CA2826316C - Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer - Google Patents

Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer Download PDF

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Publication number
CA2826316C
CA2826316C CA2826316A CA2826316A CA2826316C CA 2826316 C CA2826316 C CA 2826316C CA 2826316 A CA2826316 A CA 2826316A CA 2826316 A CA2826316 A CA 2826316A CA 2826316 C CA2826316 C CA 2826316C
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aav
aav9
galactose
vector
cells
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CA2826316A1 (en
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James M. Wilson
Christie L. Bell
Luc H. Vandenberghe
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • A61K31/00Medicinal preparations containing organic active ingredients
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    • AHUMAN NECESSITIES
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/66Phosphorus compounds
    • A61K31/683Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
    • A61K31/685Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
    • C12N2750/14045Special targeting system for viral vectors
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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CA2826316A 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer Active CA2826316C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161443879P 2011-02-17 2011-02-17
US61/443,879 2011-02-17
PCT/US2012/025550 WO2012112832A1 (en) 2011-02-17 2012-02-17 Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer

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CA2826316A1 CA2826316A1 (en) 2012-08-23
CA2826316C true CA2826316C (en) 2021-04-27

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US (5) US9884071B2 (enExample)
EP (1) EP2675902B1 (enExample)
JP (1) JP6224459B2 (enExample)
KR (1) KR101956885B1 (enExample)
CN (1) CN103502458B (enExample)
AU (1) AU2012219380B2 (enExample)
BR (1) BR112013020734A2 (enExample)
CA (1) CA2826316C (enExample)
DK (1) DK2675902T3 (enExample)
ES (1) ES2724800T3 (enExample)
HU (1) HUE043921T2 (enExample)
MX (1) MX353930B (enExample)
PL (1) PL2675902T3 (enExample)
SG (3) SG10202007803QA (enExample)
TR (1) TR201908020T4 (enExample)
WO (1) WO2012112832A1 (enExample)

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