JP6657069B2 - 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化 - Google Patents

特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化 Download PDF

Info

Publication number
JP6657069B2
JP6657069B2 JP2016502406A JP2016502406A JP6657069B2 JP 6657069 B2 JP6657069 B2 JP 6657069B2 JP 2016502406 A JP2016502406 A JP 2016502406A JP 2016502406 A JP2016502406 A JP 2016502406A JP 6657069 B2 JP6657069 B2 JP 6657069B2
Authority
JP
Japan
Prior art keywords
protein
cas9
sequence
dcas9
expression
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
JP2016502406A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016512264A (ja
Inventor
ジュン，ジェイ．キース
メーダー，モーガン
Original Assignee
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
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=51537665&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP6657069(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by ザ ジェネラル ホスピタル コーポレイション, ザ ジェネラル ホスピタル コーポレイション filed Critical ザ ジェネラル ホスピタル コーポレイション
Publication of JP2016512264A publication Critical patent/JP2016512264A/ja
Priority to JP2019176599A priority Critical patent/JP6960438B2/ja
Application granted granted Critical
Publication of JP6657069B2 publication Critical patent/JP6657069B2/ja
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
    • 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
    • C12N15/87—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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1031—Mutagenizing nucleic acids mutagenesis by gene assembly, e.g. assembly by oligonucleotide extension PCR
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • C12N9/10—Transferases (2.)
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • 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)
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/00033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/00033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/11—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with 2-oxoglutarate as one donor, and incorporation of one atom each of oxygen into both donors (1.14.11)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Developing Agents For Electrophotography (AREA)
  • Prostheses (AREA)
  • Lubricants (AREA)
JP2016502406A 2013-03-15 2014-03-14 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化 Active JP6657069B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2019176599A JP6960438B2 (ja) 2013-03-15 2019-09-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361799647P 2013-03-15 2013-03-15
US61/799,647 2013-03-15
US201361838178P 2013-06-21 2013-06-21
US201361838148P 2013-06-21 2013-06-21
US61/838,178 2013-06-21
US61/838,148 2013-06-21
US201361921007P 2013-12-26 2013-12-26
US61/921,007 2013-12-26
PCT/US2014/027335 WO2014152432A2 (en) 2013-03-15 2014-03-14 Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019176599A Division JP6960438B2 (ja) 2013-03-15 2019-09-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化

Publications (2)

Publication Number Publication Date
JP2016512264A JP2016512264A (ja) 2016-04-25
JP6657069B2 true JP6657069B2 (ja) 2020-03-04

Family

ID=51537665

Family Applications (11)

Application Number Title Priority Date Filing Date
JP2016502406A Active JP6657069B2 (ja) 2013-03-15 2014-03-14 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2016502853A Active JP6622183B2 (ja) 2013-03-15 2014-03-14 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2016502976A Active JP6980380B2 (ja) 2013-03-15 2014-03-14 短縮ガイドＲＮＡ（ｔｒｕ−ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2019176599A Expired - Fee Related JP6960438B2 (ja) 2013-03-15 2019-09-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2019210428A Expired - Fee Related JP6878554B2 (ja) 2013-03-15 2019-11-21 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2020055863A Active JP7053706B2 (ja) 2013-03-15 2020-03-26 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2021075869A Active JP7126588B2 (ja) 2013-03-15 2021-04-28 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2021138479A Pending JP2021192624A (ja) 2013-03-15 2021-08-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2022056499A Active JP7379572B2 (ja) 2013-03-15 2022-03-30 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2023011473A Withdrawn JP2023061983A (ja) 2013-03-15 2023-01-30 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2023186663A Active JP7812830B2 (ja) 2013-03-15 2023-10-31 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大

Family Applications After (10)

Application Number Title Priority Date Filing Date
JP2016502853A Active JP6622183B2 (ja) 2013-03-15 2014-03-14 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2016502976A Active JP6980380B2 (ja) 2013-03-15 2014-03-14 短縮ガイドＲＮＡ（ｔｒｕ−ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2019176599A Expired - Fee Related JP6960438B2 (ja) 2013-03-15 2019-09-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2019210428A Expired - Fee Related JP6878554B2 (ja) 2013-03-15 2019-11-21 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2020055863A Active JP7053706B2 (ja) 2013-03-15 2020-03-26 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2021075869A Active JP7126588B2 (ja) 2013-03-15 2021-04-28 ＲＮＡ誘導型ＦｏｋＩヌクレアーゼ（ＲＦＮ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2021138479A Pending JP2021192624A (ja) 2013-03-15 2021-08-27 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2022056499A Active JP7379572B2 (ja) 2013-03-15 2022-03-30 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大
JP2023011473A Withdrawn JP2023061983A (ja) 2013-03-15 2023-01-30 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
JP2023186663A Active JP7812830B2 (ja) 2013-03-15 2023-10-31 短縮ガイドＲＮＡ（ｔｒｕ－ｇＲＮＡ）を用いたＲＮＡ誘導型ゲノム編集の特異性の増大

Country Status (12)

Country Link
US (17) US10378027B2 (enExample)
EP (10) EP3741868B1 (enExample)
JP (11) JP6657069B2 (enExample)
KR (8) KR102210323B1 (enExample)
CN (6) CN105408483A (enExample)
AU (10) AU2014228981B2 (enExample)
BR (1) BR112015023489B1 (enExample)
CA (4) CA2906553C (enExample)
ES (1) ES2713503T3 (enExample)
IL (2) IL289396B2 (enExample)
WO (4) WO2014144288A1 (enExample)
ZA (1) ZA201506814B (enExample)

