WO2012170431A3 - Improved geneswitch systems - Google Patents

Improved geneswitch systems Download PDF

Info

Publication number
WO2012170431A3
WO2012170431A3 PCT/US2012/040932 US2012040932W WO2012170431A3 WO 2012170431 A3 WO2012170431 A3 WO 2012170431A3 US 2012040932 W US2012040932 W US 2012040932W WO 2012170431 A3 WO2012170431 A3 WO 2012170431A3
Authority
WO
WIPO (PCT)
Prior art keywords
improved
systems
present
provides
geneswitch
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.)
Ceased
Application number
PCT/US2012/040932
Other languages
French (fr)
Other versions
WO2012170431A2 (en
Inventor
Robert H. KUTNER
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.)
Bluebird Bio Inc
Original Assignee
Bluebird Bio Inc
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 Bluebird Bio Inc filed Critical Bluebird Bio Inc
Publication of WO2012170431A2 publication Critical patent/WO2012170431A2/en
Publication of WO2012170431A3 publication Critical patent/WO2012170431A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10051—Methods of production or purification of viral material
    • C12N2740/10052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15051—Methods of production or purification of viral material
    • C12N2740/15052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The present invention provides improved molecular switch gene expression systems and improved retroviral packaging cells and viral particle production. In various embodiments, the present invention also provides retroviral vectors that have increased control over gene expression. Further, the present invention provides vector systems that are useful in methods of gene therapy.
PCT/US2012/040932 2011-06-06 2012-06-05 Improved geneswitch systems Ceased WO2012170431A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161493918P 2011-06-06 2011-06-06
US61/493,918 2011-06-06

Publications (2)

Publication Number Publication Date
WO2012170431A2 WO2012170431A2 (en) 2012-12-13
WO2012170431A3 true WO2012170431A3 (en) 2013-04-25

Family

ID=47296699

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/040932 Ceased WO2012170431A2 (en) 2011-06-06 2012-06-05 Improved geneswitch systems

Country Status (1)

Country Link
WO (1) WO2012170431A2 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108359014A (en) * 2018-02-08 2018-08-03 四川农业大学 The polyprotein of polypeptide and its mediation containing DHAV-1 2A1-P co-expresses method
CN109402178B (en) * 2018-11-16 2021-08-03 佛山科学技术学院 A method and application for efficient reprogramming of mouse spermatogonial stem cells

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201318954D0 (en) 2013-10-28 2013-12-11 Cupid Peptide Company Ltd Cell transport
ES2578903B1 (en) * 2015-01-02 2017-07-07 Instituto De Salud Carlos Iii LENTIVIRAL VECTOR OF SELF-LIMITED EXPRESSION
JP6760964B2 (en) * 2015-01-21 2020-09-23 コーネル ユニバーシティ Viral vector for prevention and treatment of abnormal hemoglobinosis
BR112018010639A2 (en) 2015-11-24 2019-01-22 Glaxosmithkline Ip Dev Ltd transient transfection method for retroviral production.
EP3380604B1 (en) * 2015-11-24 2022-12-28 GlaxoSmithKline Intellectual Property Development Limited Stable cell lines for retroviral production
GB201603374D0 (en) 2016-02-26 2016-04-13 Ucl Business Plc Packaging cell
JP2019522481A (en) 2016-06-22 2019-08-15 アイカーン スクール オブ メディシン アット マウント サイナイ Viral delivery of RNA using self-cleaving ribozymes and its CRISPR-based application
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
KR102944997B1 (en) 2018-08-24 2026-03-30 씨에스엘 베링 진 테라피, 인크. Vector preparation in serum-free medium
MX2022002211A (en) * 2019-08-29 2022-05-24 G Tech Bio Llc Compositions and methods for treating viral infections.
CA3171231A1 (en) * 2020-03-09 2021-09-16 Sean Michael Sullivan Compositions and methods for inducing immune responses
CN112143754B (en) * 2020-09-30 2021-05-18 扬州大学 Infectious clone of porcine Geta virus and its construction method and application
CN112941036B (en) * 2021-02-08 2023-06-09 吉林惠康生物药业有限公司 A method for improving the replication level of rabies virus in human diploid cells
CN113322282A (en) * 2021-04-20 2021-08-31 华南农业大学 Canine kidney cell line MDCK-pCDH-NS1 for stably expressing NS1 protein and construction method and application thereof
CN117304276B (en) * 2023-11-30 2024-04-12 江门海关技术中心 Virus-like particles containing foot-and-mouth disease virus type O and Sein card virus nucleic acid

