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 that comprises at least a first modified capsid protein, and wherein said particle further comprises at least a first nucleic acid segment that encodes a therapeutic agent operably linked to a promoter capable of expressing said segment in a host cell.
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