Families Citing this family (465)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8053191B2 (en) 2006-08-31 2011-11-08 Westend Asset Clearinghouse Company, Llc Iterative nucleic acid assembly using activation of vector-encoded traits
WO2012048113A2 (en) 2010-10-07 2012-04-12 The General Hospital Corporation Biomarkers of cancer
CA2817697C (en) 2010-11-12 2021-11-16 Gen9, Inc. Methods and devices for nucleic acids synthesis
WO2012064975A1 (en) 2010-11-12 2012-05-18 Gen9, Inc. Protein arrays and methods of using and making the same
AR085533A1 (es) 2011-03-23 2013-10-09 Pioneer Hi Bred Int Metodos de produccion de un locus de rasgos transgenicos complejo
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP2014526899A (ja) 2011-08-26 2014-10-09 ジェン９・インコーポレイテッド 核酸の高忠実度アセンブリのための組成物および方法
EP2766498B1 (en) 2011-10-14 2019-06-19 President and Fellows of Harvard College Sequencing by structure assembly
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US10039777B2 (en) 2012-03-20 2018-08-07 Neuro-Lm Sas Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
CA2871505C (en) 2012-04-24 2021-10-12 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
WO2014004393A1 (en) 2012-06-25 2014-01-03 Gen9, Inc. Methods for nucleic acid assembly and high throughput sequencing
EP3789405A1 (en) * 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
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
WO2014093709A1 (en) 2012-12-12 2014-06-19 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
WO2014113493A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
EP2954042B1 (en) 2013-02-07 2017-12-06 The General Hospital Corporation Tale transcriptional activators
US10138509B2 (en) 2013-03-12 2018-11-27 President And Fellows Of Harvard College Method for generating a three-dimensional nucleic acid containing matrix
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
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
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US20150044772A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CN106133138A (zh) * 2013-08-22 2016-11-16 先锋国际良种公司 使用引导多核苷酸/cas内切核酸酶系统的基因组修饰及其使用方法
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) * 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
ES2681622T3 (es) 2013-09-18 2018-09-14 Kymab Limited Métodos, células y organismos
US10584358B2 (en) 2013-10-30 2020-03-10 North Carolina State University Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri
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
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
US20150166984A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
KR20160102056A (ko) * 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 ｒｎａ
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
LT3105328T (lt) 2014-02-11 2020-07-10 The Regents Of The University Of Colorado, A Body Corporate Daugiakomponentis genomo konstravimas, sudarantis sąlygas crispr
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
EP3129490A4 (en) 2014-04-08 2017-10-04 North Carolina State University Methods and compositions for rna-directed repression of transcription using crispr-associated genes
WO2015184262A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
AU2015280069B2 (en) 2014-06-23 2021-08-12 The General Hospital Corporation Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq)
WO2015200555A2 (en) * 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Rna modification to engineer cas9 activity
EP3161159B1 (en) 2014-06-25 2020-08-05 The General Hospital Corporation Targeting human satellite ii (hsatii)
WO2016007604A1 (en) * 2014-07-09 2016-01-14 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
EP3166391A1 (en) 2014-07-11 2017-05-17 E. I. du Pont de Nemours and Company Compositions and methods for producing plants resistant to glyphosate herbicide
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
BR112017003757A2 (pt) 2014-09-12 2017-12-26 Du Pont ?plantas de milho, partes de planta de milho ou sementes de milho?
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
EP3204496A1 (en) * 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US11352666B2 (en) 2014-11-14 2022-06-07 Institute For Basic Science Method for detecting off-target sites of programmable nucleases in a genome
WO2016076672A1 (ko) * 2014-11-14 2016-05-19 기초과학연구원 유전체에서 유전자 가위의 비표적 위치를 검출하는 방법
CN104531633A (zh) * 2014-11-18 2015-04-22 李云英 Cas9-scForkI融合蛋白及其应用
KR20160059994A (ko) 2014-11-19 2016-05-27 기초과학연구원 두 개의 벡터로부터 발현된 Cas9 단백질을 이용한 유전자 발현 조절 방법
CA2969619A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
CN107208079B (zh) * 2014-12-05 2021-06-29 应用干细胞有限公司 整合转基因的位点定向crispr/重组酶组合物和方法
WO2016094679A1 (en) 2014-12-10 2016-06-16 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3230452B1 (en) * 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) * 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
JP2017538427A (ja) * 2014-12-18 2017-12-28 インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド Ｃｒｉｓｐｒ系組成物及び使用方法
US20180179523A1 (en) * 2014-12-18 2018-06-28 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
US11208638B2 (en) 2015-01-12 2021-12-28 The Regents Of The University Of California Heterodimeric Cas9 and methods of use thereof
AU2016211517B2 (en) 2015-01-28 2021-05-13 Caribou Biosciences, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
EP3250689B1 (en) * 2015-01-28 2020-11-04 The Regents of The University of California Methods and compositions for labeling a single-stranded target nucleic acid
US10676726B2 (en) * 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
IL296837A (en) * 2015-02-18 2022-11-01 Univ Iowa State Res Found Inc Modification of transcriptional repressor binding site in nf-yc4 promoter for increased protein content and resistance to stress
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
BR112017017260A2 (pt) 2015-03-27 2018-04-17 Du Pont construções de dna, vetor, célula, plantas, semente, método de expressão de rna e método de modificação de local alvo
CN107787367B (zh) 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
EP4019975A1 (en) 2015-04-24 2022-06-29 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016196361A1 (en) 2015-05-29 2016-12-08 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
EP3324999A1 (en) 2015-05-29 2018-05-30 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
US11911415B2 (en) 2015-06-09 2024-02-27 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for improving transplantation
US11267852B2 (en) * 2015-06-10 2022-03-08 Firmenich Sa Cell lines for screening odorant and aroma receptors
IL298524B2 (en) 2015-06-12 2024-03-01 Lonza Walkersville Inc Methods for nuclear reprogramming using synthetic transcription factors
AU2016280002B2 (en) 2015-06-15 2022-04-14 North Carolina State University Methods and compositions for efficient delivery of nucleic acids and RNA-based antimicrobials
EP3929287A3 (en) 2015-06-18 2022-04-13 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US11279928B2 (en) * 2015-06-29 2022-03-22 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
JP2018519811A (ja) * 2015-06-29 2018-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ｃｒｉｓｐｒ ｒｎａ及び修飾単一ｃｒｉｓｐｒ ｒｎａならびにその使用
EP3957731A1 (en) * 2015-07-15 2022-02-23 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
WO2017023974A1 (en) * 2015-08-03 2017-02-09 President And Fellows Of Harvard College Cas9 genome editing and transcriptional regulation
WO2017024047A1 (en) * 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
KR20240132120A (ko) * 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
WO2017040348A1 (en) * 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
US10526590B2 (en) 2015-08-31 2020-01-07 Agilent Technologies, Inc. Compounds and methods for CRISPR/Cas-based genome editing by homologous recombination
WO2017044776A1 (en) * 2015-09-10 2017-03-16 Texas Tech University System Single-guide rna (sgrna) with improved knockout efficiency
CA3000816A1 (en) 2015-09-11 2017-03-16 The General Hospital Corporation Full interrogation of nuclease dsbs and sequencing (find-seq)
CA2999500A1 (en) 2015-09-24 2017-03-30 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US11286480B2 (en) 2015-09-28 2022-03-29 North Carolina State University Methods and compositions for sequence specific antimicrobials
