Chemistry: molecular biology and microbiology – Treatment of micro-organisms or enzymes with electrical or... – Modification of viruses
Patent
1995-06-07
1997-08-19
Guzo, David
Chemistry: molecular biology and microbiology
Treatment of micro-organisms or enzymes with electrical or...
Modification of viruses
4353201, 435 914, 435352, 435363, 435366, 435367, 435369, 435371, C12N 516, C12N 1564, C12N 1586
Patent
active
056587767
ABSTRACT:
Adeno-associated virus (AAV) vectors may have utility for gene therapy but heretofore a significant obstacle has been the inability to generate sufficient quantifies of such recombinant vectors in amounts that would be clinically useful for human gene therapy application. Stable, helper-free AAV packaging cell lines have been elusive, mainly due to the activities of Rep protein, which down-regulates its own expression and reverses cellular immortalization. This invention provides packaging systems and processes for packaging AAV vectors that efficiently circumvent these problems by replacing the AAV p5 promoter with a heterologous promoter and that allow for substantially increased packaging efficiency.
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Carter Barrie J.
Flotte Terence R.
Guggino William B.
Solow Rikki
Guzo David
Johns Hopkins University
Targeted Genetics Corporation
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