CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY

CAPSID-FREE AAV VECTORS, COMPOSITIONS, AND METHODS FOR VECTOR PRODUCTION AND GENE DELIVERY

An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid m...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: VOIT, Thomas, BELEY, Cyriaque, KOTIN, Robert Michael, GARCIA, Luis, LI, Lina
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Beschreibung
Zusammenfassung:An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.