CA3000762A1 (en) 2015-09-30 2017-04-06 The General Hospital Corporation Comprehensive in vitro reporting of cleavage events by sequencing (circle-seq)
CN111961683A (zh) 2015-10-06 2020-11-20 基础科学研究院 用于以高效率从植物原生质体生成基因组工程改造的植物的方法
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP3350327B1 (en) 2015-10-23 2018-09-26 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
CN108474022A (zh) 2015-11-03 2018-08-31 哈佛学院董事及会员团体 用于包含三维核酸的基质容积成像的设备和方法
US11566052B2 (en) 2015-11-11 2023-01-31 Lonza Ltd. CRISPR-associated (Cas) proteins with reduced immunogenicity
EP3414255B1 (en) 2015-11-20 2021-09-22 Washington University Preparative electrophoretic method for targeted purification of genomic dna fragments
EP3382018B1 (en) 2015-11-25 2022-03-30 National University Corporation Gunma University Dna methylation editing kit and dna methylation editing method
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US11345931B2 (en) 2015-12-14 2022-05-31 President And Fellows Of Harvard College Cas discrimination using tuned guide RNA
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
MX2018007987A (es) * 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.
JP7012645B2 (ja) 2016-01-11 2022-01-28 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー キメラタンパク質および免疫治療の方法
WO2017123559A2 (en) 2016-01-11 2017-07-20 The Board Of Trustees Of The Leland Stanford Junior University Chimeric proteins and methods of regulating gene expression
WO2017136794A1 (en) 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP4155397B1 (en) 2016-02-10 2024-06-26 The Regents of The University of Michigan Detection of nucleic acids
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
US10538750B2 (en) 2016-02-29 2020-01-21 Agilent Technologies, Inc. Methods and compositions for blocking off-target nucleic acids from cleavage by CRISPR proteins
IL316539B1 (en) 2016-03-15 2026-05-01 Rnaissance Ag Llc Methods and compositions for the enhanced production of double-stranded RNA
JP2019515654A (ja) 2016-03-16 2019-06-13 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 肥満及び／又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
CN106701765A (zh) * 2016-04-11 2017-05-24 广东赤萌医疗科技有限公司 用于hiv感染治疗的多核苷酸及其制备药物应用
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
AU2017257624B2 (en) 2016-04-25 2023-05-25 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
CN107326046A (zh) * 2016-04-28 2017-11-07 上海邦耀生物科技有限公司 一种提高外源基因同源重组效率的方法
SG10202102885UA (en) 2016-05-20 2021-05-28 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas
US20190100732A1 (en) * 2016-06-02 2019-04-04 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
DK3604527T3 (da) 2016-06-02 2021-05-31 Sigma Aldrich Co Llc Anvendelse af programmerbare dna-bindingsproteiner til at forbedre målrettet genommodifikation
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN109312386B (zh) * 2016-06-15 2022-10-25 株式会社图尔金 使用中靶靶标和脱靶靶标的多重靶标系统筛选靶特异性核酸酶的方法及其用途
CN109688820B (zh) 2016-06-24 2023-01-10 科罗拉多州立大学董事会（法人团体） 用于生成条形码化组合文库的方法
US11471462B2 (en) 2016-06-27 2022-10-18 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
CN109477130B (zh) 2016-07-01 2022-08-30 微软技术许可有限责任公司 通过迭代dna编辑的存储
US20230151341A1 (en) * 2016-07-13 2023-05-18 Qihan Chen Method for specifically editing genomic dna and application thereof
WO2018013840A1 (en) 2016-07-13 2018-01-18 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
AU2017302551B2 (en) 2016-07-26 2023-04-27 The General Hospital Corporation Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
KR101961332B1 (ko) * 2016-07-28 2019-03-22 기초과학연구원 Cas9 단백질 및 가이드 RNA를 포함하는 안질환 치료용 약학 조성물
CN109803530A (zh) 2016-07-29 2019-05-24 瑞泽恩制药公司 包含引起c-截短的原纤维蛋白-1表达的突变的小鼠
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
TWI791459B (zh) 2016-08-10 2023-02-11 日商領先基因生技股份有限公司 改變真核細胞基因體的標的部位之方法
JP7050215B2 (ja) * 2016-08-19 2022-04-08 ツールゲン インコーポレイテッド 人工的に操作された血管新生調節系
MX2019002004A (es) * 2016-08-19 2019-10-09 Whitehead Inst Biomedical Res Metodos para editar la metilacion del adn.
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
WO2018039440A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
GB2569252A (en) 2016-08-31 2019-06-12 Harvard College Methods of combining the detection of biomolecules into a single assay using fluorescent in situ sequencing
WO2018048194A1 (ko) * 2016-09-07 2018-03-15 울산대학교 산학협력단 dCas9 단백질 및 표적 핵산 서열에 결합하는 gRNA를 이용한 핵산 검출의 민감도 및 특이도 향상용 조성물 및 방법
WO2018049075A1 (en) 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
CN107880132B (zh) * 2016-09-30 2022-06-17 北京大学 一种融合蛋白及使用其进行同源重组的方法
CN110199015A (zh) * 2016-09-30 2019-09-03 波赛达治疗公司 修饰干细胞记忆t细胞、其制造方法与使用方法
JP2019532644A (ja) 2016-09-30 2019-11-14 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｒｎａ誘導型核酸修飾酵素及びその使用方法
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
WO2018068053A2 (en) 2016-10-07 2018-04-12 Integrated Dna Technologies, Inc. S. pyogenes cas9 mutant genes and polypeptides encoded by same
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
KR102662249B1 (ko) 2016-10-14 2024-05-03 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
EP4338799A3 (en) 2016-10-18 2024-06-05 Regents of the University of Minnesota Tumor infiltrating lymphocytes and methods of therapy
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
US20180282722A1 (en) * 2016-11-21 2018-10-04 Massachusetts Institute Of Technology Chimeric DNA:RNA Guide for High Accuracy Cas9 Genome Editing
CA3044101A1 (en) 2016-11-22 2018-05-31 Integrated Dna Technologies, Inc. Crispr/cpf1 systems and methods
EP3551757A1 (en) 2016-12-08 2019-10-16 Intellia Therapeutics, Inc. Modified guide rnas
WO2018111944A1 (en) 2016-12-12 2018-06-21 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
RU2019121801A (ru) 2016-12-14 2021-01-15 Лигандал, Инк. Композиции и способы для доставки полезной нагрузки в виде нуклеиновой кислоты и/или белка
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
JP7383478B2 (ja) * 2016-12-22 2023-11-20 インテリア セラピューティクス，インコーポレイテッド アルファ１アンチトリプシン欠乏症を治療するための組成物および方法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2017388379A1 (en) * 2016-12-28 2019-06-06 Ionis Pharmaceuticals, Inc. Modified CRISPR RNA and uses thereof
EP3565608A4 (en) 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES
WO2018129368A2 (en) 2017-01-06 2018-07-12 Editas Medicine, Inc. Methods of assessing nuclease cleavage
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
CN110234770A (zh) * 2017-01-17 2019-09-13 基础科学研究院 通过dna单链断裂识别碱基编辑脱靶位点的方法
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
CN110312797A (zh) 2017-02-10 2019-10-08 齐默尔根公司 组装和编辑用于多个宿主的多个dna构建体的模块化通用质粒设计策略
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
EP3592768B1 (en) 2017-03-08 2024-12-25 The Regents of The University of Michigan Analyte detection
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
CN108660161B (zh) * 2017-03-31 2023-05-09 中国科学院脑科学与智能技术卓越创新中心 基于CRISPR/Cas9技术的制备无嵌合基因敲除动物的方法
US11867661B2 (en) 2017-04-07 2024-01-09 Sage Science, Inc. Systems and methods for detection of genetic structural variation using integrated electrophoretic DNA purification
EP3609521A4 (en) 2017-04-14 2021-06-16 University of Massachusetts TARGETING OF CELL TROPISM RECEPTORS TO INHIBIT INFECTION BY THE CYTOMEGALOVIRUS
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
EP3612204A4 (en) 2017-04-21 2021-01-27 The General Hospital Corporation INDUCTIBLE, TUNING AND MULTIPLEX HUMAN GENE REGULATION USING CRISPR-CPF1
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
CA3063733A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
DK3635102T3 (da) 2017-06-05 2025-10-27 Regeneron Pharma B4galt1-varianter og anvendelser deraf
CN110997908A (zh) 2017-06-09 2020-04-10 爱迪塔斯医药公司 工程化的cas9核酸酶
KR102634727B1 (ko) 2017-06-14 2024-02-07 위스콘신 얼럼나이 리서어치 화운데이션 변형된 가이드 rna, crispr-리보뉴클레오프로테인 복합체 및 사용 방법
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
CN111511375A (zh) 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
KR102655021B1 (ko) * 2017-07-11 2024-04-04 시그마-알드리치 컴퍼니., 엘엘씨 표적된 게놈 변형을 개선하기 위한 뉴클레오솜 상호작용 단백질 도메인 사용
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
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
CA3065579A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
