Method of increasing the function of an aav vector
AssigneeThe Trustees of the University of Pennsylvania
InventorsLuk Vandenberghe, Guangping Gao, James M. Wilson
Life of this patent
Estimating the term…
Abstract
A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
Claims15
A recombinant adeno-associated virus (AAV) comprising an AAV capsid and a minigene having AAV inverted terminal repeats and a heterologous gene operably linked to regulatory sequences which direct expression of the heterologous gene in a host cell, wherein the AAV capsid comprises AAV vp1 proteins, AAV vp2 proteins, and AAV vp3 proteins, wherein the AAV vp3 proteins have i) the sequence of amino acids 204 to 738 of SEQ ID NO:
- 4 with an N529H modification, or ii) an amino acid sequence having at least 95% identity to the full length of amino acids 204 to 738 of SEQ ID NO:4, wherein the amino acid residue corresponding to position 529 in SEQ ID NO:
- 4 is H and the amino acid corresponding to position 665 in SEQ ID NO:
- 4 is N when aligned along the full length of amino acids 204 to 738 of SEQ ID NO:
- 4.
Dependencies are inferred from the claim text.