methods of isolating nucleic acids
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting 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)
WO2014145736A2 (en) 2013-03-15 2014-09-18 Transposagen Biopharmaceuticals, Inc. Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm)
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
KR102271292B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
AU2014227831B2 (en) 2013-03-15 2020-01-30 Regents Of The University Of Minnesota Engineering plant genomes using CRISPR/Cas systems
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
MX369747B (es) 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
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
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
HK1223401A1 (zh) 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
AU2014273490B2 (en) 2013-05-29 2019-05-09 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system
WO2014191518A1 (en) 2013-05-29 2014-12-04 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
EP3003392B1 (en) 2013-06-04 2019-10-23 President and Fellows of Harvard College Rna-guideded transcriptional regulation
US9267135B2 (en) 2013-06-04 2016-02-23 President And Fellows Of Harvard College RNA-guided transcriptional regulation
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
EP3008181B1 (en) 2013-06-11 2019-11-06 The Regents of The University of California Methods and compositions for target dna modification
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
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
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
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
WO2015006290A1 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Multiplex rna-guided genome engineering
JP2016528890A (ja) 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
WO2015006437A1 (en) 2013-07-10 2015-01-15 Majzoub Joseph A Mrap2 knockouts
AU2014287397B2 (en) 2013-07-10 2019-10-10 President And Fellows Of Harvard College Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
RS62529B1 (sr) * 2013-07-11 2021-11-30 Modernatx Inc Kompozicije koje sadrže sintetičke polinukleotide koji kodiraju proteine povezane sa crispr i sintetičke sgrnk i postupci upotrebe
US10563225B2 (en) 2013-07-26 2020-02-18 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
US20150064149A1 (en) 2013-09-04 2015-03-05 Mice With Horns, LLC. Materials and methods for correcting recessive mutations in animals
EP3041344A4 (en) 2013-09-04 2017-04-19 Dow AgroSciences LLC Rapid targeting analysis in crops for determining donor insertion
US10167466B2 (en) 2013-09-04 2019-01-01 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
WO2015040075A1 (en) 2013-09-18 2015-03-26 Genome Research Limited Genomic screening methods using rna-guided endonucleases
WO2015040402A1 (en) 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US20160251675A1 (en) 2013-10-08 2016-09-01 International Rice Research Institute Drought-Resistant Cereal Grasses and Related Materials and Methods
DE102013111099B4 (de) 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
US20150098954A1 (en) 2013-10-08 2015-04-09 Elwha Llc Compositions and Methods Related to CRISPR Targeting
WO2015066634A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal soybean loci
NZ735257A (en) 2013-11-04 2018-09-28 Dow Agrosciences Llc Optimal soybean loci
WO2015066636A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal maize loci
JP6633532B2 (ja) 2013-11-04 2020-01-22 ダウ アグロサイエンシィズ エルエルシー 最適なトウモロコシ遺伝子座
CA2926534A1 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc A universal donor system for gene targeting
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US11326209B2 (en) 2013-11-07 2022-05-10 Massachusetts Institute Of Technology Cell-based genomic recorded accumulative memory
WO2015075056A1 (en) 2013-11-19 2015-05-28 Thermo Fisher Scientific Baltics Uab Programmable enzymes for isolation of specific dna fragments
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
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
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
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
SG10201804977UA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Particle Delivery Components
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3653703A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
WO2015089462A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
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
CA2932439A1 (en) 2013-12-12 2015-06-18 The Broad Institute, Inc. Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes
ES2745769T3 (es) * 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US20150344836A1 (en) 2014-05-30 2015-12-03 Ohio State Innovation Foundation Agrobacterium Strains for Plant Transformation and Related Materials and Methods
AU2015266776A1 (en) 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
WO2015191693A2 (en) * 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
DK3354732T3 (da) 2014-06-23 2020-04-06 Regeneron Pharma Nukleasemedieret dna-samling
KR102425438B1 (ko) 2014-06-23 2022-07-27 더 제너럴 하스피탈 코포레이션 서열결정에 의해 평가된 DSB의 게놈 전체에 걸친 비편향된 확인 (GUIDE-Seq)
WO2015200555A2 (en) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
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
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
DK3186376T3 (da) 2014-08-27 2019-05-06 Caribou Biosciences Inc Fremgangsmåder til forbedring af cas9-medieret manipulationseffektivitet
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
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
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
RU2721799C2 (ru) 2014-09-26 2020-05-22 Филип Моррис Продактс С.А. Уменьшение содержания табак-специфичных нитрозаминов посредством изменения пути ассимиляции нитратов
US11015209B2 (en) 2014-09-26 2021-05-25 Pioneer Hi-Bred International, Inc. Wheat MS1 polynucleotides, polypeptides, and methods of use
EP3198036A4 (en) 2014-09-26 2018-08-08 Two Pore Guys, Inc. Target sequence detection by nanopore sensing of synthetic probes
US11408007B2 (en) 2014-09-26 2022-08-09 Yale University Compositions and methods for biocontainment of microorganisms
CN116731958A (zh) 2014-10-09 2023-09-12 细胞结构公司 胎盘来源的贴壁细胞外泌体及其用途
EP3998344A1 (en) 2014-10-09 2022-05-18 Life Technologies Corporation Crispr oligonucleotides and gene editing
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
CN107206024B (zh) 2014-10-31 2021-12-03 宾夕法尼亚大学董事会 改变cart细胞中的基因表达及其用途
US20190100769A1 (en) 2014-10-31 2019-04-04 Massachusetts Institute Of Technology Massively parallel combinatorial genetics for crispr
AU2015355546B2 (en) 2014-12-03 2021-10-14 Agilent Technologies, Inc. Guide RNA with chemical modifications
CN119320775A (zh) * 2014-12-18 2025-01-17 综合基因技术公司 基于crispr的组合物和使用方法
WO2016164356A1 (en) * 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
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
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
FI3390632T3 (fi) 2015-12-18 2025-11-25 Danisco Us Inc Menetelmät ja koostumukset polymeraasi ii (pol-ii) -pohjaisen opas-rna:n ilmentämiseen
EP3390631B1 (en) 2015-12-18 2020-04-08 Danisco US Inc. Methods and compositions for t-rna based guide rna expression
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
JP6700306B2 (ja) 2015-12-18 2020-05-27 国立研究開発法人科学技術振興機構 受精前の卵細胞、受精卵、及び標的遺伝子の改変方法
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs

