JP7066956B2 - 巨大遺伝子の切除および挿入 - Google Patents

巨大遺伝子の切除および挿入 Download PDF

Info

Publication number
JP7066956B2
JP7066956B2 JP2016532531A JP2016532531A JP7066956B2 JP 7066956 B2 JP7066956 B2 JP 7066956B2 JP 2016532531 A JP2016532531 A JP 2016532531A JP 2016532531 A JP2016532531 A JP 2016532531A JP 7066956 B2 JP7066956 B2 JP 7066956B2
Authority
JP
Japan
Prior art keywords
base pairs
nucleic acid
acid sequence
length
dna
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
JP2016532531A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016537982A5 (enExample
JP2016537982A (ja
Inventor
スーザン エム． バーン、
ジョージ エム． チャーチ、
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Harvard University
Original Assignee
Harvard University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=53173682&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP7066956(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Harvard University filed Critical Harvard University
Publication of JP2016537982A publication Critical patent/JP2016537982A/ja
Publication of JP2016537982A5 publication Critical patent/JP2016537982A5/ja
Application granted granted Critical
Publication of JP7066956B2 publication Critical patent/JP7066956B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2016532531A 2013-11-19 2014-11-19 巨大遺伝子の切除および挿入 Active JP7066956B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361906188P 2013-11-19 2013-11-19
US61/906,188 2013-11-19
PCT/US2014/066324 WO2015077290A2 (en) 2013-11-19 2014-11-19 Large gene excision and insertion

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019230265A Division JP7036512B2 (ja) 2013-11-19 2019-12-20 巨大遺伝子の切除および挿入

Publications (3)

Publication Number Publication Date
JP2016537982A JP2016537982A (ja) 2016-12-08
JP2016537982A5 JP2016537982A5 (enExample) 2017-12-28
JP7066956B2 true JP7066956B2 (ja) 2022-05-16

Family

ID=53173682

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2016532531A Active JP7066956B2 (ja) 2013-11-19 2014-11-19 巨大遺伝子の切除および挿入
JP2019230265A Active JP7036512B2 (ja) 2013-11-19 2019-12-20 巨大遺伝子の切除および挿入

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2019230265A Active JP7036512B2 (ja) 2013-11-19 2019-12-20 巨大遺伝子の切除および挿入

Country Status (9)

Country Link
US (2) US10787684B2 (enExample)
EP (3) EP3604543A1 (enExample)
JP (2) JP7066956B2 (enExample)
KR (3) KR20240104191A (enExample)
AU (3) AU2014353100B2 (enExample)
CA (1) CA2930828C (enExample)
DK (1) DK3071698T3 (enExample)
ES (1) ES2754498T3 (enExample)
WO (1) WO2015077290A2 (enExample)

