HK1256817A1 - 用於基因組編輯的組合物和方法 - Google Patents

用於基因組編輯的組合物和方法

Info

Publication number
HK1256817A1
HK1256817A1 HK18115902.5A HK18115902A HK1256817A1 HK 1256817 A1 HK1256817 A1 HK 1256817A1 HK 18115902 A HK18115902 A HK 18115902A HK 1256817 A1 HK1256817 A1 HK 1256817A1
Authority
HK
Hong Kong
Prior art keywords
compositions
methods
genome editing
editing
genome
Prior art date
Application number
HK18115902.5A
Other languages
English (en)
Inventor
Sudhakar Kadiyala
Mark T Bilodeau
Donna T Ward
Original Assignee
Tarveda Therapeutics Inc
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
Application filed by Tarveda Therapeutics Inc filed Critical Tarveda Therapeutics Inc
Publication of HK1256817A1 publication Critical patent/HK1256817A1/zh

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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
HK18115902.5A 2015-09-25 2018-12-11 用於基因組編輯的組合物和方法 HK1256817A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562232625P 2015-09-25 2015-09-25
PCT/US2016/053325 WO2017053713A1 (en) 2015-09-25 2016-09-23 Compositions and methods for genome editing

Publications (1)

Publication Number Publication Date
HK1256817A1 true HK1256817A1 (zh) 2019-10-04

Family

ID=58387401

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18115902.5A HK1256817A1 (zh) 2015-09-25 2018-12-11 用於基因組編輯的組合物和方法

Country Status (4)

Country Link
US (1) US20180258411A1 (zh)
EP (1) EP3353309A4 (zh)
HK (1) HK1256817A1 (zh)
WO (1) WO2017053713A1 (zh)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10548761B2 (en) 2011-02-04 2020-02-04 Joseph E. Kovarik Method and system for reducing the likelihood of colorectal cancer in a human being
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
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
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP4063503A1 (en) 2014-02-11 2022-09-28 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
CA2944978C (en) 2014-04-08 2024-02-13 North Carolina State University Methods and compositions for rna-directed repression of transcription using crispr-associated genes
US10077453B2 (en) 2014-07-30 2018-09-18 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
EP4039816A1 (en) 2015-05-29 2022-08-10 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
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
EP3356533A1 (en) * 2015-09-28 2018-08-08 North Carolina State University Methods and compositions for sequence specific antimicrobials
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and their uses
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
JP2019518478A (ja) 2016-06-24 2019-07-04 ザ リージェンツ オブ ザ ユニバーシティ オブ コロラド，ア ボディー コーポレイトＴＨＥ ＲＥＧＥＮＴＳ ＯＦ ＴＨＥ ＵＮＩＶＥＲＳＩＴＹ ＯＦ ＣＯＬＯＲＡＤＯ，ａ ｂｏｄｙ ｃｏｒｐｏｒａｔｅ バーコードを付けたコンビナトリアルライブラリーを生成する方法
IL308426A (en) 2016-08-03 2024-01-01 Harvard College Adenosine nuclear base editors and their uses
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 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
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018136823A1 (en) 2017-01-19 2018-07-26 Open Monoclonal Technology, Inc. Human antibodies from transgenic rodents with multiple heavy chain immunoglobulin loci
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592777A1 (en) 2017-03-10 2020-01-15 President and Fellows of Harvard College Cytosine to guanine base editor
JP7191388B2 (ja) 2017-03-23 2022-12-19 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 核酸によってプログラム可能なｄｎａ結合蛋白質を含む核酸塩基編集因子
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
EP3635113A4 (en) 2017-06-05 2021-03-17 Fred Hutchinson Cancer Research Center GENOMIC SAFE HARBORS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND MANIPULATED NANOPARTICLES TO DELIVER TARGETED GENETIC THERAPIES
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
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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN118389609A (zh) * 2017-09-11 2024-07-26 加利福尼亚大学董事会 抗体介导的cas9向哺乳动物细胞的递送
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019108670A1 (en) * 2017-11-28 2019-06-06 Emendobio Inc. Differential knockout of an allele of a heterozygous fibrinogen alpha chain (fga) gene
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
JP2021535226A (ja) * 2018-09-04 2021-12-16 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
AU2019336679A1 (en) 2018-09-04 2021-03-25 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3861120A4 (en) 2018-10-01 2023-08-16 North Carolina State University RECOMBINANT TYPE I CRISPR-CAS SYSTEM
EP3914705A1 (en) * 2019-01-21 2021-12-01 Università di Pisa Nanotransducer-based genome editing system
KR20210123309A (ko) * 2019-01-25 2021-10-13 신테고 코포레이션 Crispr 활성을 조절하는 시스템 및 방법
WO2020191243A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220213456A1 (en) * 2019-04-30 2022-07-07 Emendobio Inc. Novel omni crispr nucleases
WO2020263782A1 (en) * 2019-06-24 2020-12-30 North Carolina State University Methods and compositions for modifying nucleic acids and for killing cells
US20230331804A1 (en) * 2019-12-31 2023-10-19 Fred Hutchinson Cancer Center Nanoparticle systems to stimulate and maintain immune system responsiveness at treatment sites
TWI738168B (zh) * 2020-01-03 2021-09-01 國立高雄大學 以Cas9胜肽拓印甲殼素複合奈米粒子轉染細胞與調控基因表現的方法
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
WO2024163787A1 (en) * 2023-02-01 2024-08-08 University Hospitals Cleveland Medical Center Crispr technologies for diagnostic applications

