Modified adeno-associated virus 5 capsids and uses thereof

    公开(公告)号:US12209251B2

    公开(公告)日:2025-01-28

    申请号:US17307117

    申请日:2021-05-04

    Abstract: The present invention provides mutant adeno-associated virus serotype 5 (AAV5) that exhibit altered capsid properties, e. g., increased infectivity in human liver cells and/or minimal binding to human neutralizing antibodies. The present invention further provides libraries of mutant AAV5 comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV5 and mutant AAV5 libraries, and compositions comprising the mutant AAV5. The present invention further provides recombinant AAV5 (rAAV5) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins.

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