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PT2646557T - Método para expressão de arn celular - Google Patents

Método para expressão de arn celular Download PDF

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Publication number
PT2646557T
PT2646557T PT117914473T PT11791447T PT2646557T PT 2646557 T PT2646557 T PT 2646557T PT 117914473 T PT117914473 T PT 117914473T PT 11791447 T PT11791447 T PT 11791447T PT 2646557 T PT2646557 T PT 2646557T
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PT
Portugal
Prior art keywords
cells
pkr
rna
stem cell
fibroblasts
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PT117914473T
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English (en)
Inventor
Sahin Ugur
Beissert Tim
Poleganov Marco
Herz Stephanie
Original Assignee
Biontech Rna Pharmaceuticals Gmbh
Translationale Onkologie An Der Universitätsmedizin Der Jgu Mainz Ggmbh (Tron)
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Application filed by Biontech Rna Pharmaceuticals Gmbh, Translationale Onkologie An Der Universitätsmedizin Der Jgu Mainz Ggmbh (Tron) filed Critical Biontech Rna Pharmaceuticals Gmbh
Publication of PT2646557T publication Critical patent/PT2646557T/pt

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0696Artificially induced pluripotent stem cells, e.g. iPS
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70Enzymes
    • C12N2501/72Transferases [EC 2.]
    • C12N2501/727Kinases (EC 2.7.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Cell Biology (AREA)
  • Transplantation (AREA)
  • Developmental Biology & Embryology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Claims (12)

  1. REIVINDICAÇÕES
    1. Método para fornecer células que têm características de célula estaminal in vitro caracterizado por compreender as etapas de (i) reduzir a atividade da proteína quinase dependente de ARN (PKR) numa população de células que compreende células somáticas, (ii) introduzir ARN com capacidade para expressar um ou mais fatores que permitem a reprogramação das células somáticas para células que têm características de célula estaminal em pelo menos uma porção das células somáticas e (iii) permitir o desenvolvimento de células que têm características de célula estaminal.
  2. 2. Método, de acordo com a reivindicação 1, caracterizado por o um ou mais fatores compreenderem 0CT4 e S0X2.
  3. 3. Método, de acordo com a reivindicação 2, caracterizado por o um ou mais fatores compreenderem adicionalmente ELF4 e/ou c-MYC e/ou NANOG e/ou LIN28.
  4. 4. Método, de acordo com qualquer uma das reivindicações 1 a 3, caracterizado por a etapa de reduzir a atividade de PKR nas células resultar numa intensificação da estabilidade e/ou uma intensificação da expressão do ARN nas células.
  5. 5. Método, de acordo com a reivindicação 4, caracterizado por a intensificação da expressão do ARN nas células compreender um aumento no nivel de expressão e/ou um aumento na duração da expressão do ARN nas células.
  6. 6. Método, de acordo com qualquer uma das reivindicações 1 a 5, caracterizado por a etapa de reduzir a atividade de PKR nas células compreender tratar as células com pelo menos um inibidor de PKR ou silenciar a expressão do gene de PKR.
  7. 7. Método, de acordo com a reivindicação 6, caracterizado por o inibidor de PKR inibir a autof osf orilação de PKR induzida por ARN.
  8. 8. Método, de acordo com a reivindicação 6 ou 7, caracterizado por o inibidor de PKR ser um composto de imidazol-oxindol, 2-aminopurina ou um inibidor de PKR derivado de forma virai.
  9. 9. Método, de acordo com a reivindicação 8, caracterizado por o referido imidazol-oxindol ser 6,8-di-hidro-8-(1H-imidazol-5-ilmetileno)-7H-pirrolo[2,3—g]benzotiazol-7-ona e em que o referido inibidor de PKR derivado de forma virai é selecionado a partir do grupo que consiste em virus vaccinia E3 e/ou K3, ou seu ARN.
  10. 10. Método, de acordo com qualquer uma das reivindicações 1 a 9, caracterizado por as células somáticas serem fibroblastos.
  11. 11. Método, de acordo com a reivindicação 10, caracterizado por os referidos fibroblastos serem fibroblastos de pulmão, fibroblastos de prepúcio ou fibroblastos dérmicos.
  12. 12. Método para fornecer tipos de célula diferenciada caracterizado por compreender as etapas de (i) fornecer células que têm caracteristicas de célula estaminal com a utilização do método, conforme definido em qualquer uma das reivindicações 1 a 11, e (ii) cultivar as células que têm caracteristicas de célula estaminal sob condições que induzem ou direcionam a diferenciação parcial ou completa para um tipo de célula diferenciada. Lisboa,
PT117914473T 2010-12-03 2011-12-02 Método para expressão de arn celular PT2646557T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/EP2010/007362 WO2012072096A1 (en) 2010-12-03 2010-12-03 Method for cellular rna expression

