CN101981189A - 能够以协同方式引起基因表达转录后沉默的方法和组合物 - Google Patents

能够以协同方式引起基因表达转录后沉默的方法和组合物 Download PDF

Info

Publication number
CN101981189A
CN101981189A CN2009801118031A CN200980111803A CN101981189A CN 101981189 A CN101981189 A CN 101981189A CN 2009801118031 A CN2009801118031 A CN 2009801118031A CN 200980111803 A CN200980111803 A CN 200980111803A CN 101981189 A CN101981189 A CN 101981189A
Authority
CN
China
Prior art keywords
polynucleotide
composition
mrna
target
vector
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
Application number
CN2009801118031A
Other languages
English (en)
Chinese (zh)
Inventor
哈比尔·阿瓦德略雷特
玛丽亚普里菲卡西·福特斯阿隆索
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.)
Proyecto de Biomedicina CIMA SL
Original Assignee
Proyecto de Biomedicina CIMA SL
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 Proyecto de Biomedicina CIMA SL filed Critical Proyecto de Biomedicina CIMA SL
Publication of CN101981189A publication Critical patent/CN101981189A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Communicable Diseases (AREA)
  • Zoology (AREA)
  • Oncology (AREA)
  • Wood Science & Technology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • AIDS & HIV (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN2009801118031A 2008-01-29 2009-01-29 能够以协同方式引起基因表达转录后沉默的方法和组合物 Pending CN101981189A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ES200800222 2008-01-29
ESP200800222 2008-01-29
PCT/ES2009/000045 WO2009095517A2 (es) 2008-01-29 2009-01-29 Métodos y composiciones capaces de provocar un silenciamiento post-transcripcional de la expresión génica de forma sinérgica

Publications (1)

Publication Number Publication Date
CN101981189A true CN101981189A (zh) 2011-02-23

Family

ID=40807539

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2009801118031A Pending CN101981189A (zh) 2008-01-29 2009-01-29 能够以协同方式引起基因表达转录后沉默的方法和组合物

Country Status (10)

Country Link
US (1) US20110030075A1 (https=)
EP (1) EP2246432A2 (https=)
JP (1) JP2011510647A (https=)
CN (1) CN101981189A (https=)
AU (1) AU2009209571A1 (https=)
BR (1) BRPI0906657A2 (https=)
CA (1) CA2713458A1 (https=)
MX (1) MX2010008326A (https=)
RU (1) RU2010136040A (https=)
WO (1) WO2009095517A2 (https=)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105324392A (zh) * 2013-04-20 2016-02-10 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
CN107746857A (zh) * 2017-08-07 2018-03-02 中国农业大学 一种抑制基因表达的rna干扰方法
CN108893487A (zh) * 2018-07-19 2018-11-27 中国农业科学院北京畜牧兽医研究所 一种含有C-Myc蛋白融合标签的植物表达质粒载体及其载体的构建方法
CN113924364A (zh) * 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
CN114159459A (zh) * 2021-12-20 2022-03-11 塔里木大学 miR-26a在制备治疗子宫内膜炎的药物中的应用

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
WO2012045894A1 (es) * 2010-10-05 2012-04-12 Proyecto De Biomedicina Cima, S.L. Compuestos y composiciones para el tratamiento de enfermedades debidas a la infección por el virus de la hepatitis b
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2164088C (en) 1993-06-07 2005-06-14 Gary J. Nabel Plasmids suitable for gene therapy
CA2225460A1 (en) 1995-06-23 1997-01-09 Winston Campbell Patterson Transcriptional regulation of genes encoding vascular endothelial growth factor receptors
US6692910B2 (en) * 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
EP1384784A1 (en) 2001-03-30 2004-01-28 Instituto Cientifico Y Tecnologico De Navarra, S.A. Method for reversible inhibition of gene expression by modified ribonucleoproteins
EP2390331B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
WO2003093441A2 (en) 2002-05-03 2003-11-13 Duke University A method of regulating gene expression
US20050060771A1 (en) 2003-09-11 2005-03-17 Farmer Andrew Alan siRNA encoding constructs and methods for using the same
WO2006012221A2 (en) 2004-06-25 2006-02-02 The Regents Of The University Of California Target cell-specific short interfering rna and methods of use thereof
WO2006135436A2 (en) 2004-10-22 2006-12-21 University Of Florida Research Foundation, Inc. Inhibition of gene expression and therapeutic uses thereof
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
EP1969000A2 (en) 2005-12-06 2008-09-17 Centre National de la Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 aav의 생산에 유용한 aav-rep78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
GB0701722D0 (en) * 2007-01-30 2007-03-07 Scottish Crop Res Inst Inhibition of gene expression

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105324392A (zh) * 2013-04-20 2016-02-10 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
CN107746857A (zh) * 2017-08-07 2018-03-02 中国农业大学 一种抑制基因表达的rna干扰方法
CN108893487A (zh) * 2018-07-19 2018-11-27 中国农业科学院北京畜牧兽医研究所 一种含有C-Myc蛋白融合标签的植物表达质粒载体及其载体的构建方法
CN113924364A (zh) * 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
CN114159459A (zh) * 2021-12-20 2022-03-11 塔里木大学 miR-26a在制备治疗子宫内膜炎的药物中的应用

Also Published As

Publication number Publication date
WO2009095517A2 (es) 2009-08-06
US20110030075A1 (en) 2011-02-03
JP2011510647A (ja) 2011-04-07
WO2009095517A3 (es) 2009-09-24
RU2010136040A (ru) 2012-03-10
AU2009209571A1 (en) 2009-08-06
EP2246432A2 (en) 2010-11-03
CA2713458A1 (en) 2009-08-06
BRPI0906657A2 (pt) 2015-07-14
MX2010008326A (es) 2010-08-18

Similar Documents

Publication Publication Date Title
CN101981189A (zh) 能够以协同方式引起基因表达转录后沉默的方法和组合物
US9523093B2 (en) Huntington's disease therapeutic compounds
US8487088B2 (en) RNA interference suppression of neurodegenerative diseases and methods of use thereof
US10006028B2 (en) Alternative export pathways for vector expressed RNA interference
KR20140071949A (ko) Hbv 치료
JP2024543782A (ja) Cagリピート疾患を治療するための組成物および方法
WO2004016780A1 (en) Adeno-associated virus vector inhibiting gene's function by rnai
WO2025202080A1 (en) Acta1 expression control elements
CN118320132A (zh) 重组腺相关病毒rAAV2/Rec2作为胰腺靶向性载体的应用
WO2007100091A1 (ja) フコシルトランスフェラーゼの発現を抑制するための組成物
Li Modulation of Myocardial Genes via Use of Adenoviral Vectors and RNA Interference Approaches
HK1236224B (en) Huntington's disease therapeutic compounds
HK1236224A1 (en) Huntington's disease therapeutic compounds

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
AD01 Patent right deemed abandoned
AD01 Patent right deemed abandoned

Effective date of abandoning: 20110223