METHODS OF GENE DELIVERY USING CAPSID-MODIFIED RAAV EXPRESSION SYSTEMS
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United States of America Patent
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Issued Date -
Jan 1, 2015
app pub date -
Jul 8, 2014
filing date -
May 1, 2002
priority date (Note) -
Abandoned
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Abstract
Disclosed are methods of gene delivery using capsid-modified recombinant adeno-associated viral (rAAV) vectors. Exemplary methods are provided employing vectors that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are methods employing the rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
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