Families Citing this family (115)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
DK2986729T3 (en) 2013-04-16 2018-10-29 Regeneron Pharma TARGETED MODIFICATION OF ROOT THROUGH
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
WO2015066119A1 (en) 2013-10-30 2015-05-07 North Carolina State University Compositions and methods related to a type-ii crispr-cas system in lactobacillus buchneri
CN106459995B (zh) 2013-11-07 2020-02-21 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
WO2015088643A1 (en) 2013-12-11 2015-06-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
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
US20160029604A1 (en) 2014-04-28 2016-02-04 Recombinetics, Inc. Multiplex gene editing
RU2704283C9 (ru) 2014-06-06 2020-02-07 Регенерон Фармасьютикалз, Инк. Способы и композиции для модификации целевого локуса
PT3161128T (pt) 2014-06-26 2018-11-21 Regeneron Pharma Métodos e composições para modificações genéticas alvejadas e métodos de utilização
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
SG11201702309RA (en) 2014-10-15 2017-04-27 Regeneron Pharma Methods and compositions for generating or maintaining pluripotent cells
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
CA2968440A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
WO2016089433A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
EP3229586B1 (en) 2014-12-10 2025-07-30 Regents of the University of Minnesota Genetically modified cells, tissues, and organs for treating disease
BR112017013104A2 (en) 2014-12-19 2018-05-15 Regeneron Pharmaceuticals, Inc. methods for modifying a target genomic locus in a cell, for enhancing homologous recombination at a target genomic locus in a cell, and for producing an f0 generation from a nonhuman animal.
ES2945315T3 (es) 2015-01-27 2023-06-30 Inst Genetics & Developmental Biology Cas Método para realizar modificaciones específicas de sitio en la planta completa a través de la expresión transitoria de genes
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
EP3286571B1 (en) 2015-04-24 2021-08-18 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3095870A1 (en) 2015-05-19 2016-11-23 Kws Saat Se Methods for the in planta transformation of plants and manufacturing processes and products based and obtainable therefrom
WO2016196361A1 (en) 2015-05-29 2016-12-08 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
EP3919622A1 (en) 2015-06-01 2021-12-08 Temple University - Of The Commonwealth System of Higher Education Methods and compositions for rna-guided treatment of hiv infection
JP7051438B2 (ja) 2015-06-15 2022-04-11 ノース カロライナ ステート ユニバーシティ 核酸およびｒｎａに基づく抗菌剤の効率的な送達のための方法および組成物
GB2557123B (en) 2015-07-31 2021-11-03 Univ Minnesota Modified cells and methods of therapy
JP2018527920A (ja) * 2015-08-14 2018-09-27 インスティテュート・オブ・ジェネティクス・アンド・ディヴェロプメンタル・バイオロジー、チャイニーズ・アカデミー・オブ・サイエンシズＩｎｓｔｉｔｕｔｅ ｏｆ Ｇｅｎｅｔｉｃｓ ａｎｄ Ｄｅｖｅｌｏｐｍｅｎｔａｌ Ｂｉｏｌｏｇｙ， Ｃｈｉｎｅｓｅ Ａｃａｄｅｍｙ ｏｆ Ｓｃｉｅｎｃｅｓ 部位特異的ヌクレオチド置換によりグリホサート耐性イネを取得するための方法
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
JP2018532415A (ja) * 2015-11-06 2018-11-08 ザ ジャクソン ラボラトリー 大きなゲノムｄｎａノックインおよびその使用
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
CN105861485B (zh) * 2016-04-20 2021-08-17 上海伊丽萨生物科技有限公司 一种提高基因置换效率的方法
US20170332610A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
WO2018025206A1 (en) * 2016-08-02 2018-02-08 Kyoto University Method for genome editing
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
EP3500677A4 (en) * 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
GB2573406B (en) 2016-10-18 2021-11-10 Univ Minnesota Tumor infiltrating lymphocytes and methods of therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3612023A4 (en) 2017-04-20 2021-05-12 Egenesis, Inc. METHOD FOR PRODUCING GENETICALLY MODIFIED ANIMALS
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
SG11201912179SA (en) * 2017-06-15 2020-01-30 Univ California Targeted non-viral dna insertions
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111315889A (zh) * 2017-09-08 2020-06-19 生命技术公司 提高同源重组的方法和其组合物
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
JPWO2019093418A1 (ja) * 2017-11-13 2021-01-21 国立大学法人広島大学 哺乳動物細胞内の標的染色体部位で目的遺伝子を含むポリヌクレオチドを増幅させる方法およびベクター、ならびにその利用
WO2019099982A1 (en) * 2017-11-17 2019-05-23 The Johns Hopkins University Compositions and methods for efficient genome editing
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR102907245B1 (ko) 2018-03-14 2026-01-05 에디타스 메디신, 인코포레이티드 혈색소병증 치료를 위한 시스템 및 방법
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
US20210180045A1 (en) * 2018-08-31 2021-06-17 The Children's Hospital Of Philadelphia Scalable tagging of endogenous genes by homology-independent intron targeting
US10711267B2 (en) 2018-10-01 2020-07-14 North Carolina State University Recombinant type I CRISPR-Cas system
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
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
WO2020072253A1 (en) 2018-10-01 2020-04-09 North Carolina State University Recombinant type i crispr-cas system and uses thereof for screening for variant cells
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CA3136061A1 (en) * 2019-04-05 2020-10-08 Osaka University Method for producing knock-in cell
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4085145A4 (en) * 2019-12-30 2024-02-21 The Broad Institute Inc. Guided excision-transposition systems
JP6867635B1 (ja) * 2020-04-06 2021-04-28 株式会社Logomix ゲノム改変方法及びゲノム改変キット
AU2021254373A1 (en) * 2020-04-06 2022-11-03 Logomix Inc. Genome engineering method and genome engineering kit
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
BR112022024652B1 (pt) * 2020-06-06 2024-01-16 Lanzatech, Inc Micro-organismo geneticamente modificado, e, método para a produção de um produto
US20240424035A1 (en) * 2021-01-26 2024-12-26 Washington State University Bacteria producing gamma-aminobutyric acid (gaba) and uses of the bacteria
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
IL311570A (en) 2021-10-14 2024-05-01 Arsenal Biosciences Inc Immune cells with common expression chain and logic gate systems
WO2023077148A1 (en) 2021-11-01 2023-05-04 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
EP4452335A1 (en) 2021-12-22 2024-10-30 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
CN114891750A (zh) * 2022-04-29 2022-08-12 武汉大学 筛选经cyp3a4介导代谢毒性外源化合物的细胞模型及其构建方法、应用
TWI840799B (zh) * 2022-05-02 2024-05-01 國立臺灣海洋大學 水產生物及水產品之外源性分子追溯方法及其系統
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2025224182A2 (en) 2024-04-23 2025-10-30 Basecamp Research Ltd Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2126130E (pt) 2007-03-02 2015-08-03 Dupont Nutrition Biosci Aps Culturas com resistência melhorada a fagos
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
US20120315670A1 (en) 2009-11-02 2012-12-13 Gen9, Inc. Compositions and Methods for the Regulation of Multiple Genes of Interest in a Cell
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
EA024121B9 (ru) 2010-05-10 2017-01-30 Дзе Реджентс Ов Дзе Юниверсити Ов Калифорния Композиции эндорибонуклеаз и способы их использования
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
DK2836226T3 (en) 2012-02-24 2017-09-18 Hutchinson Fred Cancer Res COMPOSITIONS AND PROCEDURES FOR TREATING HEMOGLOBINOPATHY
NZ629427A (en) 2012-02-29 2016-04-29 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
JP6559063B2 (ja) 2012-05-07 2019-08-14 サンガモ セラピューティクス， インコーポレイテッド 導入遺伝子のヌクレアーゼ媒介標的化組み込みのための方法および組成物
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
WO2014022702A2 (en) 2012-08-03 2014-02-06 The Regents Of The University Of California Methods and compositions for controlling gene expression by rna processing
PL4397760T3 (pl) * 2012-10-23 2026-03-30 Toolgen Incorporated Kompozycja do rozszczepiania docelowego dna zawierająca prowadzące rna specyficzne dla docelowego dna i kwas nukleinowy kodujący białko cas lub białko cas oraz jej zastosowanie
IL239317B (en) * 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201912991WA (en) * 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
US20140359795A1 (en) * 2013-05-31 2014-12-04 Recombinetics, Inc. Genetic techniques for making animals with sortable sperm
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
ES2844174T3 (es) * 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
WO2015088643A1 (en) 2013-12-11 2015-06-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163394A1 (en) 2012-04-25 2013-10-31 Regeneron Pharmaceuticals, Inc. Nuclease-mediated targeting with large targeting vectors