KR102712142B1 (ko) 2017-07-31 2024-10-07 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
CN111182790A (zh) 2017-07-31 2020-05-19 瑞泽恩制药公司 Crispr报告体非人类动物及其用途
CN111278848B (zh) 2017-08-04 2023-06-27 北京大学 特异性识别甲基化修饰dna碱基的tale rvd及其应用
US11624077B2 (en) 2017-08-08 2023-04-11 Peking University Gene knockout method
WO2019040650A1 (en) * 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019067875A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
WO2019075197A1 (en) 2017-10-11 2019-04-18 The General Hospital Corporation METHODS OF DETECTION OF INDIVIDUAL SITE-SPECIFIC PARASITE GENOMIC DEAMINATION BY BASE EDITING TECHNOLOGIES
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN107602707B (zh) * 2017-10-17 2021-04-23 湖北大学 一种特异性调节枯草芽孢杆菌外源基因表达的dcas9-ω融合蛋白及其应用
SG11202003798TA (en) 2017-10-27 2020-05-28 Univ California Targeted replacement of endogenous t cell receptors
CA3081456A1 (en) * 2017-11-01 2019-05-09 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy
EP3704245A1 (en) 2017-11-01 2020-09-09 Novartis AG Synthetic rnas and methods of use
JP7423520B2 (ja) * 2017-11-16 2024-01-29 アストラゼネカ・アクチエボラーグ Ｃａｓ９ベースノックイン方針の効力を改善するための組成物及び方法
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11293019B2 (en) 2017-12-22 2022-04-05 Gflas Life Sciences, Inc. Chimeric genome engineering molecules and methods
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
WO2019161340A1 (en) 2018-02-19 2019-08-22 Yale University Phosphopeptide-encoding oligonucleotide libraries and methods for detecting phosphorylation-dependent molecular interactions
KR20200124702A (ko) 2018-02-23 2020-11-03 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 신규한 cas9 오르소로그
IL277234B2 (en) 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
EP3772928B1 (en) 2018-04-06 2025-02-26 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
AU2019256287B2 (en) 2018-04-17 2026-02-05 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
AU2019255789B2 (en) 2018-04-19 2025-11-20 The Regents Of The University Of California Compositions and methods for gene editing
WO2019213430A1 (en) * 2018-05-03 2019-11-07 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for nicking target dna sequences
CN108588123A (zh) * 2018-05-07 2018-09-28 南京医科大学 CRISPR/Cas9载体组合在制备基因敲除猪的血液制品中的应用
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
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
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3575396A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
CN110592141B (zh) 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
JP2021530212A (ja) 2018-07-13 2021-11-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California レトロトランスポゾンベースの送達媒体及びその使用方法
KR20210044795A (ko) 2018-08-15 2021-04-23 지머젠 인코포레이티드 고 처리량 대사 공학에서 CRISPRi의 응용
EP3841205A4 (en) 2018-08-22 2022-08-17 The Regents of The University of California VARIANT TYPE V CRISPR/CAS EFFECTOR POLYPEPTIDES AND METHODS OF USE THEREOF
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的な塩基エディター
EP3847251A1 (en) 2018-09-07 2021-07-14 Astrazeneca AB Compositions and methods for improved nucleases
WO2020072250A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for genome modification and alteration of expression
US12203123B2 (en) 2018-10-01 2025-01-21 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
US10711267B2 (en) 2018-10-01 2020-07-14 North Carolina State University Recombinant type I CRISPR-Cas system
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases
WO2020097445A1 (en) 2018-11-09 2020-05-14 Inari Agriculture, Inc. Rna-guided nucleases and dna binding proteins
CN113166744B (zh) 2018-12-14 2025-02-07 先锋国际良种公司 用于基因组编辑的新颖crispr-cas系统
WO2020131632A1 (en) 2018-12-20 2020-06-25 Regeneron Pharmaceuticals, Inc. Nuclease-mediated repeat expansion
WO2020148206A1 (en) 2019-01-14 2020-07-23 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and kits for generating and selecting a variant of a binding protein with increased binding affinity and/or specificity
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020159880A1 (en) 2019-01-28 2020-08-06 The Administrators Of The Tulane Educational Fund Crispr-based methods and novel compositions for treating vascular disorders
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
US12460230B2 (en) 2019-02-10 2025-11-04 The J. David Gladstone Institutes Modified mitochondrion and methods of use thereof
EP4219700A1 (en) 2019-03-07 2023-08-02 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
JP7389135B2 (ja) 2019-03-18 2023-11-29 リジェネロン・ファーマシューティカルズ・インコーポレイテッド タウ凝集に関連する遺伝的脆弱性を明らかにするためのｃｒｉｓｐｒ／ｃａｓドロップアウトスクリーニングプラットフォーム
EP4317950B1 (en) 2019-03-18 2026-01-28 Regeneron Pharmaceuticals, Inc. Crispr/cas screening platform to identify genetic modifiers of tau seeding or aggregation
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
EP3945800A1 (en) 2019-04-03 2022-02-09 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
JP7506686B2 (ja) 2019-04-04 2024-06-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヒト化凝固因子１２遺伝子座を含む非ヒト動物
AU2020253531C1 (en) 2019-04-04 2022-06-16 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
EP3953470A1 (en) 2019-04-12 2022-02-16 Astrazeneca AB Compositions and methods for improved gene editing
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
KR20220024053A (ko) 2019-06-14 2022-03-03 리제너론 파마슈티칼스 인코포레이티드 타우병증의 모델
EP4010361A4 (en) 2019-08-06 2023-08-09 Children's Medical Center Corporation METHODS AND COMPOSITIONS FOR RESTORING MICROGLIA
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
CN114616002A (zh) 2019-09-13 2022-06-10 瑞泽恩制药公司 使用由脂质纳米颗粒递送的crispr/cas系统在动物中进行的转录调控
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
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
DK3812472T3 (da) 2019-10-21 2023-02-20 Univ Freiburg Albert Ludwigs Virkelig uvildig in vitro-undersøgelse til profilering af off-target-aktivitet af en eller flere målspecifikke programmerbare nukleaser i celler (abnoba-seq)
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
US11331333B2 (en) 2019-11-08 2022-05-17 Georg-August-Universität Göttingen Stiftung Öffentichen Rechts, Universitätsmadizin Treatment of aberrant fibroblast proliferation
US20230042198A1 (en) * 2019-11-25 2023-02-09 La Jolla Institute For Immunology Methods and Compositions for Modulationg Heterochromatin Dysfunction, Genomic Instability, and Associate Conditions
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
AU2020401206A1 (en) 2019-12-11 2022-07-14 Intellia Therapeutics, Inc. Modified guide RNAs for gene editing
CN111088357B (zh) * 2019-12-31 2022-09-20 深圳大学 针对escc的肿瘤标志物及其应用
EP4093864A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
WO2021155063A1 (en) 2020-01-29 2021-08-05 Readcoor, Llc Compositions and methods for analyte detection
US12129496B2 (en) 2020-02-12 2024-10-29 Massachusetts Eye And Ear Infirmary Haplotype-based treatment of RP1 associated retinal degenerations
JP7837276B2 (ja) 2020-03-04 2026-03-30 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 免疫療法に対する腫瘍細胞の感作のための方法及び組成物
EP4114952A4 (en) * 2020-03-05 2024-05-08 Board of Regents of the University of Nebraska CRISPR/CAS9 SYSTEM FOR MULTI-STRAINS HIV-1 TREATMENT
WO2021183720A1 (en) * 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
CN115427566A (zh) * 2020-04-08 2022-12-02 阿斯利康(瑞典)有限公司 用于改善的位点特异性修饰的组合物和方法
US12115230B2 (en) * 2020-04-09 2024-10-15 Verve Therapeutics, Inc. Base editing of ANGPTL3 and methods of using same for treatment of disease
CA3182286A1 (en) * 2020-05-04 2021-11-11 Editas Medicine, Inc. Selection by essential-gene knock-in
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230279438A1 (en) 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
WO2021246165A1 (ja) * 2020-06-03 2021-12-09 国立大学法人広島大学 Ｏａｓｉｓ遺伝子の脱メチル化のための核酸及びそれを用いた脱メチル化方法
JP2023529844A (ja) * 2020-06-05 2023-07-12 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア エピゲノム編集のための組成物及び方法
US12630846B2 (en) 2020-06-05 2026-05-19 The Regents Of The University Of Michigan Type I-C CRISPR system from Neisseria lactamica and methods of use
EP4179079A1 (en) 2020-07-10 2023-05-17 Horizon Discovery Limited Method for producing genetically modified cells
US20230293726A1 (en) 2020-08-11 2023-09-21 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Method for the treatment of wwox associated diseases
