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AR040575A1 - Plasmidos sinteticos optimizados en codones de expresion en mamiferos - Google Patents

Plasmidos sinteticos optimizados en codones de expresion en mamiferos

Info

Publication number
AR040575A1
AR040575A1 AR20030102543A ARP030102543A AR040575A1 AR 040575 A1 AR040575 A1 AR 040575A1 AR 20030102543 A AR20030102543 A AR 20030102543A AR P030102543 A ARP030102543 A AR P030102543A AR 040575 A1 AR040575 A1 AR 040575A1
Authority
AR
Argentina
Prior art keywords
synthetic
expression plasmid
replication
therapeutic
plasmid
Prior art date
Application number
AR20030102543A
Other languages
English (en)
Inventor
Ruxandra Draghia-Akli
Ronald V Abruzzese
Douglas R Kern
Original Assignee
Advisys 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 Advisys Inc filed Critical Advisys Inc
Publication of AR040575A1 publication Critical patent/AR040575A1/es

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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
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/02—Drugs for disorders of the endocrine system of the hypothalamic hormones, e.g. TRH, GnRH, CRH, GRH, somatostatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/06—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/60—Growth hormone-releasing factor [GH-RF], i.e. somatoliberin
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Un aspecto de la presente es un plásmido de expresión de mamífero sintético optimizado (por ejemplo pA V0201). Este nuevo plásmido comprende un elemento terapéutico y un elemento de replicación. El elemento terapéutico del nuevo plásmido comprende un promotor eucariota; una región 5´ no traducida ("UTR"); una secuencia de un gen terapéutico eucariota optimizado en codones; y una senal de poliadenilación. Los elementos terapéuticos del plásmido están operativamente ligados y localizados en una primera disposición operativamente ligada. Adicionalmente, el plásmido de expresión en mamíferos sintético optimizado comprende elementos de replicación, en donde los elementos de replicación están operativamente ligados y localizados en una segunda disposición operativamente ligada. Los elementos de replicación comprenden un promotor de un gen de marcador de selección, un sitio de unión a ribosomas y un origen de replicación. El primer dispositivo operativamente ligado y el segundo dispositivo operativamente ligado comprenden una estructura circular de un plásmido de expresión en mamíferos sintético optimizado en codones. Reivindicación 1: Un plásmido sintético de expresión en mamíferos que comprende: (a) un promotor sintético o eucariota; (b) una secuencia de un gen terapéutico eucariota optimizada en codones; (c) una senal de poliadenilación; (d) un promotor de un gen marcador de selección; (e) un sitio de unión a ribosomas; (f) una secuencia de un gen marcador de selección; y (g) un origen de replicación; en donde el promotor sintético o eucariota, la secuencia del gen terapéutico eucariota optimizada en codones y la senal de poliadenilación comprenden elementos terapéuticos del plásmido sintético de expresión en mamíferos; los elementos terapéuticos están operablemente unidos y localizados en una primera disposición operativamente ligada; el promotor del gen marcador de selección, el sitio de unión a ribosomas, la secuencia del gen marcador de selección y el origen de replicación comprenden elementos de replicación del plásmido sintético de expresión en mamíferos; los elementos de replicación están operablemente ligados y localizados a una segunda disposición operablemente ligada; la primera y la segunda disposición operablemente ligadas comprenden una estructura circular de un plásmido sintético de expresión en mamíferos; y el plásmido sintético de expresión en mamíferos es utilizado para la suplementación génica mediada por plásmidos. Reivindicación 32: Un método para la suplementación génica mediada por plásmidos que comprende: liberar dentro de un sujeto un plásmido sintético de expresión en mamíferos optimizado en codones; en donde el plásmido sintético de expresión en mamíferos optimizado en codones codifica en el sujeto una hormona de liberación de la hormona de crecimiento ("GHRH") o un equivalente biológico funcional.
AR20030102543A 2002-07-16 2003-07-15 Plasmidos sinteticos optimizados en codones de expresion en mamiferos AR040575A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US39624702P 2002-07-16 2002-07-16

Publications (1)

Publication Number Publication Date
AR040575A1 true AR040575A1 (es) 2005-04-13

Family

ID=30115994

Family Applications (1)

Application Number Title Priority Date Filing Date
AR20030102543A AR040575A1 (es) 2002-07-16 2003-07-15 Plasmidos sinteticos optimizados en codones de expresion en mamiferos

Country Status (8)

Country Link
US (2) US7316925B2 (es)
EP (1) EP1543020B1 (es)
AR (1) AR040575A1 (es)
AT (1) ATE505476T1 (es)
AU (1) AU2003249208B2 (es)
DE (1) DE60336736D1 (es)
TW (1) TW200403338A (es)
WO (1) WO2004007678A2 (es)

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Also Published As

Publication number Publication date
US20040092009A1 (en) 2004-05-13
AU2003249208B2 (en) 2010-03-04
ATE505476T1 (de) 2011-04-15
DE60336736D1 (de) 2011-05-26
AU2003249208A1 (en) 2004-02-02
EP1543020A4 (en) 2006-11-22
WO2004007678A2 (en) 2004-01-22
US7316925B2 (en) 2008-01-08
US20080194511A1 (en) 2008-08-14
EP1543020A2 (en) 2005-06-22
WO2004007678A3 (en) 2004-08-12
WO2004007678B1 (en) 2004-10-07
TW200403338A (en) 2004-03-01
EP1543020B1 (en) 2011-04-13

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