MX2024013592A - Sistemas multicomponentes para modificaciones del genoma en sitios especificos - Google Patents

Sistemas multicomponentes para modificaciones del genoma en sitios especificos

Info

Publication number
MX2024013592A
MX2024013592A MX2024013592A MX2024013592A MX2024013592A MX 2024013592 A MX2024013592 A MX 2024013592A MX 2024013592 A MX2024013592 A MX 2024013592A MX 2024013592 A MX2024013592 A MX 2024013592A MX 2024013592 A MX2024013592 A MX 2024013592A
Authority
MX
Mexico
Prior art keywords
expression
methods
introduction
systems
site
Prior art date
Application number
MX2024013592A
Other languages
English (en)
Spanish (es)
Inventor
Kathleen Collins
Ziaozhu Zhang
Treeck Briana Van
Heather E Upton
Sarah Palm
Jeremy Mcintyre
Original Assignee
Univ California
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 Univ California filed Critical Univ California
Publication of MX2024013592A publication Critical patent/MX2024013592A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • C12N9/10—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 [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2800/40—Systems of functionally co-operating vectors
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MX2024013592A 2022-05-02 2024-11-01 Sistemas multicomponentes para modificaciones del genoma en sitios especificos MX2024013592A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263337564P 2022-05-02 2022-05-02
PCT/US2023/066470 WO2023215727A2 (en) 2022-05-02 2023-05-02 Multicomponent systems for site-specific genome modifications

Publications (1)

Publication Number Publication Date
MX2024013592A true MX2024013592A (es) 2025-02-10

Family

ID=88647154

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024013592A MX2024013592A (es) 2022-05-02 2024-11-01 Sistemas multicomponentes para modificaciones del genoma en sitios especificos

Country Status (10)

Country Link
US (1) US20250049960A1 (enExample)
EP (1) EP4519424A4 (enExample)
JP (1) JP2025517630A (enExample)
KR (1) KR20250006975A (enExample)
CN (1) CN119630786A (enExample)
AU (1) AU2023264067A1 (enExample)
CA (1) CA3251169A1 (enExample)
IL (1) IL316725A (enExample)
MX (1) MX2024013592A (enExample)
WO (1) WO2023215727A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024020114A2 (en) * 2022-07-20 2024-01-25 Addition Therapeutics Genome insertions in cells
WO2025264587A1 (en) * 2024-06-17 2025-12-26 Addition Therapeutics, Inc. Rna tails for target primed reverse transcription

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2523657A1 (en) * 2003-04-25 2005-03-31 Medimmune Vaccines, Inc. Recombinant parainfluenza virus expression systems and vaccines comprising heterologous antigens derived from metapneumovirus
AU2019327449B2 (en) * 2018-08-28 2026-04-09 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
US20220298495A1 (en) * 2019-06-12 2022-09-22 Emendobio Inc. Novel genome editing tool
IL296095A (en) * 2020-03-04 2022-11-01 Flagship Pioneering Innovations Vi Llc Improved methods and compositions for genome modulation
WO2021233975A1 (en) * 2020-05-20 2021-11-25 Commissariat à l'Energie Atomique et aux Energies Alternatives In-cell continuous target-gene evolution, screening and selection
CN116745428A (zh) 2021-01-14 2023-09-12 加利福尼亚大学董事会 位点特异性基因修饰

Also Published As

Publication number Publication date
KR20250006975A (ko) 2025-01-13
CN119630786A (zh) 2025-03-14
US20250049960A1 (en) 2025-02-13
IL316725A (en) 2024-12-01
WO2023215727A3 (en) 2024-04-18
EP4519424A2 (en) 2025-03-12
EP4519424A4 (en) 2025-09-24
JP2025517630A (ja) 2025-06-10
WO2023215727A2 (en) 2023-11-09
AU2023264067A1 (en) 2024-11-28
CA3251169A1 (en) 2023-11-09

Similar Documents

Publication Publication Date Title
JP7698828B2 (ja) Ｒｎａを編集する方法および組成物
US20240336892A1 (en) Compositions and methods for the induction and tuning of gene expression
US12065669B2 (en) Methods and compositions for modulating a genome
Chen et al. Decorating chromatin for enhanced genome editing using CRISPR-Cas9
Reautschnig et al. Precise in vivo RNA base editing with a wobble-enhanced circular CLUSTER guide RNA
WO2023215727A3 (en) Multicomponent systems for site-specific genome modifications
JP7473969B2 (ja) 固定ガイドｒｎａペアを用いた遺伝子編集ベクターの作製方法
Proudfoot Ending the message: poly (A) signals then and now
US9957505B2 (en) Polynucleotides for multivalent RNA interference, compositions and methods of use thereof
CA3196116A1 (en) Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
O’Prey et al. Application of CRISPR/Cas9 to autophagy research
KR20150083115A (ko) 리보조절인자 조성물 및 사용 방법
JP2021529518A5 (enExample)
CN114144519B (zh) 单碱基置换蛋白以及包含其的组合物
KR20230135630A (ko) 유전자 전사 프레임워크, 벡터 시스템, 게놈 서열 편집 방법 및 응용
EP4688872A1 (en) Click-to-install genome editing
JP7031893B2 (ja) ゲノム編集酵素の活性を阻害する核酸アプタマー
KR102638799B1 (ko) CRISPR/Cas12f1(Cas14a1) system 효율화를 위한 engineered guide RNA 및 이의 용도
Liao et al. CRATES: A one-step assembly method for Class 2 CRISPR arrays
Skowron et al. An efficient method for the construction of artificial, concatemeric DNA, RNA and proteins with genetically programmed functions, using a novel, vector-enzymatic DNA fragment amplification-expression technology
AU784832B2 (en) Self-cleaving RNA sequences and their use for the control of protein synthesis
WO2022216804A3 (en) Specific oligonucleotide-programmed readthrough of nonsense codons
US20250354177A1 (en) System and methods for duplicating target fragments
Erkelenz Regulation of human and HIV-1 splice sites
Borchardt MOLECULAR APPROACHES FOR CONTROLLING RNA STABILITY