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 aptâmeroInfo
- 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
- Authority
- BR
- Brazil
- Prior art keywords
- aptamer
- transcript
- modulation
- gene expression
- expressible
- Prior art date
Links
- 108091023037 Aptamer Proteins 0.000 title abstract 4
- 230000014509 gene expression Effects 0.000 title abstract 3
- 230000001404 mediated effect Effects 0.000 title 1
- 230000008488 polyadenylation Effects 0.000 title 1
- 239000011230 binding agent Substances 0.000 abstract 4
- 108091026898 Leader sequence (mRNA) Proteins 0.000 abstract 2
- 238000000354 decomposition reaction Methods 0.000 abstract 1
- 230000005764 inhibitory process Effects 0.000 abstract 1
- 239000003446 ligand Substances 0.000 abstract 1
- 238000011144 upstream manufacturing Methods 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
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- 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
- C12N15/115—Aptamers, 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
-
- 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
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- 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
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING 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
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
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- 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
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- 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
- C12N2310/16—Aptamers
-
- 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
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
Landscapes
- 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.
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)
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)
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 |
-
2016
- 2016-11-11 WO PCT/US2016/061665 patent/WO2017083747A1/en active Application Filing
- 2016-11-11 AU AU2016353339A patent/AU2016353339B2/en active Active
- 2016-11-11 BR BR112018009621A patent/BR112018009621A8/pt active Search and Examination
- 2016-11-11 US US15/775,804 patent/US20180327747A1/en not_active Abandoned
- 2016-11-11 RU RU2018121041A patent/RU2769991C2/ru active
- 2016-11-11 CA CA3005224A patent/CA3005224A1/en active Pending
- 2016-11-11 ES ES16865151T patent/ES2969015T3/es active Active
- 2016-11-11 MX MX2018005950A patent/MX2018005950A/es unknown
- 2016-11-11 KR KR1020187015503A patent/KR20180083347A/ko not_active Application Discontinuation
- 2016-11-11 EP EP16865151.1A patent/EP3374506B1/en active Active
- 2016-11-11 JP JP2018524382A patent/JP7100361B2/ja active Active
- 2016-11-11 CN CN201680066010.2A patent/CN108350451A/zh active Pending
-
2019
- 2019-01-14 HK HK19100606.5A patent/HK1258245A1/zh unknown
-
2021
- 2021-01-08 US US17/144,456 patent/US20210310003A1/en active Pending
-
2022
- 2022-01-07 JP JP2022001604A patent/JP2022062019A/ja active Pending
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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Legal Events
Date | Code | Title | Description |
---|---|---|---|
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. |
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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] |