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
ＢｉｏＣｈｅｍ．Ｂｉｏｐｈｙｓ．Ｒｅｓ．Ｃｏｍｍｕｎ．，２０１３．１１．０６（ｏｎｌｉｎｅ），Ｖｏｌ．４４１，ｐｐ．８１５－８１９
Ｇｅｎｅｔｉｃｓ，２０１３．０８，Ｖｏｌ．１９４，ｐｐ．１０２９－１０３５，Ｓｕｐｐｏｒｔｉｎｇ Ｉｎｆｏｒｍａｔｉｏｎ
Ｊ．Ｂｉｏｌ．Ｃｈｅｍ．，２０００，Ｖｏｌ．２７５，Ｎｏ．４０，ｐｐ．３０８３３－３０８３８
Ｊｏｕｒｎａｌ ｏｆ Ｇｕａｎｇｘｉ Ａｇｒｉｃ．ａｎｄ Ｂｉｏｌ．Ｓｃｉｅｎｃｅ，２００７，Ｖｏｌ．２６，Ｓｕｐ．，ｐｐ．１－６
Ｎｕｃｌｅｉｃ Ａｃｉｄｓ Ｒｅｓｅａｒｃｈ，２０１３．０８（ｏｎｌｉｎｅ），Ｖｏｌ．４１，Ｎｏ．２０，ｅ１８７
Ｐｒｏｃ．Ｎａｔｌ．Ａｃａｄ．Ｓｃｉ．ＵＳＡ，２００７，Ｖｏｌ．１０４，Ｎｏ．９，ｐｐ．３０５５－３０６０
Ｓｃｉｅｎｃｅ，２０１３．０１（ｏｎｌｉｎｅ），Ｖｏｌ．３３９，ｐｐ．８２３－８２６ ａｎｄ Ｓｕｐｐｌｅｍｅｎｔａｒｙ Ｍａｔｅｒｉａｌｓ

