Capsid-modified rAAV vectors and methods of use
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsLi Zhong, Sergei Zolotukhin, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, Arun Srivastava
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Abstract
Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
Claims23
A recombinant adeno-associated viral (rAAV) particle comprising a mutated capsid protein, wherein the mutated capsid protein comprises a non-tyrosine amino acid at a position that corresponds to Tyr444 in a wild-type AAV2 capsid protein.
A recombinant adeno-associated viral (rAAV) particle comprising a mutated capsid protein, wherein the mutated capsid protein comprises a non-tyrosine amino acid at a position that corresponds to Tyr730 in a wild-type AAV2 capsid protein.
A recombinant adeno-associated viral (rAAV) particle comprising a mutated capsid protein, wherein the mutated capsid protein comprises a non-tyrosine amino acid at a position that corresponds to Tyr500 in a wild-type AAV2 capsid protein.
Dependencies are inferred from the claim text.