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
CN116391040A (zh) 2020-10-13 2023-07-04 法国国家科学研究中心(Cnrs) 结合接合和Crispr/Cas系统的靶向抗菌质粒及其用途
RU2762831C1 (ru) * 2020-10-26 2021-12-23 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) Молекула рнк-проводника для геномного редактирования протомоторной области гена vrn-a1 однодольных зерновых с применением системы crispr/cas9
CN112430622A (zh) * 2020-10-26 2021-03-02 扬州大学 一种FokI和dCpf1融合蛋白表达载体及其介导的定点基因编辑方法
US20240002839A1 (en) 2020-12-02 2024-01-04 Decibel Therapeutics, Inc. Crispr sam biosensor cell lines and methods of use thereof
KR20220082186A (ko) 2020-12-10 2022-06-17 한세준 유,무선 충전이 가능한 보조배터리형 uv-led살균기
CA3207144A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Limited Method for producing genetically modified cells
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022180153A1 (en) 2021-02-25 2022-09-01 INSERM (Institut National de la Santé et de la Recherche Médicale) Allele-specific genome editing of the nr2e3 mutation g56r
KR102882704B1 (ko) * 2021-03-03 2025-11-12 중앙대학교 산학협력단 CRISPR/Cas9 시스템을 이용한 유전체 단일염기 편집 방법 및 이의 용도
CN113846019B (zh) * 2021-03-05 2023-08-01 海南师范大学 一种海洋微拟球藻靶向表观基因组遗传调控方法
EP4095243A1 (en) 2021-05-25 2022-11-30 European Molecular Biology Laboratory System for hybridization-based precision genome cleavage and editing, and uses thereof
WO2022248645A1 (en) 2021-05-27 2022-12-01 Astrazeneca Ab Cas9 effector proteins with enhanced stability
TW202317764A (zh) 2021-06-10 2023-05-01 美商英特利亞醫療公司 用於基因編輯之包含內部連接子之經修飾引導rna
WO2023000081A1 (en) * 2021-07-14 2023-01-26 The Governing Council Of The University Of Toronto Trans-activators and methods and use thereof
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CA3229450A1 (en) 2021-08-20 2023-02-23 Wisconsin Alumni Research Foundation Nonviral generation of genome edited chimeric antigen receptor t cells
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
EP4596696A3 (en) 2021-09-28 2025-08-27 Acrigen Biosciences Compositions and methods for nucleic acid modifications
EP4409000A1 (en) 2021-09-28 2024-08-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
JP2024536135A (ja) 2021-09-30 2024-10-04 アストラゼネカ・アクチエボラーグ ＣＲＩＳＰＲ／Ｃａｓ挿入の効率を増大させるための阻害剤の使用
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados y sistemas de compuerta lógica.
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
EP4423271A2 (en) 2021-10-28 2024-09-04 Regeneron Pharmaceuticals, Inc. Crispr/cas-related methods and compositions for knocking out c5
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
EP4426822A2 (en) 2021-11-03 2024-09-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
EP4463549A2 (en) 2022-01-14 2024-11-20 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene repression
US20250134999A1 (en) 2022-01-14 2025-05-01 Tune Therapeutics, Inc. Compositions, systems, and methods for programming t cell phenotypes through targeted gene activation
WO2023141487A1 (en) * 2022-01-20 2023-07-27 Inari Agriculture Technology, Inc. Improved soybean explant preparation and transformation
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023144104A1 (en) 2022-01-25 2023-08-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of βeta-thalassemia
IL314482A (en) 2022-02-02 2024-09-01 Regeneron Pharma Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
US20250152677A1 (en) 2022-02-14 2025-05-15 Institut National de la Santé et de la Recherche Médicale Treatment of liver cancers by disrupting the beta-catenin/tcf-4 binding site located upstreatm of meg3 in the dlk1/dio3 locus
WO2023212677A2 (en) 2022-04-29 2023-11-02 Regeneron Pharmaceuticals, Inc. Identification of tissue-specific extragenic safe harbors for gene therapy approaches
AU2023269134A1 (en) 2022-05-09 2024-12-12 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023217888A1 (en) 2022-05-10 2023-11-16 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from βeta-thalassemia
WO2023235726A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr interference therapeutics for c9orf72 repeat expansion disease
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
JP2025525569A (ja) 2022-07-18 2025-08-05 レナゲード セラピューティクス マネージメント インコーポレイテッド 遺伝子編集成分、システム、及び使用方法
WO2024018056A1 (en) 2022-07-22 2024-01-25 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from βeta-thalassemia
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
WO2024040254A2 (en) 2022-08-19 2024-02-22 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
AU2023329392A1 (en) 2022-08-25 2025-02-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
JP2025531268A (ja) 2022-09-19 2025-09-19 チューン セラピューティクス インコーポレイテッド Ｔ細胞機能を調節するための組成物、システム、および方法
KR20250075694A (ko) 2022-09-28 2025-05-28 리제너론 파마슈티칼스 인코포레이티드 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체
WO2024084025A1 (en) 2022-10-21 2024-04-25 Keygene N.V. Rna transfection in plant cells with modified rna
AU2023371614A1 (en) 2022-11-04 2025-05-15 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
CN115820603B (zh) * 2022-11-15 2024-07-05 吉林大学 一种基于dCasRx-NSUN6单基因特异性M5C修饰编辑方法
WO2024121354A1 (en) 2022-12-08 2024-06-13 Keygene N.V. Duplex sequencing with covalently closed dna ends
KR20250128349A (ko) * 2022-12-23 2025-08-27 에피제닉 테라퓨틱스 피티이 리미티드 융합체 및 이의 용도
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024165484A1 (en) 2023-02-06 2024-08-15 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through multiplex base editing
CN116376975B (zh) * 2023-02-27 2024-05-14 中国科学院脑科学与智能技术卓越创新中心 激活异染色质基因的方法及应用
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
CN118684781A (zh) * 2023-03-21 2024-09-24 深圳赫兹生命科学技术有限公司 GnRH-VLP重组去势疫苗及其制备方法
CN121127605A (zh) 2023-03-29 2025-12-12 阿斯利康(瑞典)有限公司 使用抑制剂来提高CRISPR/Cas插入的效率
WO2024209000A1 (en) 2023-04-04 2024-10-10 Keygene N.V. Linkers for duplex sequencing
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
US20250002946A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods And Compositions For Increasing Homology-Directed Repair
WO2025017030A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
EP4747377A1 (en) 2023-07-17 2026-05-27 Institut National de la Santé et de la Recherche Médicale Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법
AU2024315073A1 (en) 2023-07-28 2026-01-22 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
AR133384A1 (es) 2023-07-28 2025-09-24 Regeneron Pharma Anti-tfr:esfingomielinasa ácida para el tratamiento de la deficiencia de esfingomielinasa ácida
IL326018A (en) 2023-07-28 2026-03-01 Regeneron Pharma Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease
CN121909284A (zh) 2023-07-31 2026-04-21 图恩疗法股份有限公司 用于调节il-2基因表达的组合物和方法
EP4754248A1 (en) 2023-07-31 2026-06-10 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038648A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
EP4771049A1 (en) 2023-08-28 2026-07-08 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025202473A1 (en) 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2025261475A1 (zh) * 2024-06-21 2025-12-26 益杰立科(上海)生物科技有限公司 靶向血管内皮生长因子的工程化基因转录阻抑工具及用途
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026077659A1 (en) 2024-09-17 2026-04-16 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through epigenome editing
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence
WO2026080515A2 (en) 2024-10-08 2026-04-16 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cells and cell lines and methods of use thereof
WO2026083309A1 (en) 2024-10-17 2026-04-23 Astrazeneca Ab Use of inhibitors or modulators of artemis in gene editing
WO2026102058A1 (en) 2024-11-06 2026-05-15 Tune Therapeutics, Inc. Methods for treating chronic hepatitis b infection and related formulations
WO2026104502A1 (en) 2024-11-13 2026-05-21 Institut National de la Santé et de la Recherche Médicale Epigenetic reactivation of gamma-globin expression as a novel curative option for β-hemoglobinopathies
WO2026161895A1 (en) 2025-01-27 2026-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for epigenetic regulation of lipoprotein a (lpa) gene expression
WO2026163162A1 (en) 2025-01-31 2026-08-06 Intellia Therapeutics, Inc. Enhanced cas9 variants and uses thereof
CN121554544B (zh) * 2026-01-23 2026-04-28 成都医学院 一种NFT蛋白及其制备方法和应用，以及一种NusA或NusA融合蛋白的纯化方法

