AU2008222663B2 - Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell - Google Patents

Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell Download PDF

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Publication number
AU2008222663B2
AU2008222663B2 AU2008222663A AU2008222663A AU2008222663B2 AU 2008222663 B2 AU2008222663 B2 AU 2008222663B2 AU 2008222663 A AU2008222663 A AU 2008222663A AU 2008222663 A AU2008222663 A AU 2008222663A AU 2008222663 B2 AU2008222663 B2 AU 2008222663B2
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Australia
Prior art keywords
rna
cell
region
expression
coding region
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AU2008222663A
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AU2008222663A1 (en
Inventor
Dong Sung An
David Baltimore
Irvin S.Y. Chen
Carlos Lois-Caballe
Xiao-Feng Qin
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California Institute of Technology
University of California, San Diego
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California Institute of Technology
University of California Berkeley
University of California, San Diego
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    • 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
    • C12N15/113—Non-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
    • C12N15/1131—Non-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
    • C12N15/1132—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1138—Non-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
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    • 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
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    • 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
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16045—Special targeting system for viral vectors
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    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20111—Lyssavirus, e.g. rabies virus
    • C12N2760/20122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
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    • 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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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • 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)
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AU2008222663A 2007-03-08 2008-03-07 Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell Ceased AU2008222663B2 (en)

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 (2)

Publication Number Publication Date
AU2008222663A1 AU2008222663A1 (en) 2008-09-12
AU2008222663B2 true AU2008222663B2 (en) 2013-09-05

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AU2008222663A Ceased AU2008222663B2 (en) 2007-03-08 2008-03-07 Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell

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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 (31)

* 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
CA2457282C (en) * 2001-09-13 2015-01-13 California Institute Of Technology Method for expression of small antiviral rna molecules within a cell
EP1425400B1 (en) 2001-09-13 2016-11-02 California Institute Of Technology Method for producing transgenic animals
GB2421948A (en) * 2004-12-30 2006-07-12 Ist Superiore Sanita Retrotransposon inhibition to treat cancer
EP2963118B1 (en) 2005-06-01 2017-08-09 California Institute of Technology Method of targeted gene delivery using viral vectors
WO2008011636A2 (en) 2006-07-21 2008-01-24 California Institute Of Technology 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
US20120201794A1 (en) * 2009-07-15 2012-08-09 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus
EP2456786B2 (en) 2009-07-24 2017-04-19 Immune Design Corp Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein
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
AU2013237900B2 (en) 2012-03-30 2017-07-27 Immune Design Corp. Lentiviral vector particles having improved transduction efficiency for cells expressing DC- SIGN
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
CA2986469A1 (en) * 2015-05-18 2016-11-24 Calimmune, Inc. Gene therapeutic for the treatment of hiv and uses thereof
US11980663B2 (en) * 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization 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
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
JP7153332B2 (ja) * 2016-02-08 2022-10-14 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 Ｈｉｖワクチン接種および免疫療法
WO2017143266A1 (en) * 2016-02-19 2017-08-24 The Regents Of The University Of California Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
JP7568507B2 (ja) * 2017-11-15 2024-10-16 ウィヤード・サイエンス・エルエルシー 非骨髄破壊的な骨髄再構成のための方法および組成物
EP3876952A4 (en) 2018-11-05 2022-08-24 American Gene Technologies International Inc. VECTOR SYSTEM FOR EXPRESSING REGULATORY RNA
JP2025501273A (ja) * 2021-12-31 2025-01-17 北京三▲諾▼佳邑生物技▲術▼有限▲責▼任公司 Ｈｉｖ感染細胞を標的とするキメラ抗原受容体ｔ細胞

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
CA2300957A1 (en) * 1997-08-15 1999-02-25 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
KR20010079671A (ko) 1998-08-20 2001-08-22 스티븐 에이. 서윈. 엠.디. 재조합 유전자 생성물을 생성시키는 동안 세포독성을조절하는 억제 ｔＲＮＡ의 용도
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
WO2001029059A1 (en) * 1999-10-15 2001-04-26 The Ohio State University Research Foundation 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
WO2002097114A2 (en) * 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of ras, her2 and hiv
BR0211079A (pt) * 2001-07-10 2005-08-30 Johnson & Johnson Res Pty Ltd Processos para a modificação genética de células hematopoiéticas progenitoras e empregos das células modificadas
US20030219823A1 (en) * 2001-09-07 2003-11-27 Alsobrook John P. Therapeutic polypeptides, nucleic acids encoding same, and methods of use
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
US7195916B2 (en) 2001-09-13 2007-03-27 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
CA2457282C (en) * 2001-09-13 2015-01-13 California Institute Of Technology Method for expression of small antiviral rna molecules 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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
An, D.S. et al., Molecular Therapy. 2006 vol. 14 no. 4 pages 494-504 *
Qin, X-F. et al., Proceedings of the National Academy of Sciences. 2003 vol. 100 no. 1 pages 183-188 *

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