JP5399927B2 - 細胞内で細胞毒性を低減して抗ウイルス性小rna分子を発現させる方法 - Google Patents

細胞内で細胞毒性を低減して抗ウイルス性小rna分子を発現させる方法 Download PDF

Info

Publication number
JP5399927B2
JP5399927B2 JP2009552911A JP2009552911A JP5399927B2 JP 5399927 B2 JP5399927 B2 JP 5399927B2 JP 2009552911 A JP2009552911 A JP 2009552911A JP 2009552911 A JP2009552911 A JP 2009552911A JP 5399927 B2 JP5399927 B2 JP 5399927B2
Authority
JP
Japan
Prior art keywords
rna
region
cell
cells
expression
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.)
Expired - Fee Related
Application number
JP2009552911A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010520757A5 (enExample
JP2010520757A (ja
Inventor
ロイス−カバリエ,カルロス
ボルティモア,デイビッド
チン,シャオ−フェン
エス.ワイ. チェン,アーヴィン
サン アン,ドン
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.)
University of California
Original Assignee
University of California
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 University of California filed Critical University of California
Publication of JP2010520757A publication Critical patent/JP2010520757A/ja
Publication of JP2010520757A5 publication Critical patent/JP2010520757A5/ja
Application granted granted Critical
Publication of JP5399927B2 publication Critical patent/JP5399927B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/1131Non-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 against viruses
    • C12N15/1132Non-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 against viruses against retroviridae, e.g. 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
    • A61P31/14Antivirals 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
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • 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
    • 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
    • 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
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16045Special targeting system for viral vectors
    • 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
    • C12N2760/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011Details
    • C12N2760/20011Rhabdoviridae
    • C12N2760/20111Lyssavirus, e.g. rabies virus
    • C12N2760/20122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/609Vectors comprising as targeting moiety peptide derived from defined protein from viruses positive strand RNA viruses
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/001Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • AIDS & HIV (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2009552911A 2007-03-08 2008-03-07 細胞内で細胞毒性を低減して抗ウイルス性小rna分子を発現させる方法 Expired - Fee Related JP5399927B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11/683,962 US7737124B2 (en) 2001-09-13 2007-03-08 Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
US11/683,962 2007-03-08
PCT/US2008/056245 WO2008109837A2 (en) 2007-03-08 2008-03-07 Method for expression of small antiviral rna molecules with reduced cytotoxicity within a cell

Publications (3)

Publication Number Publication Date
JP2010520757A JP2010520757A (ja) 2010-06-17
JP2010520757A5 JP2010520757A5 (enExample) 2011-04-21
JP5399927B2 true JP5399927B2 (ja) 2014-01-29

Family

ID=39816837

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009552911A Expired - Fee Related JP5399927B2 (ja) 2007-03-08 2008-03-07 細胞内で細胞毒性を低減して抗ウイルス性小rna分子を発現させる方法

Country Status (6)

