ES2168096T3 - Virus adeno-asociado con secuencias terminales invertidas de repeticion como promotor para la transferencia in vivo de un gen cftr funcional. - Google Patents

Virus adeno-asociado con secuencias terminales invertidas de repeticion como promotor para la transferencia in vivo de un gen cftr funcional.

Info

Publication number
ES2168096T3
ES2168096T3 ES93916425T ES93916425T ES2168096T3 ES 2168096 T3 ES2168096 T3 ES 2168096T3 ES 93916425 T ES93916425 T ES 93916425T ES 93916425 T ES93916425 T ES 93916425T ES 2168096 T3 ES2168096 T3 ES 2168096T3
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Spain
Prior art keywords
vector
promoter
adeno
gene
gen
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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 - Lifetime
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ES93916425T
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English (en)
Inventor
Barrie J Carter
Terrence Flotte
Sandra Afione
Rikki Solow
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US Department of Health and Human Services
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US Department of Health and Human Services
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Publication of ES2168096T3 publication Critical patent/ES2168096T3/es
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Expired - Lifetime legal-status Critical Current

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    • 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
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

SE DESCRIBEN LAS CONSTRUCCIONES DE DNA RECOMBINANTE QUE COMPRENDE LAS SECUENCIAS DNA DEL VIRUS (AAV) ASOCIADO DE ADENO MODIFICADO CAPAZ DE FUNCIONAR COMO UN VECTOR DE EXPRESION EUCARIOTICO PARA EXPRESAR LAS SECUENCIAS DNA EXTRAÑAS QUE USAN UN NUEVO PROMOTOR DE TRANSCRIPCION QUE COMPRENDE EL TERMINO DEL DNA AAV. SE MUESTRA QUE LA EXPRESION DE UN GEN REPORTERO DE TEST PUEDE SER OBTENIDO DE SU VECTOR EN LAS CELULAS DE LOS MAMIFEROS. ADEMAS SE MUESTRA QUE ESTA COMBINACION DEL VECTOR Y EL PROMOTOR PUEDEN SER USADOS PARA INTRODUCIR Y EXPRESAR UN GEN HUMANO Y CORREGIR UN DEFECTO GENETICO EN LAS CELULAS HUMANAS QUE RESULTAN DE LA MALFUNCION DE UN GEN ENDOGENO MUTANTE. ADEMAS, EL VECTOR PUEDE SER USADO PARA CORREGIR EL DEFECTO GENETICO POR EXPRESAR UNA VERSION MODIFICADA DEL GEN HUMANO QUE CONSTA DE UNA FUSION DE PARTE DEL DICHO GEN Y UNA SECUENCIA SINTETICA CONTENIDA EN EL VECTOR.
ES93916425T 1992-06-02 1993-06-02 Virus adeno-asociado con secuencias terminales invertidas de repeticion como promotor para la transferencia in vivo de un gen cftr funcional. Expired - Lifetime ES2168096T3 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US07/891,962 US5587308A (en) 1992-06-02 1992-06-02 Modified adeno-associated virus vector capable of expression from a novel promoter

Publications (1)

Publication Number Publication Date
ES2168096T3 true ES2168096T3 (es) 2002-06-01

Family

ID=25399130

Family Applications (1)

Application Number Title Priority Date Filing Date
ES93916425T Expired - Lifetime ES2168096T3 (es) 1992-06-02 1993-06-02 Virus adeno-asociado con secuencias terminales invertidas de repeticion como promotor para la transferencia in vivo de un gen cftr funcional.

Country Status (11)

Country Link
US (5) US5587308A (es)
EP (2) EP1164195B1 (es)
AT (2) ATE209254T1 (es)
AU (1) AU673367B2 (es)
CA (1) CA2136441C (es)
DE (2) DE69331194T2 (es)
DK (1) DK0644944T3 (es)
ES (1) ES2168096T3 (es)
HK (1) HK1014549A1 (es)
PT (1) PT644944E (es)
WO (1) WO1993024641A2 (es)

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EP0644944B1 (en) 2001-11-21
US6165781A (en) 2000-12-26
CA2136441C (en) 2007-04-24
EP0644944A1 (en) 1995-03-29
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WO1993024641A2 (en) 1993-12-09
DE69331194D1 (de) 2002-01-03
DE69331194T2 (de) 2002-07-25
US5587308A (en) 1996-12-24
CA2136441A1 (en) 1993-12-09
US5989540A (en) 1999-11-23
AU4598193A (en) 1993-12-30
ATE209254T1 (de) 2001-12-15
US5866696A (en) 1999-02-02
DK0644944T3 (da) 2002-05-13
EP1164195B1 (en) 2010-10-06
ATE483817T1 (de) 2010-10-15
PT644944E (pt) 2002-05-31
EP1164195A3 (en) 2005-11-23
AU673367B2 (en) 1996-11-07
DE69334343D1 (de) 2010-11-18
WO1993024641A3 (en) 1994-05-11
US5990279A (en) 1999-11-23
HK1014549A1 (en) 1999-09-30

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