MA55527A - Polynucléotides, compositions et procédés d'expression de polypeptides - Google Patents

Polynucléotides, compositions et procédés d'expression de polypeptides

Info

Publication number
MA55527A
MA55527A MA055527A MA55527A MA55527A MA 55527 A MA55527 A MA 55527A MA 055527 A MA055527 A MA 055527A MA 55527 A MA55527 A MA 55527A MA 55527 A MA55527 A MA 55527A
Authority
MA
Morocco
Prior art keywords
polynucleotides
compositions
methods
expressing polypeptides
polypeptides
Prior art date
Application number
MA055527A
Other languages
English (en)
Inventor
Seth C Alexander
Christian Dombrowski
Bradley Andrew Murray
Original Assignee
Intellia 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 Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of MA55527A publication Critical patent/MA55527A/fr

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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
    • C12N15/67—General methods for enhancing the expression
    • 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
    • C12N15/11—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
    • C12N15/63—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
    • 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
    • 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
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
MA055527A 2019-03-28 2020-03-27 Polynucléotides, compositions et procédés d'expression de polypeptides MA55527A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962825656P 2019-03-28 2019-03-28

Publications (1)

Publication Number Publication Date
MA55527A true MA55527A (fr) 2022-02-09

Family

ID=70416544

Family Applications (1)

Application Number Title Priority Date Filing Date
MA055527A MA55527A (fr) 2019-03-28 2020-03-27 Polynucléotides, compositions et procédés d'expression de polypeptides

Country Status (17)

Country Link
US (1) US20230012687A1 (fr)
EP (1) EP3947670A2 (fr)
JP (1) JP2022527302A (fr)
KR (1) KR20220004649A (fr)
CN (1) CN113993994A (fr)
AU (1) AU2020248470A1 (fr)
BR (1) BR112021019224A2 (fr)
CA (1) CA3135172A1 (fr)
CO (1) CO2021014400A2 (fr)
EA (1) EA202192637A1 (fr)
IL (1) IL286579A (fr)
MA (1) MA55527A (fr)
MX (1) MX2021011757A (fr)
PH (1) PH12021552299A1 (fr)
SG (1) SG11202110135YA (fr)
TW (1) TW202102529A (fr)
WO (1) WO2020198641A2 (fr)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (fr) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions et procédés pour activer une réparation dirigée par homologie
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
AU2022382975A1 (en) 2021-11-03 2024-05-02 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CA3246480A1 (fr) * 2022-01-07 2023-07-13 Precision Biosciences, Inc. Polynucléotides optimisés pour l'expression de protéines
WO2023146230A1 (fr) * 2022-01-27 2023-08-03 에스케이바이오사이언스 주식회사 Arnm pour l'expression de protéines et matrice associée
US20250163454A1 (en) * 2022-02-09 2025-05-22 The Regents Of The University Of California In vitro and in vivo protein translation via in situ circularized rnas
TW202423959A (zh) * 2022-08-24 2024-06-16 美商步行魚醫療公司 用於治療法布瑞氏症(fabry disease)之組成物及方法
TW202540417A (zh) 2023-11-10 2025-10-16 美商英特利亞醫療公司 用於基因體編輯之組合物、方法及系統
WO2025128871A2 (fr) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition de gènes et en tant que vaccins et agents thérapeutiques

Family Cites Families (23)

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US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
EP1044987B1 (fr) 1991-12-24 2006-02-15 Isis Pharmaceuticals, Inc. Oligonucléotides modifiés en 2' à ouverture
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
JP5250850B2 (ja) * 2006-06-29 2013-07-31 ディーエスエム アイピー アセッツ ビー．ブイ． 改善されたポリペプチド発現を達成する方法
CA2825722A1 (fr) * 2011-01-28 2012-08-02 Medimmune, Llc Expression de glycoproteines de fusion virales solubles dans des cellules de mammiferes
WO2014093655A2 (fr) 2012-12-12 2014-06-19 The Broad Institute, Inc. Fabrication et optimisation de systèmes, de procédés et de compositions pour la manipulation de séquence avec des domaines fonctionnels
WO2014093694A1 (fr) 2012-12-12 2014-06-19 The Broad Institute, Inc. Systèmes, procédés et compositions de crispr-nickase cas pour la manipulation de séquences dans les eucaryotes
EP4282970A3 (fr) 2012-12-17 2024-01-17 President and Fellows of Harvard College Ingénierie de génome humain guidée par arn
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US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3623361B1 (fr) 2013-12-19 2021-08-18 Novartis AG Lipides et compositions de lipides pour l'administration d'agents actifs
WO2016010840A1 (fr) 2014-07-16 2016-01-21 Novartis Ag Procédé d'encapsulation d'un acide nucléique dans une nanoparticule lipidique hôte
WO2016141224A1 (fr) 2015-03-03 2016-09-09 The General Hospital Corporation Nucléases crispr-cas9 génétiquement modifiées présentant une spécificité pam modifiée
FI4104687T3 (fi) 2015-09-21 2024-03-01 Trilink Biotechnologies Llc Koostumuksia ja menetelmiä 5-huputettujen RNA:iden syntetisoimiseksi
RU2021102893A (ru) * 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
EP3405579A1 (fr) * 2016-01-22 2018-11-28 Modernatx, Inc. Acides ribonucléiques messagers pour la production de polypeptides de liaison intracellulaires et leurs procédés d'utilisation
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KR20250093420A (ko) * 2017-09-29 2025-06-24 인텔리아 테라퓨틱스, 인크. 게놈 편집을 위한 폴리뉴클레오티드, 조성물 및 방법

Also Published As

Publication number Publication date
CA3135172A1 (fr) 2020-10-01
WO2020198641A3 (fr) 2020-11-05
IL286579A (en) 2021-10-31
WO2020198641A2 (fr) 2020-10-01
SG11202110135YA (en) 2021-10-28
US20230012687A1 (en) 2023-01-19
EA202192637A1 (ru) 2022-03-18
JP2022527302A (ja) 2022-06-01
KR20220004649A (ko) 2022-01-11
CN113993994A (zh) 2022-01-28
CO2021014400A2 (es) 2021-11-19
AU2020248470A1 (en) 2021-11-11
PH12021552299A1 (en) 2022-08-22
EP3947670A2 (fr) 2022-02-09
MX2021011757A (es) 2021-12-10
TW202102529A (zh) 2021-01-16
BR112021019224A2 (pt) 2021-11-30

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