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AssigneesThe Regents of the University of California, Integrative Gene Therapeutics
InventorsDavid V. Schaffer, Brian Kaspar, Narendra Maheshri
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The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
An isolated nucleic acid comprising a nucleotide sequence that encodes a mutant adeno-associated virus (AAV) capsid protein, wherein the mutant AAV capsid protein comprises at least one amino acid substitution relative to a wild-type AAV capsid protein.
An adeno-associated virus (AAV) library, comprising a plurality of nucleic acids comprising a nucleotide sequence that encodes a mutant AAV capsid protein, wherein the mutant AAV capsid protein comprises at least one amino acid substitution relative to a wild-type AAV capsid protein.
A library of mutant adeno-associated virus (AAV) particles, comprising a plurality of AAV particles comprising an AAV capsid protein comprising at least one amino acid substitution relative to a wild-type AAV capsid protein.
A recombinant adeno-associated virus (rAAV) virion comprising an AAV capsid protein comprising at least one amino acid substitution relative to a wild-type AAV capsid protein;
A method of generating a mutant adeno-associated virus (AAV) particle comprising an AAV capsid protein comprising at least one amino acid substitution relative to a wild-type AAV capsid protein, wherein the mutant AAV particle exhibits an altered capsid property, the method comprising:
Dependencies are inferred from the claim text.