Publications (1)

Publication Number Publication Date
PT2646557T true PT2646557T (pt) 2017-10-03

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Family Applications (1)

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PT117914473T PT2646557T (pt) 2010-12-03 2011-12-02 Método para expressão de arn celular

Country Status (10)

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US (1) US20140030808A1 (pt)
JP (2) JP6131433B2 (pt)
AU (1) AU2011335428B2 (pt)
CA (1) CA2819522C (pt)
DK (1) DK2646557T3 (pt)
ES (1) ES2640875T3 (pt)
HU (1) HUE034558T2 (pt)
LT (1) LT2646557T (pt)
PT (1) PT2646557T (pt)
WO (2) WO2012072096A1 (pt)

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* Cited by examiner, † Cited by third party
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CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3590949T (pt) 2010-10-01 2022-08-02 Modernatx Inc Ácidos ribonucleicos contendo n1-metilpseudouracilos e suas utilizações
ME02871B (me) * 2010-12-03 2018-04-20 Biontech Rna Pharmaceuticals Gmbh Postupak za ćelijsku ekspresiju rnk
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
SG11201402666WA (en) 2011-12-16 2014-10-30 Moderna Therapeutics Inc Modified nucleoside, nucleotide, and nucleic acid compositions
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
EP2847329A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF CYTOPLASMA AND CYTOSCELETTE PROTEINS
ES2676470T3 (es) 2012-11-09 2018-07-19 Biontech Rna Pharmaceuticals Gmbh Método para la expresión de ARN en células
RS63237B1 (sr) 2012-11-26 2022-06-30 Modernatx Inc Terminalno modifikovana rnk
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
EP4501318A2 (en) 2014-04-23 2025-02-05 ModernaTX, Inc. Nucleic acid vaccines
EP3750993B1 (en) * 2014-07-11 2023-12-27 Celgene Corporation Methods of improving vector transduction efficiency into t lymphocytes
KR101668074B1 (ko) * 2015-02-12 2016-10-21 전북대학교산학협력단 Pkr 저해제를 유효성분으로 포함하는 기관지 천식의 예방 또는 치료용 조성물
EP3261605B2 (en) 2015-02-26 2022-04-20 SiO2 Medical Products, Inc. Cycloolefin polymer container with a scratch resistant and anti-static coating
EP3405579A1 (en) 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
US20190167811A1 (en) 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
HUE061077T2 (hu) 2016-05-18 2023-05-28 Modernatx Inc Interleukin-12 (IL12) kódoló polinukleotidok és felhasználásuk
CA3027428A1 (en) * 2016-06-14 2017-12-21 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
US12195788B2 (en) * 2017-01-12 2025-01-14 University Of Central Florida Research Foundation, Inc. High-throughput and single nucleotide resolution techniques for the determination of RNA post-transcriptional modifications
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Also Published As

Publication number Publication date
LT2646557T (lt) 2017-10-10
CA2819522A1 (en) 2012-06-07
WO2012072269A1 (en) 2012-06-07
JP2013545469A (ja) 2013-12-26
CA2819522C (en) 2019-07-16
JP6131433B2 (ja) 2017-05-24
US20140030808A1 (en) 2014-01-30
JP2017079749A (ja) 2017-05-18
DK2646557T3 (en) 2017-10-02
HUE034558T2 (en) 2018-02-28
AU2011335428A1 (en) 2013-05-30
WO2012072096A1 (en) 2012-06-07
ES2640875T3 (es) 2017-11-07
AU2011335428B2 (en) 2016-12-22
WO2012072096A8 (en) 2012-07-26

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