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005014836A2 (en) * 2003-08-08 2005-02-17 Cambridge University Technical Services Limited Chimaeric vector system
WO2005026316A2 (en) * 2003-09-15 2005-03-24 Bioption Ab Alphavirus vaccines
US20080241929A1 (en) * 1999-04-29 2008-10-02 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20100221789A1 (en) * 2002-02-01 2010-09-02 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080241929A1 (en) * 1999-04-29 2008-10-02 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20100221789A1 (en) * 2002-02-01 2010-09-02 Life Technologies Corporation HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
WO2005014836A2 (en) * 2003-08-08 2005-02-17 Cambridge University Technical Services Limited Chimaeric vector system
WO2005026316A2 (en) * 2003-09-15 2005-03-24 Bioption Ab Alphavirus vaccines

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SURYANARAYANA, V. V. S. ET AL.: "Non-segmented negative sense RNA viruses as vectors for vaccine development", CURRENT SCIENCE, vol. 98, no. 3, 10 February 2010 (2010-02-10), pages 379 - 391, XP055066072 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108359014A (en) * 2018-02-08 2018-08-03 四川农业大学 The polyprotein of polypeptide and its mediation containing DHAV-1 2A1-P co-expresses method
CN109402178B (en) * 2018-11-16 2021-08-03 佛山科学技术学院 A method and application for efficient reprogramming of mouse spermatogonial stem cells

Also Published As

Publication number Publication date
WO2012170431A2 (en) 2012-12-13

Similar Documents

Publication Publication Date Title
WO2013014294A3 (en) Improved baculovirus expression systems
WO2014093661A9 (en) Crispr-cas systems and methods for altering expression of gene products
WO2014077866A8 (en) Replicative minicircle vectors with improved expression
EP4089172A3 (en) Multiple vector system and uses thereof
WO2012112578A3 (en) Improved aav8 vector with enhanced functional activity and methods of use thereof
MX347453B (en) Mutant channelrhodopsin 2.
EP4273257A3 (en) Inducible coexpression system
HK1207665A1 (en) Polypeptides having transgalactosylating activity
WO2013102144A3 (en) Ph20 polypeptide variants, formulations and uses thereof
WO2013063383A3 (en) Vectors encoding rod-derived cone viability factor
WO2012094560A3 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
HK1211981A1 (en) Methods to control protein heterogeneity
WO2012162534A3 (en) Hyaluronic acid-binding synthetic peptidoglycans, preparation, and methods of use
WO2012156839A3 (en) Splice - less lentiviral vectors for safter gene therapy applications
HK1213521A1 (en) Methods of using fix polypeptides
EP3246406A4 (en) Gene expression system using stealthy rna, and gene introduction/expression vector including said rna
WO2011084647A3 (en) Toxin-immunity system
EP3998341A3 (en) Adenoviral vectors
WO2014100073A3 (en) Expression vectors for recombinant protein production in mammalian cells
PH12016501689B1 (en) Tatk-cdkl5 fusion proteins, compositions, formulations, and use thereof
IN2015DN03206A (en)
EP3760713A3 (en) Lipase variants and polynucleotides encoding same
WO2014097318A3 (en) Agents for eliminating tumour-initiating cells
WO2013053857A3 (en) Gene cluster for biosynthesis of griselimycin and methylgriselimycin
WO2014036524A3 (en) Genetically modified msc and therapeutic methods

Legal Events

Date Code Title Description
NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 12796118

Country of ref document: EP

Kind code of ref document: A2