AAV vectors with high transduction efficiency and uses thereof for gene therapy
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsArun Srivastava, George Vladimirovich Aslanidi, Kim M. Van Vliet, Mavis Agbandje-McKenna
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Abstract
The present invention provides AAV capsid proteins comprising modification of one or a combination of the surface-exposed lysine, serine, threonine and/or tyrosine residues in the VP3 region. Also provided are rAAV virions comprising the AAV capsid proteins of the present invention, as well as nucleic acid molecules and rAAV vectors encoding the AAV capsid proteins of the present invention. Advantageously, the rAAV vectors and virions of the present invention have improved efficiency in transduction of a variety of cells, tissues and organs of interest, when compared to wild-type rAAV vectors and virions.
Claims28
An AAV VP3 protein comprising:
- a non-threonine amino acid residue at a position that corresponds to T251, T330, T454, T455, T503, T550, T592, T491, T671, T659, T660, T701, T713, or T716 of the wild-type AAV2 capsid protein of SEQ ID NO:2 wherein the non-threonine amino acid residue is selected from valine (V), aspartic acid (D), or histidine (H).
Dependencies are inferred from the claim text.