DK2573170T3 - Sekvenser af adenoassocieret virus (AAV) serotype 9, vektorer indeholdende disse, og brug heraf - Google Patents

Sekvenser af adenoassocieret virus (AAV) serotype 9, vektorer indeholdende disse, og brug heraf Download PDF

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DK2573170T3
DK2573170T3 DK12163949.6T DK12163949T DK2573170T3 DK 2573170 T3 DK2573170 T3 DK 2573170T3 DK 12163949 T DK12163949 T DK 12163949T DK 2573170 T3 DK2573170 T3 DK 2573170T3
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Guangping Gao
Mauricio Alvira
James N Wilson
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Univ Pennsylvania
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    • 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
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    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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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  • Wood Science & Technology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Claims (11)

1. Et rekombinant adenoassocieret virus (AAV) omfattende en AAV9-kapsid med aminosyresekvensen SEQ ID NO:2 eller en aminosyresekvens, der er mindst 95% identisk hermed, idet nævnte rekombinante AAV desuden omfatter et minigen med AAV ITR (inverted terminal repeats) og et transgen knyttet til regulatorsekvenser, som fører det til aktivering i en værtscelle.
2. Det rekombinante AAV ifølge krav 1, idet aminosyresekvensen af nævnte kapsid er mindst 99% identisk med aminosyrer 1 til 736 med SEQ ID NO: 2.
3. Det rekombinante AAV ifølge krav 2, idet den nævnte kapsid har aminosyresekvensen SEQ ID NO: 2.
4. Det rekombinante AAV ifølge krav 1, idet ITR er fra en AAV2.
5. Det rekombinante AAV ifølge et hvilket som helst af kravene 1 til 4, idet transgenet er beregnet til behandling af lysosomal sygdom.
6. Det rekombinante AAV ifølge et hvilket som helst af kravene 1 til 4, idet transgenet er beregnet til behandling afkræft.
7. Det rekombinante AAV ifølge et hvilket som helst af kravene 1 til 4, idet transgenet består af et polypeptid omfattende en variabel region af et antistof.
8. Det rekombinante AAV ifølge et hvilket som helst af kravene 1 til 4, idet transgenet er valgt fra alfa-1-antitrypsin (Al AT), rhCG og faktor IX (FIX).
9. En sammensætning omfattende det rekombinante AAV ifølge et hvilket som helst af de foregående krav og et fysiologisk foreneligt bærestof.
10. Et isoleret adenoassocieret virus (AAV) omfattende en AAV9-kapsid med aminosyresekvensen SEQ ID NO:2 eller en aminosyresekvens, der er mindst 95% identisk hermed.
11. Det rekombinante AAV ifølge krav 10, idet det nævnte virus omfatter nukleinsyresekvensen SEQ ID NO:l.
DK12163949.6T 2001-12-17 2002-11-12 Sekvenser af adenoassocieret virus (AAV) serotype 9, vektorer indeholdende disse, og brug heraf DK2573170T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US34115001P 2001-12-17 2001-12-17
US38613202P 2002-06-05 2002-06-05
EP02793807.5A EP1463805B1 (en) 2001-12-17 2002-11-12 Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor

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DK2573170T3 true DK2573170T3 (da) 2018-04-09

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DK12163949.6T DK2573170T3 (da) 2001-12-17 2002-11-12 Sekvenser af adenoassocieret virus (AAV) serotype 9, vektorer indeholdende disse, og brug heraf
DK02793807.5T DK1463805T3 (da) 2001-12-17 2002-11-12 Sekvenser af adenoassocieret virus (AAV) serotype 9, vektorer indeholdende disse samt deres anvendelser

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US (1) US7198951B2 (da)
EP (3) EP1463805B1 (da)
JP (1) JP4769417B2 (da)
AU (1) AU2002359284A1 (da)
CA (1) CA2469053C (da)
CY (1) CY1120209T1 (da)
DK (2) DK2573170T3 (da)
ES (2) ES2526341T3 (da)
PT (1) PT2573170T (da)
WO (1) WO2003052052A2 (da)

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