CA2870736C - Composition and methods for highly efficient gene transfer using aav capsid variants - Google Patents

Composition and methods for highly efficient gene transfer using aav capsid variants Download PDF

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Publication number
CA2870736C
CA2870736C CA2870736A CA2870736A CA2870736C CA 2870736 C CA2870736 C CA 2870736C CA 2870736 A CA2870736 A CA 2870736A CA 2870736 A CA2870736 A CA 2870736A CA 2870736 C CA2870736 C CA 2870736C
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aav
nucleic acid
aav vector
vector
capsid protein
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French (fr)
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CA2870736A1 (en
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Mustafa N. Yazicioglu
Federico Mingozzi
Xavier ANGUELA
Katherine A. High
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Childrens Hospital of Philadelphia CHOP
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
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  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
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  • Communicable Diseases (AREA)
  • Diabetes (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
CA2870736A 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants Active CA2870736C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261635273P 2012-04-18 2012-04-18
US61/635,273 2012-04-18
US201361794995P 2013-03-15 2013-03-15
US61/794,995 2013-03-15
PCT/US2013/037170 WO2013158879A1 (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

Publications (2)

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CA2870736A1 CA2870736A1 (en) 2013-10-24
CA2870736C true CA2870736C (en) 2021-11-02

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CA2870736A Active CA2870736C (en) 2012-04-18 2013-04-18 Composition and methods for highly efficient gene transfer using aav capsid variants

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US (2) US9909142B2 (h)
EP (1) EP2839014B1 (h)
JP (1) JP6342886B2 (h)
KR (1) KR102063483B1 (h)
CN (1) CN104487579B (h)
AU (1) AU2013249202B2 (h)
BR (2) BR122021020419B1 (h)
CA (1) CA2870736C (h)
CO (1) CO7200251A2 (h)
DK (1) DK2839014T3 (h)
ES (1) ES2862912T3 (h)
HU (1) HUE054087T2 (h)
IL (1) IL235102B (h)
IN (1) IN2014DN08812A (h)
MX (1) MX359518B (h)
MY (1) MY172457A (h)
NZ (1) NZ701693A (h)
PE (1) PE20150163A1 (h)
PH (1) PH12014502347B1 (h)
PL (1) PL2839014T3 (h)
PT (1) PT2839014T (h)
RU (1) RU2683497C2 (h)
SG (1) SG11201406776TA (h)
SI (1) SI2839014T1 (h)
WO (1) WO2013158879A1 (h)
ZA (1) ZA201407582B (h)

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