Family Cites Families (166)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo Improvements in or relating to binding proteins for recognition of dna
US20030017149A1 (en) 1996-10-10 2003-01-23 Hoeffler James P. Single chain monoclonal antibody fusion reagents that regulate transcription in vivo
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
AU2001257331A1 (en) 2000-04-28 2001-11-12 Sangamo Biosciences, Inc. Methods for designing exogenous regulatory molecules
EP1276859B1 (en) 2000-04-28 2007-02-07 Sangamo Biosciences Inc. Targeted modification of chromatin structure
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
AU2003219847A1 (en) 2002-02-21 2003-09-09 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
EP1532178A4 (en) * 2002-06-11 2006-10-25 Scripps Research Inst Artificial transcription factors
AU2003304086A1 (en) 2002-10-23 2004-11-26 Massachussetts Institute Of Technlogy Context sensitive parallel optimization of zinc finger dna binding domains
US7021555B2 (en) 2004-01-06 2006-04-04 Zoo Med Laboratories, Inc. Spraying/misting for plants and animals
US7919277B2 (en) 2004-04-28 2011-04-05 Danisco A/S Detection and typing of bacterial strains
AU2006214064B2 (en) 2005-02-18 2012-04-26 J. Craig Venter Institute, Inc. Proteins and nucleic acids from meningitis/sepsis-associated escherichia coli
US20100055793A1 (en) * 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
EP1913149A4 (en) 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
EP2325332B1 (en) 2005-08-26 2012-10-31 DuPont Nutrition Biosciences ApS Use of CRISPR associated genes (CAS)
CA2631422A1 (en) 2005-11-28 2007-05-31 The Scripps Research Institute Zinc finger binding domains for tnn
ES2373586T3 (es) 2006-05-19 2012-02-06 Danisco A/S Microorganismos marcados y métodos para marcar.
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
PL2034848T3 (pl) 2006-06-16 2017-08-31 Dupont Nutrition Bioscences Aps Bakteria Streptococcus thermophilus
US9201063B2 (en) 2006-11-16 2015-12-01 General Electric Company Sequential analysis of biological samples
WO2008093152A1 (en) * 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
MX2009009071A (es) 2007-03-02 2009-09-24 Danisco Cultivos con resistencia mejorada a fagos.
WO2008118394A1 (en) 2007-03-23 2008-10-02 New York University Methods of affecting nitrogen assimilation in plants
CA2678055C (en) 2007-04-10 2016-02-16 Qiagen Gmbh Rna interference tags
WO2008151032A2 (en) 2007-05-31 2008-12-11 Washington University In St. Louis Arrays and methods comprising m. smithii gene products
CN107129524B (zh) 2007-09-25 2023-11-21 田园温室气体研究有限公司 用于抑制微生物细胞的疫苗和疫苗组分
FR2925918A1 (fr) 2007-12-28 2009-07-03 Pasteur Institut Typage et sous-typage moleculaire de salmonella par identification des sequences nucleotidiques variables des loci crispr
FR2930264B1 (fr) 2008-04-18 2013-02-22 Gervais Danone Sa Nouvelle souche de lactobacillus paracasei subsp. paracasei dotee de proprietes antimicrobiennes et immunomodulatrices.
JP2010017178A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010017179A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
US8546553B2 (en) 2008-07-25 2013-10-01 University Of Georgia Research Foundation, Inc. Prokaryotic RNAi-like system and methods of use
JP2010048566A (ja) 2008-08-19 2010-03-04 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010068800A (ja) 2008-08-19 2010-04-02 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
WO2010037001A2 (en) * 2008-09-26 2010-04-01 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins
US20110201007A1 (en) 2008-10-21 2011-08-18 Animal Health Trust Diagnostic test for streptococcus equi
US20110294873A1 (en) 2008-10-23 2011-12-01 Université de Lausanne Gene Transfer Vectors Comprising At Least One Isolated DNA Molecule Having Insulator Or Boundary Properties And Methods To Identify The Same
WO2010054108A2 (en) 2008-11-06 2010-05-14 University Of Georgia Research Foundation, Inc. Cas6 polypeptides and methods of use
DK2362915T3 (en) * 2008-11-07 2017-03-27 Dupont Nutrition Biosci Aps CRISPR SEQUENCES OF BIFIDOBACTERIA
AU2009315298A1 (en) 2008-11-11 2010-05-20 Alimentary Health Limited Bifidobacterium longum
GB2466177A (en) 2008-12-03 2010-06-16 Arab Science & Technology Found Bacteriophage selection and breeding
EP2367938B1 (en) 2008-12-12 2014-06-11 DuPont Nutrition Biosciences ApS Genetic cluster of strains of streptococcus thermophilus having unique rheological properties for dairy fermentation
KR20100093626A (ko) 2009-02-17 2010-08-26 서강대학교산학협력단 슈도모나스 애루지노사에 대한 파아지 치료
EP2414524B1 (en) 2009-04-03 2017-08-23 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
US8501405B2 (en) 2009-04-27 2013-08-06 Pacific Biosciences Of California, Inc. Real-time sequencing methods and systems
US8609421B2 (en) 2009-06-12 2013-12-17 Pacific Biosciences Of California, Inc. Single-molecule real-time analysis of protein synthesis
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
PH12012500517A1 (en) 2009-09-25 2012-11-12 Basf Plant Science Co Gmbh Plants having enhanced yield-related traits and a method for making the same
US9677125B2 (en) 2009-10-21 2017-06-13 General Electric Company Detection of plurality of targets in biological samples
US20110269119A1 (en) 2009-10-30 2011-11-03 Synthetic Genomics, Inc. Encoding text into nucleic acid sequences
JP6137596B2 (ja) 2010-02-08 2017-05-31 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作された切断ハーフドメイン
WO2011101696A1 (en) * 2010-02-18 2011-08-25 Cellectis Improved meganuclease recombination system
US20120027786A1 (en) 2010-02-23 2012-02-02 Massachusetts Institute Of Technology Genetically programmable pathogen sense and destroy
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
WO2011112781A2 (en) 2010-03-12 2011-09-15 Brookhaven Science Associates/Brookhaven National Laboratory Enterobacter sp. 638 and methods of use thereof
CA2798703A1 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
WO2011159369A1 (en) 2010-06-14 2011-12-22 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
WO2012047726A1 (en) 2010-09-29 2012-04-12 The Broad Institute, Inc. Methods for chromatin immuno-precipitations
DK2630156T3 (en) 2010-10-20 2018-12-17 Dupont Nutrition Biosci Aps CRISPR-CAS SEQUENCES OF LACTOCOCCUS
