BR112022017735A2 - Métodos e composições de supressão de defesa de hospedeiro para modular um genoma - Google Patents

Métodos e composições de supressão de defesa de hospedeiro para modular um genoma

Info

Publication number
BR112022017735A2
BR112022017735A2 BR112022017735A BR112022017735A BR112022017735A2 BR 112022017735 A2 BR112022017735 A2 BR 112022017735A2 BR 112022017735 A BR112022017735 A BR 112022017735A BR 112022017735 A BR112022017735 A BR 112022017735A BR 112022017735 A2 BR112022017735 A2 BR 112022017735A2
Authority
BR
Brazil
Prior art keywords
compositions
genome
modulate
host defense
host response
Prior art date
Application number
BR112022017735A
Other languages
English (en)
Inventor
James Citorik Robert
Rosenblum Rubens Jacob
Giovanna Silvia Cotta-Ramusino Cecilia
Edward Salomon William
Jun Wang Zi
Original Assignee
Flagship Pioneering Innovations Vi Llc
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 Flagship Pioneering Innovations Vi Llc filed Critical Flagship Pioneering Innovations Vi Llc
Publication of BR112022017735A2 publication Critical patent/BR112022017735A2/pt

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
    • C12N15/10—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
    • C12N15/113—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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/13—Exoribonucleases producing 5'-phosphomonoesters (3.1.13)
    • C12Y301/13002—Exoribonuclease H (3.1.13.2)
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16311—Human Immunodeficiency Virus, HIV concerning HIV regulatory proteins
    • C12N2740/16322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

MÉTODOS E COMPOSIÇÕES DE SUPRESSÃO DE DEFESA DE HOSPEDEIRO PARA MODULAR UM GENOMA. A presente invenção refere-se, por exemplo, a composições e métodos para modular uma resposta de hospedeiro a um sistema Gene Writer. Em algumas modalidades, a modulação da resposta de hospedeiro resulta em integração aumentada de uma sequência de ácidos nucleicos heterólogos de interesse em um genoma-alvo. Em algumas modalidades, a modulação da resposta de hospedeiro resulta em uma estabilidade aumentada, por exemplo, manutenção de uma inserção ou expressão da mesma. Em algumas modalidades, a modulação da resposta de hospedeiro resulta em citotoxicidade diminuída.
BR112022017735A 2020-03-05 2021-03-05 Métodos e composições de supressão de defesa de hospedeiro para modular um genoma BR112022017735A2 (pt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202062985750P 2020-03-05 2020-03-05
US202063035653P 2020-06-05 2020-06-05
US202163147529P 2021-02-09 2021-02-09
PCT/US2021/021213 WO2021178898A1 (en) 2020-03-05 2021-03-05 Host defense suppressing methods and compositions for modulating a genome

Publications (1)

Publication Number Publication Date
BR112022017735A2 true BR112022017735A2 (pt) 2022-11-29

Family

ID=77614443

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022017735A BR112022017735A2 (pt) 2020-03-05 2021-03-05 Métodos e composições de supressão de defesa de hospedeiro para modular um genoma

Country Status (8)