Patent Citations (3)

* 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
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

Also Published As

Publication number Publication date
AU2022204254B2 (en) 2024-06-27
AU2016246450B2 (en) 2022-03-17
US20220195427A1 (en) 2022-06-23
US20220195426A1 (en) 2022-06-23
CN114231527A (zh) 2022-03-25
JP2018513682A (ja) 2018-05-31
JP6873911B2 (ja) 2021-05-19
ES2884838T3 (es) 2021-12-13
US20220195425A1 (en) 2022-06-23
KR20240038141A (ko) 2024-03-22
WO2016164356A1 (en) 2016-10-13
AU2016246450A1 (en) 2017-11-23
US11851652B2 (en) 2023-12-26
EP3280803A1 (en) 2018-02-14
CA2981715A1 (en) 2016-10-13
EP3280803B1 (en) 2021-05-26
CN107787367A (zh) 2018-03-09
KR102648489B1 (ko) 2024-03-15
US20180119140A1 (en) 2018-05-03
US11306309B2 (en) 2022-04-19
US11535846B2 (en) 2022-12-27
KR20170134649A (ko) 2017-12-06
US20240401034A1 (en) 2024-12-05
AU2022204254A1 (en) 2022-07-07
CN107787367B (zh) 2021-10-26

Similar Documents

Publication Publication Date Title
KR102888521B1 (ko) Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
KR102628801B1 (ko) 세포내 유전자 변형 및 증가된 상동 재조합을 위한 보호 dna 주형 및 이용 방법
KR102723347B1 (ko) 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물
JP2025020189A (ja) 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
JP2025134087A (ja) 異常ヘモグロビン症の治療用組成物及び方法
US20030162265A1 (en) Dna joining method
CA3175106A1 (en) Compositions and methods for modifying a target nucleic acid
EP4323521A1 (en) Casrx/cas13d systems targeting c9orf72
US20210155927A1 (en) Methods for treating sickle cell disease
CN114836444A (zh) 重组肠道病毒a71病毒样颗粒及其用途
CN103834692B (zh) 一种用于表达lncRNA的慢病毒载体及其应用
KR102084227B1 (ko) 인간 형질전환 연골세포주 기반 연골질환 치료제 스크리닝 시스템
CA2402321C (en) Dna joining method
WO2021260227A1 (en) Pyruvate kinase deficiency (pkd) gene editing treatment method
EP4433592A1 (en) Compositions and methods for production of circular nucleic acid molecules
CN113073118B (zh) Tet-off表达调控系统在级联放大启动子活性中的用途
KR100927098B1 (ko) 에피토프 태그 치환용 재조합 벡터
KR20150045878A (ko) 구제역 바이러스의 ｖｐ1 유전자를 포함하는 돼지 생식기 호흡기 증후군 탈당 바이러스

Legal Events

Date Code Title Description
A107 Divisional application of patent
PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A18-div-PA0104

St.27 status event code: A-0-1-A10-A16-div-PA0104

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601