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
WO2012093833A2 (en) * 2011-01-03 2012-07-12 Toolgen Incorporation Genome engineering via designed tal effector nucleases
US20120214160A1 (en) 2011-01-14 2012-08-23 Life Technologies Corporation Methods, compositions, and kits for detecting rare cells
US20140201857A1 (en) 2013-01-14 2014-07-17 Recombinetics, Inc. Hornless livestock
US20140113376A1 (en) 2011-06-01 2014-04-24 Rotem Sorek Compositions and methods for downregulating prokaryotic genes
DK2543255T4 (da) 2011-07-04 2023-03-20 Dsm Ip Assets Bv Antilisteriel blandet kultur og fremgangsmåde til fremstilling af ost
CA2841710C (en) * 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
AU2013256240B2 (en) 2012-05-02 2018-09-20 Corteva Agriscience Llc Targeted modification of malate dehydrogenase
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
US11120889B2 (en) 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
EP2674501A1 (en) 2012-06-14 2013-12-18 Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail Method for detecting and identifying enterohemorrhagic Escherichia coli
US10025647B2 (en) * 2012-06-30 2018-07-17 Intel Corporation Memory poisoning with hints
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
CN116622704A (zh) 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
BR112015004995B1 (pt) 2012-09-07 2023-05-02 Sangamo Biosciences, Inc. Método para modificação do genoma de uma célula, uso de uma célula, semente ou planta obtida pelo referido método e nuclease de dedo de zinco sítio específica
SG11201502693SA (en) 2012-10-09 2015-05-28 Liposcience Inc Nmr quantification of branched chain amino acids
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
US20150315576A1 (en) 2012-11-01 2015-11-05 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
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
EP3064585B1 (en) 2012-12-12 2020-02-05 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201707569YA (en) * 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
KR102955556B1 (ko) 2012-12-12 2026-04-22 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
WO2014093709A1 (en) 2012-12-12 2014-06-19 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
CA2893579A1 (en) 2012-12-13 2014-06-19 Dow Agrosciences Llc Dna detection methods for site specific nuclease activity
IL308158A (en) * 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
US20140173780A1 (en) * 2012-12-19 2014-06-19 Dow Agrosciences Llc Soybean transformation for efficient and high-throughput transgenic event production
CN103233028B (zh) 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
US20140212869A1 (en) 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
EP2954042B1 (en) 2013-02-07 2017-12-06 The General Hospital Corporation Tale transcriptional activators
WO2014124226A1 (en) 2013-02-07 2014-08-14 The Rockefeller University Sequence specific antimicrobials
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014131833A1 (en) 2013-02-27 2014-09-04 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Gene editing in the oocyte by cas9 nucleases
CA2901545C (en) * 2013-03-08 2019-10-08 Oxford Nanopore Technologies Limited Use of spacer elements in a nucleic acid to control movement of a helicase
WO2014143381A1 (en) 2013-03-09 2014-09-18 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple crispr/cas selections of recombineering events
ES2749624T3 (es) 2013-03-14 2020-03-23 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
JP2016512048A (ja) 2013-03-15 2016-04-25 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ ＣＲＩＳＰＲ／Ｃａｓシステムを使用した植物ゲノム操作
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
CN105263312A (zh) 2013-04-05 2016-01-20 美国陶氏益农公司 用于在植物基因组内整合外源序列的方法和组合物
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
CN103224947B (zh) 2013-04-28 2015-06-10 陕西师范大学 一种基因打靶系统
US10604771B2 (en) 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
US20150067922A1 (en) 2013-05-30 2015-03-05 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
EP3022304B1 (en) 2013-07-19 2018-12-26 Larix Biosciences LLC Methods and compositions for producing double allele knock outs
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
AU2014312123A1 (en) 2013-08-29 2016-03-17 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
KR20160048212A (ko) 2013-09-04 2016-05-03 씨에스아이알 유전자 발현을 침묵화시키기 위해 유전자 조절 인자들을 표적으로하는 부위-특이적인 뉴클레아제 단일 세포 분석법
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
KR20160102056A (ko) 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 멀티플렉스 가이드 ｒｎａ
JP2017511694A (ja) 2014-02-12 2017-04-27 トーマス・ジェファーソン・ユニバーシティ マイクロｒｎａ阻害剤を使用するための組成物および方法
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
EP3117004A4 (en) 2014-03-14 2017-12-06 University of Washington Genomic insulator elements and uses thereof
EP3126498A4 (en) 2014-03-20 2017-08-23 Université Laval Crispr-based methods and products for increasing frataxin levels and uses thereof
WO2015153940A1 (en) 2014-04-03 2015-10-08 Massachusetts Institute Of Technology Methods and compositions for the production of guide rna
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
MA41349A (fr) 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
AU2016211517B2 (en) 2015-01-28 2021-05-13 Caribou Biosciences, Inc. CRISPR hybrid DNA/RNA polynucleotides and methods of use
US20190388469A1 (en) 2015-01-30 2019-12-26 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
EP3302575A4 (en) 2015-05-28 2019-01-16 Coda Biotherapeutics GEMONEDITIERUNGSVEKTOREN
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016308339A1 (en) 2015-08-18 2018-04-12 Baylor College Of Medicine Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
KR102662249B1 (ko) 2016-10-14 2024-05-03 더 제너럴 하스피탈 코포레이션 후성적으로 조절되는 부위-특이적 뉴클레아제
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
EP3612204A4 (en) 2017-04-21 2021-01-27 The General Hospital Corporation INDUCTIBLE, TUNING AND MULTIPLEX HUMAN GENE REGULATION USING CRISPR-CPF1
CA3100726A1 (en) 2018-05-17 2019-11-21 The General Hospital Corporation Ccctc-binding factor variants
US20230036273A1 (en) 2019-11-27 2023-02-02 The General Hospital Corporation System and method for activating gene expression
EP4176434A4 (en) 2020-05-29 2024-09-11 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)