Also Published As

Publication number Publication date
AU2021201624A1 (en) 2021-04-08
US20150140664A1 (en) 2015-05-21
US20210147879A1 (en) 2021-05-20
EP3071698A4 (en) 2017-06-28
EP4560022A3 (en) 2025-08-20
CA2930828C (en) 2022-05-03
DK3071698T3 (da) 2019-11-18
WO2015077290A2 (en) 2015-05-28
KR20220100090A (ko) 2022-07-14
US10787684B2 (en) 2020-09-29
JP2020062033A (ja) 2020-04-23
EP3604543A1 (en) 2020-02-05
JP7036512B2 (ja) 2022-03-15
EP3071698A2 (en) 2016-09-28
AU2024203000A1 (en) 2024-05-30
EP3071698B1 (en) 2019-09-04
JP2016537982A (ja) 2016-12-08
AU2021201624B2 (en) 2024-02-08
KR20240104191A (ko) 2024-07-04
KR102417127B1 (ko) 2022-07-05
AU2014353100B2 (en) 2020-12-17
KR20160078502A (ko) 2016-07-04
ES2754498T3 (es) 2020-04-17
EP4560022A2 (en) 2025-05-28
WO2015077290A3 (en) 2015-08-06
CA2930828A1 (en) 2015-05-28
AU2014353100A1 (en) 2016-06-02

Similar Documents

Publication Publication Date Title
JP7036512B2 (ja) 巨大遺伝子の切除および挿入
JP7676351B2 (ja) 多重ｒｎａ誘導型ゲノム編集
US20240352489A1 (en) Methods and compositions for modifying a targeted locus
JP6896786B2 (ja) 配列操作のためのＣＲＩＳＰＲ−Ｃａｓ成分系、方法および組成物
JP2023168564A (ja) Ｒｎａ誘導性ヒトゲノム改変
US9074199B1 (en) Mutant Cas9 proteins
CN111304230B (zh) 基因组工程
CN113423831A (zh) 核酸酶介导的重复扩增
WO2021206054A1 (ja) ゲノム改変方法及びゲノム改変キット
WO2020036653A2 (en) Improved method for homology directed repair in cells
HK40023048A (en) Large gene excision and insertion
HK1229380A1 (en) Large gene excision and insertion
HK1229380B (en) Large gene excision and insertion

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20171120

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20171120

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20181218

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20190311

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190418

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20190820

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191220

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20191220

C11 Written invitation by the commissioner to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C11

Effective date: 20200107

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200205

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

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20200228

C21 Notice of transfer of a case for reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C21

Effective date: 20200303

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20200403

C211 Notice of termination of reconsideration by examiners before appeal proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C211

Effective date: 20200407

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20200923

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20210316

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20210413

C302 Record of communication

Free format text: JAPANESE INTERMEDIATE CODE: C302

Effective date: 20210603

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210611

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20211005

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211115

C23 Notice of termination of proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C23

Effective date: 20220222

C03 Trial/appeal decision taken

Free format text: JAPANESE INTERMEDIATE CODE: C03

Effective date: 20220329

C30A Notification sent

Free format text: JAPANESE INTERMEDIATE CODE: C3012

Effective date: 20220329

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220405

R150 Certificate of patent or registration of utility model

Ref document number: 7066956

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