Country Link
US (1) US20230235358A1 (pt)
EP (1) EP4114928A4 (pt)
JP (1) JP2023516694A (pt)
CN (1) CN115485372A (pt)
AU (1) AU2021232069A1 (pt)
BR (1) BR112022017735A2 (pt)
CA (1) CA3174553A1 (pt)
WO (1) WO2021178898A1 (pt)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and their uses
US11752213B2 (en) 2015-12-21 2023-09-12 Duke University Surfaces having reduced non-specific binding and antigenicity
US11155584B2 (en) 2016-09-23 2021-10-26 Duke University Unstructured non-repetitive polypeptides having LCST behavior
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
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
US11680083B2 (en) 2017-06-30 2023-06-20 Duke University Order and disorder as a design principle for stimuli-responsive biopolymer networks
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR20210049859A (ko) 2018-08-28 2021-05-06 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하는 방법 및 조성물
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
US11512314B2 (en) 2019-07-12 2022-11-29 Duke University Amphiphilic polynucleotides
JP2022546592A (ja) 2019-09-03 2022-11-04 マイエロイド・セラピューティクス，インコーポレーテッド ゲノム組込みのための方法および組成物
BR112022017736A2 (pt) 2020-03-04 2022-11-29 Flagship Pioneering Innovations Vi Llc Métodos e composições aprimorados para modular um genoma
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
CN113293140A (zh) * 2021-05-25 2021-08-24 昆明理工大学 一种端粒酶阴性的小鼠alt细胞模型及其构建方法
MX2024002927A (es) 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
EP4422605A1 (en) * 2021-10-25 2024-09-04 Duke University Poegma-based lipid nanoparticles
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023183550A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Messenger ribonucleic acids with extended half-life
CN114717207B (zh) * 2022-04-25 2023-03-07 苏州泓迅生物科技股份有限公司 一种酵母细胞同源重组酶系、dna体外组装试剂及其应用
CN116239665B (zh) * 2023-03-10 2024-08-23 山东大学 Ncf4在制备调控炎症小体激活的制剂中的应用和作为炎症性疾病的治疗靶点中的应用
CN116327748B (zh) * 2023-04-21 2024-09-17 武汉科技大学 联合用药物在制备抗肿瘤药物中的用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110070241A1 (en) * 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
WO2015027142A2 (en) * 2013-08-23 2015-02-26 The Scripps Research Institute Enhancing efficiency of retroviral transduction of host cells
US11390884B2 (en) * 2015-05-11 2022-07-19 Editas Medicine, Inc. Optimized CRISPR/cas9 systems and methods for gene editing in stem cells
AU2017277647B2 (en) * 2016-06-08 2023-07-27 President And Fellows Of Harvard College Engineered viral vector reduces induction of inflammatory and immune responses
KR20210049859A (ko) * 2018-08-28 2021-05-06 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하는 방법 및 조성물
MX2022010991A (es) * 2020-03-04 2023-02-09 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.

Also Published As

Publication number Publication date
US20230235358A1 (en) 2023-07-27
EP4114928A1 (en) 2023-01-11
WO2021178898A1 (en) 2021-09-10
AU2021232069A1 (en) 2022-11-03
JP2023516694A (ja) 2023-04-20
WO2021178898A9 (en) 2021-11-25
EP4114928A4 (en) 2024-10-16
CN115485372A (zh) 2022-12-16
CA3174553A1 (en) 2021-09-10

Similar Documents

Publication Publication Date Title
BR112022017735A2 (pt) Métodos e composições de supressão de defesa de hospedeiro para modular um genoma
BR112022017715A2 (pt) Métodos e composições para modular um genoma
WO2018136396A3 (en) Crisprs
BR112022017736A2 (pt) Métodos e composições aprimorados para modular um genoma
MX2020006072A (es) Métodos y composiciones relacionados con cpf1 para la edición genica.
BR112022007481A2 (pt) Lipídios ionizáveis e composições de nanopartículas dos mesmos
EP4253551A3 (en) Novel crispr dna and rna targeting enzymes and systems
MX2019002339A (es) Analogos de 4'-fosfato y oligonucleotidos que los comprenden.
BR112017014986A2 (pt) erradicação guiada por rna de herpes simples tipo i e outros herpesvírus relacionados
BR112014000495A2 (pt) micro-organismos recombinante para a produção de metabólitos úteis
BR112017024534A2 (pt) sistemas de rna-guia/endonuclease cas9 inovadores
WO2018191278A3 (en) Targeted compositions
BR112018007177A2 (pt) edição de genoma multiplexado
BRPI0519690A2 (pt) composiÇÕes e mÉtodos para modular a expressço de genes usando oligonucleotÍdeos autoprotegidos
BR112016003127A2 (pt) “composições e métodos para modular rna”
BR112021007403A2 (pt) Composições e métodos para entrega de transgenes
BR112022017107A2 (pt) Derivados de piridazina para modulação de splicing de ácido nucleico
BR112021010781A2 (pt) Silenciamento genético por meio de edição de genoma
BR112022008468A2 (pt) Método para modificação direcionada de uma sequência de genoma de planta
ZA202202628B (en) Novel crispr dna targeting enzymes and systems
AU2018363837A1 (en) Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
BR112022026311A2 (pt) Nucleases crispr-cas de ocorrência não natural para edição de genoma
BR112022003389A2 (pt) Proteína vp1 modificada isolada do capsídeo do sorotipo 5 do vírus adeno-associado (aav5), ácido nucleico isolado, capsídeo isolado, vetor baseado em raav5 e seu uso, composição farmacêutica, método para entrega de produto gênico
BR112018009837A2 (pt) composição catalítica e composição curável
BR112021005777A2 (pt) Produtos e composições