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8772471B2 (en) * 2007-01-26 2014-07-08 Industry-University Cooperation Foundation Hanyang University Targeted delivery of siRNA
AU2013369982B2 (en) * 2012-12-28 2016-11-17 Tva (Abc), Llc Targeted conjugates encapsulated in particles and formulations thereof
EP2971167B1 (en) * 2013-03-14 2019-07-31 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US9228207B2 (en) * 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
US20150191744A1 (en) * 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling

Also Published As

Publication number Publication date
WO2017053713A1 (en) 2017-03-30
EP3353309A1 (en) 2018-08-01
EP3353309A4 (en) 2019-04-10
US20180258411A1 (en) 2018-09-13

Similar Documents

Publication Publication Date Title
HK1256817A1 (zh) 用於基因組編輯的組合物和方法
IL304398A (en) Preparations and methods for genome modification
IL273817A (en) Preparations and methods for editing rna
HK1257295A1 (zh) 用於免疫腫瘤學的組合物和方法
ZA201801578B (en) Methods and compositions for marker-free genome modification
IL271232A (en) Compounds and methods for editing the genome
HK1252358A1 (zh) 用於確定倍性的方法及組合物
ZA201802461B (en) Multiplexed genome editing
EP3548625C0 (en) COMPOSITIONS AND METHODS FOR INCREASING GENE EXPRESSION
GB201613135D0 (en) Genome editing
IL252970A0 (en) Compositions and methods for genome editing in the living body with high efficiency
GB2546350B (en) Compositions and methods
SG10201609929QA (en) Etching compositions and methods for using same
EP3350315A4 (en) METHODS FOR GENOME AUTOCATALYTIC EDITING AND NEUTRALIZATION OF GENOME AUTOCATALYTIC EDITION AND COMPOSITIONS THEREOF
GB201506509D0 (en) Nuclease-mediated genome editing
GB201421096D0 (en) Genome editing methods
GB201718876D0 (en) Antithrombin-heparin compositions and methods
IL258292A (en) Methods and compositions for reducing metastases
IL246879A0 (en) Preparations of Epilimod and methods of using them
HK1245158A1 (zh) 阿吡莫德組合物及其使用方法
WO2017107898A9 (en) Compositions and methods for gene editing
PL3256589T3 (pl) Kompozycje i sposoby kontrolowania leptinotarsa
GB2550632B (en) Compositions and uses and methods relating thereto
ZA201704942B (en) Compositions for improving budbreak and flowering
ZA201703123B (en) Combination long acting compositions and methods for hepatitis c