Also Published As

Publication number Publication date
AU2021203370A1 (en) 2021-06-24
CN105247066A (zh) 2016-01-13
AU2022209254B2 (en) 2025-01-23
EP3467125B1 (en) 2023-08-30
CA2907198A1 (en) 2014-09-18
KR102602047B1 (ko) 2023-11-15
WO2014144761A2 (en) 2014-09-18
KR20210013304A (ko) 2021-02-03
EP4428141A2 (en) 2024-09-11
EP2971125A2 (en) 2016-01-20
JP2016512691A (ja) 2016-05-09
JP2016517276A (ja) 2016-06-16
CN110540991B (zh) 2023-10-24
JP2023061983A (ja) 2023-05-02
US10119133B2 (en) 2018-11-06
US11634731B2 (en) 2023-04-25
US20160024523A1 (en) 2016-01-28
WO2014152432A3 (en) 2015-10-29
AU2020201465A1 (en) 2020-03-19
KR20210013302A (ko) 2021-02-03
US11168338B2 (en) 2021-11-09
CN113563476A (zh) 2021-10-29
IL289396B1 (en) 2023-08-01
JP6878554B2 (ja) 2021-05-26
JP2021192624A (ja) 2021-12-23
EP2971041A1 (en) 2016-01-20
IL289396A (en) 2022-02-01
EP3467125A1 (en) 2019-04-10
JP7053706B2 (ja) 2022-04-12
KR20230157540A (ko) 2023-11-16
JP2021118726A (ja) 2021-08-12
JP6980380B2 (ja) 2021-12-15
CN105408497A (zh) 2016-03-16
US10844403B2 (en) 2020-11-24
JP7379572B2 (ja) 2023-11-14
AU2019204675A1 (en) 2019-07-18
EP4428141A3 (en) 2024-12-18
US20140295557A1 (en) 2014-10-02
AU2021203370B2 (en) 2023-07-27
CN113563476B (zh) 2026-06-19
AU2017204909A1 (en) 2017-08-31
AU2020207840B2 (en) 2022-07-21
KR20150132395A (ko) 2015-11-25
CN105408483A (zh) 2016-03-16
US20200224222A1 (en) 2020-07-16
AU2020207840A1 (en) 2020-08-13
KR102210322B1 (ko) 2021-02-01
JP2022106710A (ja) 2022-07-20
CN105247066B (zh) 2020-10-20
BR112015023489B1 (pt) 2022-06-07
ZA201506814B (en) 2018-11-28
EP2970986A4 (en) 2016-09-07
JP2020120661A (ja) 2020-08-13
CN110540991A (zh) 2019-12-06
EP2970986B1 (en) 2020-05-06
AU2014227653B2 (en) 2017-04-20
US20200071730A1 (en) 2020-03-05
AU2022252788A1 (en) 2022-11-03
US20160010076A1 (en) 2016-01-14
AU2014228981B2 (en) 2019-11-28
KR102210323B1 (ko) 2021-02-01
US11920152B2 (en) 2024-03-05
AU2014239665A1 (en) 2015-10-01
US20170327805A1 (en) 2017-11-16
US9567604B2 (en) 2017-02-14
KR102405549B1 (ko) 2022-06-08
BR112015023489A2 (pt) 2017-10-10
US20180340189A1 (en) 2018-11-29
AU2014228981A1 (en) 2015-10-01
IL241671B (en) 2022-03-01
US20180208921A1 (en) 2018-07-26
CA3161835A1 (en) 2014-09-25
KR102874079B1 (ko) 2025-10-22
US20170152508A1 (en) 2017-06-01
EP3744842A1 (en) 2020-12-02
KR102210319B1 (ko) 2021-02-01
EP2970208A2 (en) 2016-01-20
JP2024012446A (ja) 2024-01-30
JP6622183B2 (ja) 2019-12-18
CA2907198C (en) 2019-12-10
EP2971125B2 (en) 2023-11-22
JP2020039350A (ja) 2020-03-19
ES2713503T3 (es) 2019-05-22
US20160024524A1 (en) 2016-01-28
JP2016512264A (ja) 2016-04-25
AU2020201465B2 (en) 2022-05-12
KR20150131250A (ko) 2015-11-24
EP2971125B1 (en) 2020-05-06
EP3741868A1 (en) 2020-11-25
EP2971041B1 (en) 2018-11-28
EP3988667A1 (en) 2022-04-27
US12065668B2 (en) 2024-08-20
WO2014152432A2 (en) 2014-09-25
JP6960438B2 (ja) 2021-11-05
WO2014144288A1 (en) 2014-09-18
IL289396B2 (en) 2023-12-01
US9885033B2 (en) 2018-02-06
EP3865586A1 (en) 2021-08-18
CN112301024A (zh) 2021-02-02
KR20210013303A (ko) 2021-02-03
AU2014239665B2 (en) 2020-04-30
US20140295556A1 (en) 2014-10-02
US9567603B2 (en) 2017-02-14
US10378027B2 (en) 2019-08-13
US11098326B2 (en) 2021-08-24
AU2019204675B2 (en) 2021-03-11
KR20220080012A (ko) 2022-06-14
AU2022252788B2 (en) 2025-02-20
US20190376090A1 (en) 2019-12-12
EP2970986A2 (en) 2016-01-20
CA2906724A1 (en) 2014-09-18
US10415059B2 (en) 2019-09-17
AU2022209254A1 (en) 2022-08-18
JP7126588B2 (ja) 2022-08-26
WO2014144592A3 (en) 2014-12-31
EP2970208A4 (en) 2016-11-30
CN105408497B (zh) 2019-09-13
AU2014227653A2 (en) 2015-11-12
WO2014144761A3 (en) 2015-10-29
US20220090145A1 (en) 2022-03-24
CA2906553A1 (en) 2014-09-25
EP2971041A4 (en) 2016-09-07
WO2014144592A2 (en) 2014-09-18
US10544433B2 (en) 2020-01-28
AU2017204909B2 (en) 2019-04-04
JP7812830B2 (ja) 2026-02-10
KR20150131251A (ko) 2015-11-24
JP2020031637A (ja) 2020-03-05
US20230407341A1 (en) 2023-12-21
EP3741868B1 (en) 2024-05-22
CA2906553C (en) 2022-08-02
KR102271292B1 (ko) 2021-07-02
US20240240207A1 (en) 2024-07-18
US20250034597A1 (en) 2025-01-30
US20210130850A1 (en) 2021-05-06
KR102271291B1 (ko) 2021-07-02
AU2014227653A1 (en) 2015-10-01
US10138476B2 (en) 2018-11-27
EP2971125A4 (en) 2016-08-24

Similar Documents

Publication Publication Date Title
JP6960438B2 (ja) 特定のゲノム遺伝子座へのゲノムおよびエピゲノム調節タンパク質のｒｎａ誘導型標的化
Moradpour et al. CRISPR/dC as9 platforms in plants: strategies and applications beyond genome editing
US20200354704A1 (en) RNA-Guided Targeting of Genetic and Epigenomic Regulatory Proteins to Specific Genomic Loci
JP7138712B2 (ja) ゲノム編集のためのシステム及び方法
JP2023503618A (ja) 遺伝子発現を活性化するためのシステムおよび方法
JP2022512868A (ja) Ｃ２ｃ１ヌクレアーゼに基づくゲノム編集のためのシステムおよび方法
CN112266418A (zh) 改进的基因组编辑系统及其应用
US9353358B2 (en) Sequence-specific engineered ribonuclease H and the method for determining the sequence preference of DNA-RNA hybrid binding proteins
CN112585266A (zh) 新型转录激活物

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170308

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180612

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20180907

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20181112

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181212

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190528

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190927

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20191128

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20200107

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20200205

R150 Certificate of patent or registration of utility model

Ref document number: 6657069

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250