Targeted Genetics Corporation

Haim Burstein

Anthony M Stepan

Carmel M Lynch

Dara H Lockert

Anthony M Stephan

Isolated recombinant polynucleotides comprising elements which promote encapsidation into AAV particles, packaging cells comprising the recombinant polynucleotides, and methods for their use are provided in the present invention. These isolated recombinant polynucleotides comprise a non-AAV ITR encapsidation element (such as the P1 sequence located within the AAV S1 integration site of human chromosome 19) operably linked to one or more heterologous genes to be encapsidated. The constructs may be either integrated into a mammalian cell genome, maintained episomally, or provided transiently.

Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles

High-efficiency AAV packaging constructs and methods for their use are provided. in the present invention. These high-efficiency packaging constructs comprise an activating element (such as the P1 sequence located within the AAV S1 integration site of human chromosome 19) amplifiably linked to one or more AAV packaging genes. The constructs may be either integrated into a mammalian cell genome or maintained episomally. Use of the high-efficiency AAV packaging vectors of the invention provides for controlled amplifiable production of rAAV vector constructs.

Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors

The present invention provides recombinant adeno-associated virus (rAAV) vectors encoding a tumor necrosis factor (TNF) antagonist and methods using these vectors to reduce levels of TNF in a mammal. The invention also provides methods of using these rAAV vectors in palliating TNF-associated disorders.

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Redmond, WA, United States of America

Haim Burstein