CY1113731T1 - Μεθοδοι για δημιουργια παρασκευασματων κομιστων ανασυνδυασμενου aav υψηλου τιτλου απαλλαγμενων απο βοηθητικο ιο - Google Patents

Μεθοδοι για δημιουργια παρασκευασματων κομιστων ανασυνδυασμενου aav υψηλου τιτλου απαλλαγμενων απο βοηθητικο ιο

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Publication number
CY1113731T1
CY1113731T1 CY20131100190T CY131100190T CY1113731T1 CY 1113731 T1 CY1113731 T1 CY 1113731T1 CY 20131100190 T CY20131100190 T CY 20131100190T CY 131100190 T CY131100190 T CY 131100190T CY 1113731 T1 CY1113731 T1 CY 1113731T1
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Cyprus
Prior art keywords
aav
replication
helper virus
methods
preparations
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CY20131100190T
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English (en)
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Edward Morrow Atkinson
Victor P Fung
Perry C Wilkins
Ryan K Takeya
Thomas C Reynolds
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Genzyme Corporation
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Publication of CY1113731T1 publication Critical patent/CY1113731T1/el

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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Η εφεύρεση παρέχει μεθόδους και συνθέσεις για παραγωγή κατ'ουσίαν καθαρισμένων παρασκευασμάτων ανασυνδυασμένου αδενο-συναφούς ιού (AAV) υψηλού τίτλου τα οποία θα μπορούν να χρησιμοποιηθούν ως κομιστές για γονιδιοδιανομή. Στην αρχή της παραγωγής κομιστή, κύτταρα-AAV παραγωγοί της παρούσας εφεύρεσης κατά κανόνα περιλαμβάνουν ένα ή περισσότερα AAV γονίδια συσκευασίας, AAV κομιστή που περιλαμβάνει ετερόλογο (δηλαδή μη-AAV) διαγονίδιο ενδιαφέροντος, και βοηθητικό ιό όπως αδενοϊό. Τα παρασκευάσματα AAV κομιστή που παράγονται είναι γενικώς μη ικανά για αναδιπλασιασμό ωστόσο είναι ικανά να μεσολαβήσουν στη διανομή διαγονιδίου ενδιαφέροντος (όπως θεραπευτικού γονιδίου) σε οιοδήποτε από μία ευρεία ποικιλία ιστών και κυττάρων. Τα παρασκευάσματα AAV κομιστή που παράγονται σύμφωνα με την παρούσα εφεύρεση είναι κατ'ουσίαν απαλλαγμένα από βοηθητικό ιό καθώς και από πρωτεΐνες βοηθητικού ιού και κυτταρικές πρωτεΐνες και άλλους επιμολυντές. Επίσης παρέρχεται ποσοτική ανάλυση υψηλής ρυθμοδότησης χρήσιμη στην αξιολόγηση ιικής μολυσματικότητας και αναδιπλασιασμού, καθώς και στη διαλογή παραγόντων που επηρεάζουν την ιική μολυσματικότητα και/ή αναδιπλασιασμό.
CY20131100190T 1997-09-05 2013-03-01 Μεθοδοι για δημιουργια παρασκευασματων κομιστων ανασυνδυασμενου aav υψηλου τιτλου απαλλαγμενων απο βοηθητικο ιο CY1113731T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US92581597A 1997-09-05 1997-09-05
US7173398P 1998-01-16 1998-01-16
EP98945922A EP1009808B1 (en) 1997-09-05 1998-09-04 Methods for generating high titer helper-free preparations of recombinant aav vectors

Publications (1)

Publication Number Publication Date
CY1113731T1 true CY1113731T1 (el) 2016-06-22

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CY20131100190T CY1113731T1 (el) 1997-09-05 2013-03-01 Μεθοδοι για δημιουργια παρασκευασματων κομιστων ανασυνδυασμενου aav υψηλου τιτλου απαλλαγμενων απο βοηθητικο ιο
CY20161100051T CY1117384T1 (el) 1997-09-05 2016-01-20 Μεθοδοι για την παραγωγη παρασκευασματων ανασυνδυασμενων φορεων aav υψηλου τιτλου απαλλαγμενων απο βοηθητικους ιους

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CY20161100051T CY1117384T1 (el) 1997-09-05 2016-01-20 Μεθοδοι για την παραγωγη παρασκευασματων ανασυνδυασμενων φορεων aav υψηλου τιτλου απαλλαγμενων απο βοηθητικους ιους

Country Status (9)

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EP (3) EP2325299A3 (el)
JP (4) JP2001514845A (el)
AU (1) AU758708B2 (el)
CA (4) CA2830694C (el)
CY (2) CY1113731T1 (el)
DK (2) DK1944362T3 (el)
ES (2) ES2399232T3 (el)
PT (2) PT1944362E (el)
WO (1) WO1999011764A2 (el)

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