Patent Number: 6,428,988

Title: Methods using cre-lox for production of recombinant adeno-associated viruses

Abstract: Methods for efficient production of recombinant AAV are described. In one aspect, three vectors are introduced into a host cell. A first vector directs expression of cre recombinase, a second vector contains a promoter, a spacer sequence flanked by loxP sites and rep/cap, and a third vector contains a minigene containing a transgene and regulatory sequences flanked by AAV ITRs. In another aspect, the host cell stably or unducibly expresses cre recombinase and two vectors carrying the other elements of the system are introduced into the host cell.

Inventors: Wilson; James M. (Gladwyne, PA), Phaneuf; Daniel (Philadelphia, PA)

Assignee: The Trustees of the University of Pennsylvania

International Classification: C07K 14/015 (20060101); C07K 14/005 (20060101); C12N 15/864 (20060101); C12N 9/00 (20060101); C12N 15/861 (20060101); A61K 48/00 (20060101); C12N 015/63 (); C12N 015/64 ()

Expiration Date: 08/06/2019