CN104169419A - 用于输送生物活性rna的组合物和方法 - Google Patents

用于输送生物活性rna的组合物和方法 Download PDF

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Publication number
CN104169419A
CN104169419A CN201280070535.5A CN201280070535A CN104169419A CN 104169419 A CN104169419 A CN 104169419A CN 201280070535 A CN201280070535 A CN 201280070535A CN 104169419 A CN104169419 A CN 104169419A
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CN
China
Prior art keywords
rna
sequence
cell
biologically active
cells
Prior art date
Legal status (The legal status 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 status listed.)
Pending
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CN201280070535.5A
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English (en)
Chinese (zh)
Inventor
K.波拉克
J.费威尔
K.安沃
L.S.威尔金森
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
CLSN LAB Inc
Original Assignee
Egen Inc
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Publication date
Application filed by Egen Inc filed Critical Egen Inc
Publication of CN104169419A publication Critical patent/CN104169419A/zh
Pending legal-status Critical Current

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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
    • C12N15/111—General methods applicable to biologically active non-coding 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
    • C12N15/63—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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN201280070535.5A 2011-12-23 2012-12-24 用于输送生物活性rna的组合物和方法 Pending CN104169419A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161579815P 2011-12-23 2011-12-23
US61/579,815 2011-12-23
PCT/US2012/071576 WO2013096958A1 (en) 2011-12-23 2012-12-24 Compositions and methods for the delivery of biologically active rnas

Publications (1)

Publication Number Publication Date
CN104169419A true CN104169419A (zh) 2014-11-26

Family

ID=48654938

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280070535.5A Pending CN104169419A (zh) 2011-12-23 2012-12-24 用于输送生物活性rna的组合物和方法

Country Status (10)

Country Link
US (1) US20130164845A1 (https=)
EP (1) EP2794879A4 (https=)
JP (1) JP2015507474A (https=)
KR (1) KR20140123054A (https=)
CN (1) CN104169419A (https=)
AU (1) AU2012358181A1 (https=)
CA (1) CA2860228A1 (https=)
HK (1) HK1203548A1 (https=)
IN (1) IN2014CN04734A (https=)
WO (1) WO2013096958A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114807194A (zh) * 2020-08-31 2022-07-29 南京工业大学 一种提高梭菌中蛋白表达效率的方法

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11639371B2 (en) * 2016-02-17 2023-05-02 The Chinese University Of Hong Kong Peptidylic inhibitors of nucleolin (NCL) targeting CAG-repeat RNA toxicity and methods for reducing polyQ-mediated toxicity in polyQ diseases
US11352625B2 (en) 2017-01-12 2022-06-07 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
BR112019027692A2 (pt) * 2017-06-28 2020-11-24 University Of South Florida gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman
WO2020144295A1 (en) * 2019-01-10 2020-07-16 Biontech Rna Pharmaceuticals Gmbh Localized administration of rna molecules for therapy
KR102592844B1 (ko) * 2019-02-15 2023-10-20 아트레카, 인크. 종양 조직에 결합하는 항체, 및 그의 진단 및 치료 용도
CA3218711A1 (en) * 2021-05-13 2022-11-17 Roee Amit Methods and compositions for treatment of viral infection
US20250197888A1 (en) * 2022-03-24 2025-06-19 Cell & Brain Co., Ltd. Novel recombinant vector and use thereof
EP4303305A1 (en) * 2022-07-07 2024-01-10 Kutzner, Christoph Fusion polypeptides for cell penetration and cell targeting

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5880276A (en) * 1994-05-20 1999-03-09 The Research Foundation Of The State University Of Ny Purified retroviral constitutive transport enhancer elements that enhance nucleocytoplasmic transport of mRNA, and methods of making and using the elements
US20050008617A1 (en) * 2002-09-28 2005-01-13 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering RNA and short hairpin RNA
US20100233141A1 (en) * 2009-03-13 2010-09-16 Egen, Inc. Compositions And Methods For The Delivery Of Biologically Active RNAs

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1540004A4 (en) * 2002-07-31 2007-10-03 Nucleonics Inc DOUBLE-STRING RNA STRUCTURES AND CONSTRUCTS AND METHOD FOR THE PRODUCTION AND USE THEREOF
US20090004668A1 (en) * 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation
SI2539451T1 (sl) * 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5880276A (en) * 1994-05-20 1999-03-09 The Research Foundation Of The State University Of Ny Purified retroviral constitutive transport enhancer elements that enhance nucleocytoplasmic transport of mRNA, and methods of making and using the elements
US20050008617A1 (en) * 2002-09-28 2005-01-13 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering RNA and short hairpin RNA
US20100233141A1 (en) * 2009-03-13 2010-09-16 Egen, Inc. Compositions And Methods For The Delivery Of Biologically Active RNAs

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114807194A (zh) * 2020-08-31 2022-07-29 南京工业大学 一种提高梭菌中蛋白表达效率的方法
CN114807194B (zh) * 2020-08-31 2023-05-12 南京工业大学 一种提高梭菌中蛋白表达效率的方法

Also Published As

Publication number Publication date
JP2015507474A (ja) 2015-03-12
US20130164845A1 (en) 2013-06-27
AU2012358181A8 (en) 2015-06-18
CA2860228A1 (en) 2013-06-27
KR20140123054A (ko) 2014-10-21
EP2794879A4 (en) 2015-10-14
WO2013096958A8 (en) 2014-07-24
HK1203548A1 (en) 2015-10-30
AU2012358181A1 (en) 2014-07-10
EP2794879A1 (en) 2014-10-29
WO2013096958A1 (en) 2013-06-27
IN2014CN04734A (https=) 2015-09-18

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