JP2011510647A - 遺伝子発現の転写後抑制を相乗的に生じさせることができる方法および組成物 - Google Patents

遺伝子発現の転写後抑制を相乗的に生じさせることができる方法および組成物 Download PDF

Info

Publication number
JP2011510647A
JP2011510647A JP2010544739A JP2010544739A JP2011510647A JP 2011510647 A JP2011510647 A JP 2011510647A JP 2010544739 A JP2010544739 A JP 2010544739A JP 2010544739 A JP2010544739 A JP 2010544739A JP 2011510647 A JP2011510647 A JP 2011510647A
Authority
JP
Japan
Prior art keywords
polynucleotide
mrna
composition
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
JP2010544739A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011510647A5 (enExample
Inventor
リョレトゥ、シャビエル アバ
アロンソ、マリア プリフィカシオン フォルテス
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 JP2011510647A publication Critical patent/JP2011510647A/ja
Publication of JP2011510647A5 publication Critical patent/JP2011510647A5/ja
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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • AIDS & HIV (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2010544739A 2008-01-29 2009-01-29 遺伝子発現の転写後抑制を相乗的に生じさせることができる方法および組成物 Pending JP2011510647A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ES200800222 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 (2)

Publication Number Publication Date
JP2011510647A true JP2011510647A (ja) 2011-04-07
JP2011510647A5 JP2011510647A5 (enExample) 2012-03-08

Family

ID=40807539

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2010544739A Pending JP2011510647A (ja) 2008-01-29 2009-01-29 遺伝子発現の転写後抑制を相乗的に生じさせることができる方法および組成物

Country Status (10)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson 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
WO2014172669A1 (en) * 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
CN107746857B (zh) * 2017-08-07 2021-02-05 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
CA3132388A1 (en) * 2019-03-08 2020-09-17 Rutgers, The State University Of New Jersey Compositions and methods for treating huntington's disease
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2008093141A1 (en) * 2007-01-30 2008-08-07 Scottish Crop Research Institute Inhibition of gene expression

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5910488A (en) 1993-06-07 1999-06-08 Vical Incorporated Plasmids suitable for gene therapy
EP0833907A1 (en) 1995-06-23 1998-04-08 President And Fellows Of Harvard College 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
WO2003029459A2 (en) 2001-09-28 2003-04-10 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna molecules
PT1504126E (pt) 2002-05-03 2014-06-02 Univ Duke Um método para regular a expressão génica
US20050060771A1 (en) 2003-09-11 2005-03-17 Farmer Andrew Alan siRNA encoding constructs and methods for using the same
US20080131940A1 (en) 2004-06-25 2008-06-05 Robert Chiu 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
EP1945779B1 (en) 2005-10-20 2013-03-06 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
CN101506369B (zh) 2006-06-21 2014-02-12 尤尼克尔生物制药股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav-rep78翻译起始密码子的载体

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2008093141A1 (en) * 2007-01-30 2008-08-07 Scottish Crop Research Institute Inhibition of gene expression

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JPN5011005346; LIU, P., et al.: 'MODIFIED U1 snRNA SUPPRESSES EXPRESSION OF A TARGETED ENDOGENOUS RNA BY INHIBITING POLYADENYLATION O' NUCLEIC ACIDS RESEARCH 32(4), 20040101, pp.1512-1517, OXFORD UNIVERSITY PRESS *
JPN5011005347; SAJIC, R., et al.: 'USE OF MODIFIED U1 SNRNAS TO INHIBIT HIV-1 REPLICATION' NUCLEIC ACIDS RESEARCH 35(1), 2007, pp.247-255 *
JPN5011005348; BECKLEY, S.A., et al.: 'REDUCTION OF TARGET GENE EXPRESSION BY A MODIFIED U1 SNRNA' MOLECULAR AND CELLULAR BIOLOGY 21(8), 200104, pp.2815-2825, AMERICAN SOCIETY FOR MICROBIOLOGY *

Also Published As

Publication number Publication date
RU2010136040A (ru) 2012-03-10
EP2246432A2 (en) 2010-11-03
CA2713458A1 (en) 2009-08-06
US20110030075A1 (en) 2011-02-03
MX2010008326A (es) 2010-08-18
CN101981189A (zh) 2011-02-23
BRPI0906657A2 (pt) 2015-07-14
AU2009209571A1 (en) 2009-08-06
WO2009095517A3 (es) 2009-09-24
WO2009095517A2 (es) 2009-08-06

Similar Documents

Publication Publication Date Title
JP2011510647A (ja) 遺伝子発現の転写後抑制を相乗的に生じさせることができる方法および組成物
RU2711147C2 (ru) Терапевтические соединения для лечения болезни хантингтона
US10006028B2 (en) Alternative export pathways for vector expressed RNA interference
CA2691617A1 (en) Reduction of off-target rna interference toxicity
US20200199625A1 (en) RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3
JP2021529758A (ja) 麻酔誘発性神経毒性の処置のための組成物及び方法
Price Applications of antisense oligonucleotides in designing a therapy for spinal muscular atrophy
HK1236224A1 (en) Huntington's disease therapeutic compounds
HK1236224B (en) Huntington's disease therapeutic compounds

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120117

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120117

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130827

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140304