Country Link
US (6) US7737124B2 (enExample)
EP (1) EP2115164B1 (enExample)
JP (1) JP5399927B2 (enExample)
AU (1) AU2008222663B2 (enExample)
CA (2) CA3012986A1 (enExample)
WO (1) WO2008109837A2 (enExample)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7737124B2 (en) * 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
AU2002326906C1 (en) * 2001-09-13 2009-01-29 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
EP1451316B1 (en) 2001-09-13 2014-08-06 California Institute Of Technology Method for producing transgenic birds
GB2421948A (en) * 2004-12-30 2006-07-12 Ist Superiore Sanita Retrotransposon inhibition to treat cancer
AU2006252406B2 (en) 2005-06-01 2012-05-17 California Institute Of Technology Method of targeted gene delivery using viral vectors
SG173337A1 (en) 2006-07-21 2011-08-29 California Inst Of Techn Targeted gene delivery for dendritic cell vaccination
EP2166107A1 (en) * 2008-09-12 2010-03-24 TaconicArtemis GmbH Lentiviral vectors for the expression of shRNA
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
EP2425001A4 (en) * 2009-04-30 2012-11-14 Univ California ANTI-HIV COMBINATION VECTORS, TARGETING VECTORS AND METHOD OF USE
DK3329772T3 (da) * 2009-07-15 2020-01-27 Calimmune Inc Dobbeltvektor til inhibering af human immundefektvirus
WO2011011584A1 (en) 2009-07-24 2011-01-27 Immune Design Corp Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein
EP2831095B1 (en) 2012-03-30 2018-11-28 Immune Design Corp. Lentiviral vector particles having improved transduction efficiency for cells expressing dc-sign
US8323662B1 (en) 2012-03-30 2012-12-04 Immune Design Corp. Methods useful for generating highly mannosylated pseudotyped lentiviral vector particles comprising a Vpx protein
US9713635B2 (en) 2012-03-30 2017-07-25 Immune Design Corp. Materials and methods for producing improved lentiviral vector particles
CN104119285B (zh) * 2013-04-28 2016-06-29 中国科学院广州生物医药与健康研究院 用作雌激素相关受体调节剂的化合物及其应用
EP3027755B1 (en) 2013-08-02 2019-10-09 The Regents of The University of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
CN108026543A (zh) * 2015-05-18 2018-05-11 卡琳缪恩股份有限公司 用于hiv的治疗的基因疗法及其用途
EP3319631A4 (en) * 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
CA3011529A1 (en) 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta t-cells
US10888613B2 (en) * 2016-02-08 2021-01-12 American Gene Technologies International Inc. Method of producing cells resistant to HIV infection
JP6975465B2 (ja) * 2016-02-19 2021-12-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California 遺伝子改変細胞をポジティブに選択して、排除するための短ヘアピンrna(shrna734)およびその使用
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. INTEGRATED VIRAL ADMINISTRATION SYSTEM AND RELATED METHODS
IL300730A (en) 2016-07-08 2023-04-01 American Gene Tech Int Inc Pre-HIV vaccine and immunotherapy
JP7176756B2 (ja) 2016-07-21 2022-11-22 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド パーキンソン病を処置するためのウイルスベクター
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
AU2018367546B2 (en) * 2017-11-15 2024-07-18 Weird Science Llc Methods and compositions for non-myeloablative bone marrow reconstitution
WO2020097049A1 (en) 2018-11-05 2020-05-14 American Gene Technologies International Inc. Vector system for expressing regulatory rna
US20250057884A1 (en) * 2021-12-31 2025-02-20 Beijing Solobio Genetechnology Co. , Ltd. Chimeric Antigen Receptor-T Cells Targeting HIV-Infected Cells

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6096536A (en) * 1990-07-06 2000-08-01 University Of New Mexico In vitro cell culture in media containing beta-alanyl-taurine or carbobenzoxy beta-alanyl-taurine
US5258289A (en) 1990-09-05 1993-11-02 Davis Claude G Method for the selecting of genes encoding catalytic antibodies
US6664107B1 (en) * 1993-05-26 2003-12-16 Ontario Cancer Institute, University Health Network CD45 disrupted nucleic acid
US5859310A (en) 1993-06-14 1999-01-12 Basf Aktiengesellschaft Mice transgenic for a tetracycline-controlled transcriptional activator
IT1261847B (it) * 1993-09-01 1996-06-03 San Romanello Centro Fond Metodo per marcare cellule eucariote.
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5958713A (en) * 1995-01-31 1999-09-28 Novo Nordisk A/S Method of detecting biologically active substances by using green fluorescent protein
US5650309A (en) 1995-05-16 1997-07-22 The Regents Of The University Of California Viral vectors
GB9510272D0 (en) 1995-05-22 1995-07-19 Isis Innovation Retroviral vectors
US5883081A (en) 1995-07-26 1999-03-16 The Regents Of The University Of California Isolation of novel HIV-2 proviruses
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells
US6255071B1 (en) 1996-09-20 2001-07-03 Cold Spring Harbor Laboratory Mammalian viral vectors and their uses
DE19651443A1 (de) 1996-12-11 1998-06-18 Hoechst Ag Selbstverstärkende, pharmakologisch kontrollierbare Expressionssysteme
AU8828598A (en) * 1997-08-15 1999-03-08 Rubicon Laboratory, Inc. Retrovirus and viral vectors
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6100087A (en) * 1998-03-11 2000-08-08 City Of Hope Ribozymes targeted to human CCR5 mRNA
US6060317A (en) * 1998-08-11 2000-05-09 The United States Of America As Represented By The Department Of Health And Human Services Method of transducing mammalian cells, and products related thereto
JP2002532058A (ja) * 1998-08-20 2002-10-02 セル ジェネシス インコーポレイテッド 組換え遺伝子産物の産生の間の細胞傷害性を調節するためのサプレッサーtRNAの使用
US6274788B1 (en) 1998-09-23 2001-08-14 International Centre For Genetic Engineering And Biotechnology Bicistronic DNA construct comprising X-myc transgene for use in production of transgenic animal model systems for human hepatocellular carcinoma and transgenic animal model systems so produced
US6312956B1 (en) * 1999-10-01 2001-11-06 Vanderbilt University Nuclear targeted peptide nucleic acid oligomer
AU1333401A (en) * 1999-10-15 2001-04-30 Ohio State University Research Foundation, The Methods for analyzing the insertion capabilities of modified group ii introns
US6218186B1 (en) * 1999-11-12 2001-04-17 Trustees Of The University Of Pennsylvania HIV-MSCV hybrid viral vector for gene transfer
US20030084471A1 (en) * 2000-03-16 2003-05-01 David Beach Methods and compositions for RNA interference
EP1390472A4 (en) 2001-05-29 2004-11-17 Sirna Therapeutics Inc NUCLEIC ACID TREATMENT OF DISEASES OR SIDES RELATED TO RAS, HER2 AND HIV LEVELS
WO2003006691A1 (en) 2001-07-10 2003-01-23 Johnson & Johnson Research Pty Limited Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells
US20030219823A1 (en) 2001-09-07 2003-11-27 Alsobrook John P. Therapeutic polypeptides, nucleic acids encoding same, and methods of use
AU2002326906C1 (en) * 2001-09-13 2009-01-29 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
US7195916B2 (en) * 2001-09-13 2007-03-27 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
US7737124B2 (en) 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
EP2000160A3 (en) * 2002-10-30 2009-03-11 Gambro Lundia AB Method and apparatuses for determining the efficiency of dialysis
US20050221354A1 (en) * 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes

