BR112023021283A2 - Junção de extremidade mediada por homologia não viral - Google Patents

Junção de extremidade mediada por homologia não viral

Info

Publication number
BR112023021283A2
BR112023021283A2 BR112023021283A BR112023021283A BR112023021283A2 BR 112023021283 A2 BR112023021283 A2 BR 112023021283A2 BR 112023021283 A BR112023021283 A BR 112023021283A BR 112023021283 A BR112023021283 A BR 112023021283A BR 112023021283 A2 BR112023021283 A2 BR 112023021283A2
Authority
BR
Brazil
Prior art keywords
mediated
viral
edge joining
homology
viral homology
Prior art date
Application number
BR112023021283A
Other languages
English (en)
Inventor
Aaron Cooper
Annie Truong
Brian Hsu
Pei-Ken Hsu
Robert Moot
Thomas Gardner
Original Assignee
Arsenal Biosciences 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 Arsenal Biosciences Inc filed Critical Arsenal Biosciences Inc
Publication of BR112023021283A2 publication Critical patent/BR112023021283A2/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
    • 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
    • C12N15/11—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
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • 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
    • C12N9/93—Ligases (6)
    • 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
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2510/00—Genetically modified 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
    • 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
    • C12R—INDEXING SCHEME ASSOCIATED WITH SUBCLASSES C12C - C12Q, RELATING TO MICROORGANISMS
    • C12R2001/00—Microorganisms ; Processes using microorganisms
    • C12R2001/01—Bacteria or Actinomycetales ; using bacteria or Actinomycetales
    • C12R2001/46—Streptococcus ; Enterococcus; Lactococcus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Hematology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

junção de extremidade mediada por homologia não viral. trata-se de composições e métodos para produzir células geneticamente modificadas, como células t, com uma inserção de um cassete em um lócus genômico designado com o uso de técnicas de edição de gene não viral. métodos para usar as células geneticamente modificadas para tratar ou prevenir uma doença em um sujeito também são fornecidos.
BR112023021283A 2021-04-13 2022-04-13 Junção de extremidade mediada por homologia não viral BR112023021283A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163174468P 2021-04-13 2021-04-13
PCT/US2022/024703 WO2022221467A1 (en) 2021-04-13 2022-04-13 Non-viral homology mediated end joining

Publications (1)

Publication Number Publication Date
BR112023021283A2 true BR112023021283A2 (pt) 2023-12-19

Family

ID=83639705

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023021283A BR112023021283A2 (pt) 2021-04-13 2022-04-13 Junção de extremidade mediada por homologia não viral

Country Status (13)

Country Link
US (1) US20240240164A1 (pt)
EP (1) EP4323514A1 (pt)
JP (1) JP2024513967A (pt)
KR (1) KR20230169221A (pt)
CN (1) CN117355607A (pt)
AU (1) AU2022257004A1 (pt)
BR (1) BR112023021283A2 (pt)
CA (1) CA3215080A1 (pt)
CL (1) CL2023003030A1 (pt)
CO (1) CO2023015207A2 (pt)
IL (1) IL307447A (pt)
MX (1) MX2023012044A (pt)
WO (1) WO2022221467A1 (pt)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170000743A1 (en) * 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
US20220017882A1 (en) * 2018-12-12 2022-01-20 The Regents Of The University Of California Compositions and methods for modifying a target nucleic acid

Also Published As

Publication number Publication date
IL307447A (en) 2023-12-01
EP4323514A1 (en) 2024-02-21
JP2024513967A (ja) 2024-03-27
AU2022257004A1 (en) 2023-11-23
MX2023012044A (es) 2024-01-05
CN117355607A (zh) 2024-01-05
KR20230169221A (ko) 2023-12-15
WO2022221467A1 (en) 2022-10-20
CA3215080A1 (en) 2022-10-20
CO2023015207A2 (es) 2024-02-05
CL2023003030A1 (es) 2024-03-22
US20240240164A1 (en) 2024-07-18

Similar Documents

Publication Publication Date Title
BR112022000641A2 (pt) Modificação genética de célula efetora imune e uso da mesma
BR112021006844A8 (pt) Métodos para editar um rna alvo e para tratar ou prevenir uma doença ou condição, rna editado ou uma célula hospedeira tendo um rna editado, rna que recruta desaminase, construção, biblioteca, composição, célula hospedeira, e, kit para editar um rna alvo .
MX2022011562A (es) Métodos y composiciones para edición dirigida de genoma.
BR112021020608A8 (pt) Rna recrutador de desaminase, construção, método para editar um rna alvo em uma célula hospedeira, e, construção, composição, célula, biblioteca ou kit
NZ741778A (en) Multiplexed genome editing
MX2021003457A (es) Composiciones y métodos para la edición génica de lactato deshidrogenasa (ldha).
CR20220063A (es) Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
MX2018001617A (es) Composiciones de crispr-cas9 diseñados y metodos de uso.
CO2021006363A2 (es) Composiciones y métodos para la expresión de transgenes a partir de un locus de albúmina
BR112022005602A2 (pt) Células efetoras de múltiplos direcionamentos e uso das mesmas
WO2022140577A3 (en) Compositions and methods for epigenetic editing
BR112022003970A2 (pt) Métodos e composições para integração genômica
BR112014029441A2 (pt) rna tendo como alvo dna; polinucleotídeo de dna; vetor de expressão recombinante; célula hospedeira geneticamente modificada in vitro; polipeptídeo de modificação dirigido a sítio variante; polipeptídeo de modificação dirigido a sítio quimérico; polinucleotídeo de rna; célula modificada geneticamente; organismo não humano transgênico cujo genoma compreende um transgene; composição; método de modificação específica de sítio de um dna de alvo; método de modulação de transcrição específica de sítio dentro de um dna de alvo; método de modificação específica de sítio em dna de alvo; método de promover clivagem e modificação específica de sítio de um dna de alvo em uma célula; método de produzir uma célula modificada geneticamente em um sujeito; método de modificar dna de alvo em uma célula modificada geneticamente; kit; kit para direcionar dna de alvo; método de modular seletivamente transcrição de um dna de alvo em uma célula hospedeira; ácido nucleico isolado; célula hospedeira modificada geneticamente; e biblioteca de ácidos nucleicos
BRPI0814781A8 (pt) ''proteína, sequência de ácido nucleico, método para a produção de ácido itacônico, vetor, célula hospedeira, e, uso de um ácido cis-aconítico descarboxilase''
BR112015012375A2 (pt) modificação e regulação de genoma à base de crispr
BR112022015969A2 (pt) Composições e métodos para enxerto de células editadas de base
AR054599A1 (es) Proteccion de la proteina krp mutante negativa dominante contra una inhibicion activa del complejo cdk -ciclina por la krp de tipo salvaje
BR112021019772A2 (pt) Reconstituição de cd3 em células efetoras imunes e ipsc geneticamente modificadas
WO2022051020A9 (en) Systems, methods, and compositions for rna-guided rna-targeting crispr effectors
BR112021007403A2 (pt) Composições e métodos para entrega de transgenes
BR112021021149A2 (pt) Métodos e composições para a geração de alelos dominantes utilizando a edição de genomas
BR112022005963A2 (pt) Células com expressão de transgenes sustentada
BR112021010781A2 (pt) Silenciamento genético por meio de edição de genoma
Abou-Alfa et al. Advances in cholangiocarcinoma research: report from the third Cholangiocarcinoma Foundation Annual Conference
MX2023002455A (es) Células inmunitarias modificadas con receptores cebadores.