BR112018009621A8 - controle exógeno da expressão gênica de mamíferos através da modulação de poliadenilação mediada por aptâmero - Google Patents

controle exógeno da expressão gênica de mamíferos através da modulação de poliadenilação mediada por aptâmero

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Publication number
BR112018009621A8
BR112018009621A8 BR112018009621A BR112018009621A BR112018009621A8 BR 112018009621 A8 BR112018009621 A8 BR 112018009621A8 BR 112018009621 A BR112018009621 A BR 112018009621A BR 112018009621 A BR112018009621 A BR 112018009621A BR 112018009621 A8 BR112018009621 A8 BR 112018009621A8
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BR
Brazil
Prior art keywords
aptamer
transcript
modulation
gene expression
expressible
Prior art date
Application number
BR112018009621A
Other languages
English (en)
Other versions
BR112018009621A2 (pt
Inventor
Yen Laising
Luo Liming
Chao Pei-Wen
Original Assignee
Baylor College Medicine
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 Baylor College Medicine filed Critical Baylor College Medicine
Publication of BR112018009621A2 publication Critical patent/BR112018009621A2/pt
Publication of BR112018009621A8 publication Critical patent/BR112018009621A8/pt

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    • 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
    • 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
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • 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
    • 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
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
    • 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
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

as modalidades da descrição dizem respeito ao uso de construções de expressão nas quais pelo menos um sinal polia é incorporado a montante de um transcrito expressável, tal como dentro de um 5' utr para o transcrito, por exemplo. em certas modalidades, o sinal polia é compreendido dentro de um aptâmero de ligação de ligante, e a ligação do ligante ao aptâmero, ou a falta dele, dita o resultado para o transcrito expressável. em modalidades específicas, a ausência do ligante faz com que o transcrito expressado possuindo um polia na sua 5' utr seja expressado mas depois decomposto, enquanto que a presença do ligante causa inibição da decomposição mediante a expressão do transcrito expressável. mais do que um aptâmero de ligação ao ligante pode estar presente na mesma construção de expressão.
BR112018009621A 2015-11-12 2016-11-11 controle exógeno da expressão gênica de mamíferos através da modulação de poliadenilação mediada por aptâmero BR112018009621A8 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562254435P 2015-11-12 2015-11-12
PCT/US2016/061665 WO2017083747A1 (en) 2015-11-12 2016-11-11 Exogenous control of mammalian gene expression through aptamer-mediated modulation of polyadenylation

Publications (2)

Publication Number Publication Date
BR112018009621A2 BR112018009621A2 (pt) 2018-11-06
BR112018009621A8 true BR112018009621A8 (pt) 2019-02-26

Family

ID=58696115

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018009621A BR112018009621A8 (pt) 2015-11-12 2016-11-11 controle exógeno da expressão gênica de mamíferos através da modulação de poliadenilação mediada por aptâmero

Country Status (13)

Country Link
US (2) US20180327747A1 (pt)
EP (1) EP3374506B1 (pt)
JP (2) JP7100361B2 (pt)
KR (1) KR20180083347A (pt)
CN (1) CN108350451A (pt)
AU (1) AU2016353339B2 (pt)
BR (1) BR112018009621A8 (pt)
CA (1) CA3005224A1 (pt)
ES (1) ES2969015T3 (pt)
HK (1) HK1258245A1 (pt)
MX (1) MX2018005950A (pt)
RU (1) RU2769991C2 (pt)
WO (1) WO2017083747A1 (pt)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220049619A (ko) * 2019-08-30 2022-04-21 베이롤 칼리지 오브 메드신 유전자 발현 조절 시스템
WO2023019203A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Inducible systems for altering gene expression in hypoimmunogenic cells

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1092777A1 (en) * 1999-10-15 2001-04-18 Aventis Pharma S.A. Self-cleaving RNA sequences and their use for the control of protein synthesis.
WO2002062949A2 (en) * 2000-10-20 2002-08-15 Canji, Inc Aptamer-mediated regulation of gene expression
WO2004027035A2 (en) 2002-09-20 2004-04-01 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches.
AU2003282538A1 (en) 2002-10-10 2004-05-04 Oxford Biomedica (Uk) Limited Gene regulation with aptamer and modulator complexes for gene therapy
US20110190386A1 (en) * 2008-02-11 2011-08-04 Kuo Paul C Aptamer Inhibitors of Osteopontin and Methods of Use Thereof
WO2010132665A1 (en) * 2009-05-15 2010-11-18 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
US8680017B2 (en) * 2010-02-13 2014-03-25 Gregory Francis Lopreato Lariat aptamer: aptamer candidate exclusion by nuclease digestion
US20160130585A1 (en) * 2013-05-28 2016-05-12 The Johns Hopkins University Aptamers for the treatment of sickle cell disease
CN104651501A (zh) * 2015-01-30 2015-05-27 湖南大学 裂开型激活式核酸适配体诊疗探针及其应用
CA3048620A1 (en) 2016-02-02 2017-08-10 Meiragtx Uk Ii Limited Regulation of gene expression through aptamer-modulated polyadenylation

Also Published As

Publication number Publication date
AU2016353339A1 (en) 2018-06-07
US20210310003A1 (en) 2021-10-07
WO2017083747A1 (en) 2017-05-18
HK1258245A1 (zh) 2019-11-08
KR20180083347A (ko) 2018-07-20
CN108350451A (zh) 2018-07-31
CA3005224A1 (en) 2017-05-18
JP2018533955A (ja) 2018-11-22
ES2969015T3 (es) 2024-05-16
MX2018005950A (es) 2018-11-09
JP7100361B2 (ja) 2022-07-13
EP3374506B1 (en) 2023-12-27
RU2018121041A (ru) 2019-12-16
US20180327747A1 (en) 2018-11-15
EP3374506A1 (en) 2018-09-19
RU2769991C2 (ru) 2022-04-12
AU2016353339B2 (en) 2023-06-01
JP2022062019A (ja) 2022-04-19
RU2018121041A3 (pt) 2020-04-27
BR112018009621A2 (pt) 2018-11-06
EP3374506A4 (en) 2019-04-17

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]