Also Published As

Publication number Publication date
EP2115164B1 (en) 2013-08-21
AU2008222663B2 (en) 2013-09-05
EP2115164A4 (en) 2011-01-19
US20150218564A1 (en) 2015-08-06
CA2680129C (en) 2018-09-11
EP2115164A2 (en) 2009-11-11
US20100267146A1 (en) 2010-10-21
WO2008109837A3 (en) 2008-11-13
JP2010520757A (ja) 2010-06-17
US8361982B2 (en) 2013-01-29
US20170107519A1 (en) 2017-04-20
US9567592B2 (en) 2017-02-14
AU2008222663A1 (en) 2008-09-12
WO2008109837A2 (en) 2008-09-12
US10041072B2 (en) 2018-08-07
US10876118B2 (en) 2020-12-29
US20190002883A1 (en) 2019-01-03
US20130295615A1 (en) 2013-11-07
CA3012986A1 (en) 2008-09-12
US20080003682A1 (en) 2008-01-03
US7737124B2 (en) 2010-06-15
CA2680129A1 (en) 2008-09-12
US9034577B2 (en) 2015-05-19

Similar Documents

Publication Publication Date Title
JP5399927B2 (ja) 細胞内で細胞毒性を低減して抗ウイルス性小rna分子を発現させる方法
US7732207B2 (en) Method for expression of small antiviral RNA molecules within a cell
US7195916B2 (en) Method for expression of small antiviral RNA molecules within a cell
CA2952590C (en) Method for expression of small antiviral rna molecules within a cell
AU2002326906A1 (en) Method for expression of small antiviral RNA molecules within a cell
Ringpis et al. Engineering HIV-1-resistant T-cells from short-hairpin RNA-expressing hematopoietic stem/progenitor cells in humanized BLT mice
RU2729385C2 (ru) Биопроизводство лентивирусных векторов
RU2426788C1 (ru) Генетические конструкции для антивич-терапии
RU2533817C1 (ru) Усовершенствование генетических конструкций для повышения эффективности антивич терапии
Spanevello Development of lentiviral vectors expressing multiple small interfering RNAs as a gene therapy approach against HIV-1 infection
t McManus Using Lentiviruses to Perform RNAi Knockdown of Neuronal Genes
Trobridge et al. Protection of Stem Cell-Derived Lymphocytes in a Primate AIDS Gene Therapy Model
Newton Production of self-inactivating lentiviral vectors to establish transgenic fetal equine kidney cell lines

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20110307

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20110307

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130305

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130604

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130611

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130701

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130708

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130829

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20131001

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20131024

R150 Certificate of patent or registration of utility model

Ref document number: 5399927

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees