CONTROLLED MODIFICATION OF ADENO-ASSOCIATED VIRUS (AAV) FOR ENHANCED GENE THERAPY

CONTROLLED MODIFICATION OF ADENO-ASSOCIATED VIRUS (AAV) FOR ENHANCED GENE THERAPY

The present invention discloses platforms for chemically modify AAV capsids with control over site and stoichiometry. An AAV packaging system is described that allows the introduction of site-directed natural and unnatural amino acid mutations into any subset of the three capsid proteins. These engi...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: PHAM, Quan, ERICKSON, Sarah B, KELEMEN, Rachel E, CHATTERJEE, Abhishek
Format: Patent
Sprache:eng
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:The present invention discloses platforms for chemically modify AAV capsids with control over site and stoichiometry. An AAV packaging system is described that allows the introduction of site-directed natural and unnatural amino acid mutations into any subset of the three capsid proteins. These engineered residues can be subsequently used to chemically functionalize the resulting capsids with precise control over site and stoichiometry. Such controlled modification strategy can be used to attach a wide variety of entities to AAV capsids to engineer its tropism, immunogenicity, etc.