ATE310096T1 - Expressionsvektoren mit hybriden ubiquitin- promotoren - Google Patents

Expressionsvektoren mit hybriden ubiquitin- promotoren

Info

Publication number
ATE310096T1
ATE310096T1 AT01971050T AT01971050T ATE310096T1 AT E310096 T1 ATE310096 T1 AT E310096T1 AT 01971050 T AT01971050 T AT 01971050T AT 01971050 T AT01971050 T AT 01971050T AT E310096 T1 ATE310096 T1 AT E310096T1
Authority
AT
Austria
Prior art keywords
expression
cmv
promoter
day
pdna
Prior art date
Application number
AT01971050T
Other languages
English (en)
Inventor
Nelson Yew
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of ATE310096T1 publication Critical patent/ATE310096T1/de

Links

Classifications

    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N15/67—General methods for enhancing the expression
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Pretreatment Of Seeds And Plants (AREA)
  • Detergent Compositions (AREA)
  • Developing Agents For Electrophotography (AREA)
AT01971050T 2000-09-18 2001-09-13 Expressionsvektoren mit hybriden ubiquitin- promotoren ATE310096T1 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US23393800P 2000-09-18 2000-09-18
US25956701P 2001-01-03 2001-01-03
PCT/US2001/028870 WO2002024932A2 (en) 2000-09-18 2001-09-13 Expression vectors containing hybrid ubiquitin promoters

Publications (1)

Publication Number Publication Date
ATE310096T1 true ATE310096T1 (de) 2005-12-15

Family

ID=26927393

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01971050T ATE310096T1 (de) 2000-09-18 2001-09-13 Expressionsvektoren mit hybriden ubiquitin- promotoren

Country Status (9)

Country Link
US (2) US6667174B2 (de)
EP (1) EP1319082B1 (de)
JP (4) JP2004516016A (de)
AT (1) ATE310096T1 (de)
AU (1) AU2001290984A1 (de)
CA (1) CA2423082A1 (de)
DE (1) DE60115070T2 (de)
ES (1) ES2252293T3 (de)
WO (1) WO2002024932A2 (de)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004516016A (ja) * 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
US20040002468A1 (en) * 2001-08-08 2004-01-01 Genzyme Corporation Methods of treating diabetes and other blood sugar disorders
US20040143104A1 (en) * 2001-08-08 2004-07-22 Wadsworth Samuel C. Methods of treating diabetes and other blood sugar disorders
US7341847B2 (en) * 2003-04-02 2008-03-11 Agency For Science, Technology And Research Promoter construct for gene expression in neuronal cells
US20050101017A1 (en) * 2003-11-10 2005-05-12 Wojtek Auerbach Method of improving gene targeting using a ubiquitin promoter
US20050107318A1 (en) * 2003-11-17 2005-05-19 Samuel Wadsworth Methods of treating diabetes and other blood sugar disorders
JPWO2005054467A1 (ja) 2003-12-03 2007-12-06 中外製薬株式会社 哺乳類βアクチンプロモーターを利用した発現系
CN107007842A (zh) 2005-05-02 2017-08-04 建新公司 神经代谢疾病的基因治疗
ES2394482T3 (es) * 2005-05-02 2013-02-01 Genzyme Corporation Terapia génica para trastornos de la médula espinal
BRPI0711965A2 (pt) 2006-06-07 2012-01-24 Genzyme Corp terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal
CN108853517A (zh) 2006-10-03 2018-11-23 建新公司 肌萎缩性侧索硬化症和其它脊髓病症的基因治疗
EP2076291A2 (de) * 2006-10-12 2009-07-08 Copernicus Therapeutics, Inc. Codon-optimierter cftp
BRPI0811265A2 (pt) 2007-05-16 2014-09-30 Brigham & Womens Hospital Tratamento de sinucleinopatias
PL2158322T3 (pl) 2007-06-06 2017-10-31 Genzyme Corp Terapia genowa dla lizosomalnych chorób spichrzeniowych
EP2271358B1 (de) 2008-03-25 2018-11-21 Amarantus Therapeutics, Inc. verwendung von mesencephalischem von Astrozyten abstammenden neurotrophen Faktor zur Behandlung von Parkinsonscher Erkrankung
HRP20212024T1 (hr) 2009-05-02 2022-04-01 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
WO2012099540A1 (en) * 2011-01-17 2012-07-26 Agency For Science, Technology And Research Cmv promoter variants
US9580699B2 (en) * 2014-04-17 2017-02-28 University of Pittsburgh—of the Commonwealth System of Higher Education TRPV1 modulatory gene product that affects TRPV1-specific pain behavioral responses identified in a functional screen of an HSV-based cDNA library
KR102244434B1 (ko) * 2014-08-11 2021-04-23 삼성전자주식회사 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법
CN104357474A (zh) * 2014-10-17 2015-02-18 四川农业大学 一种猪Sox6蛋白的体外表达及其多克隆抗体的制备方法
AU2015338923B2 (en) * 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
AU2016256895B2 (en) 2015-05-07 2022-05-26 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7014721B2 (ja) 2015-09-18 2022-02-01 ディーエヌエーアールエックス インビボでの核酸発現のためのシステム及び方法
WO2018175932A1 (en) * 2017-03-23 2018-09-27 DNARx Systems and methods for nucleic acid expression in vivo
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078464A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
DK3850089T5 (da) * 2019-02-04 2024-08-19 Freeline Therapeutics Ltd Polynukleotider
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
MX2021011958A (es) 2019-04-10 2021-12-15 Prevail Therapeutics Inc Terapias genicas para trastornos lisosomales.

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19539493A1 (de) * 1995-10-24 1997-04-30 Thomae Gmbh Dr K Starker homologer Promotor aus Hamster
FR2750704B1 (fr) * 1996-07-04 1998-09-25 Rhone Poulenc Rorer Sa Procede de production d'adn therapeutique
EP0961830A1 (de) * 1997-01-29 1999-12-08 Neurosearch A/S Expressionvektoren und methoden zur in vivo expression von therapeutischen polypeptiden
US5981275A (en) * 1997-04-14 1999-11-09 Genzyme Corporation Transgene expression system for increased persistence
AU2231099A (en) * 1998-01-16 1999-08-02 Genzyme Corporation Novel promoter elements for persistent gene expression
US20020042383A1 (en) * 1998-09-09 2002-04-11 Nelson S. Yew METHYLATION AND/Or CpG REMOVAL OF PLASMID VECTORS
JP2004516016A (ja) * 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター

Also Published As

Publication number Publication date
JP2016019524A (ja) 2016-02-04
JP2018007673A (ja) 2018-01-18
ES2252293T3 (es) 2006-05-16
DE60115070T2 (de) 2006-07-27
CA2423082A1 (en) 2002-03-28
EP1319082A2 (de) 2003-06-18
US7452716B2 (en) 2008-11-18
DE60115070D1 (en) 2005-12-22
WO2002024932A2 (en) 2002-03-28
AU2001290984A1 (en) 2002-04-02
JP6209566B2 (ja) 2017-10-04
US6667174B2 (en) 2003-12-23
EP1319082B1 (de) 2005-11-16
US20020090719A1 (en) 2002-07-11
JP2013048624A (ja) 2013-03-14
JP2004516016A (ja) 2004-06-03
US20070003521A1 (en) 2007-01-04
WO2002024932A3 (en) 2002-08-15

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