Avigen, Inc.

Other

Johns Hopkins University

Genzyme Corporation

Genovo, Inc.

Gary J Kurtzman

Gregory M Podsakoff

Paul D Kessler

Barry J Byrne

Peter C Colosi

Hideho Okada

Georges Natsoulis

Masaaki Mizuno

Jun Yoshida

Dirk G Brockstedt

Greg M Podsakoff

Edgar G Engelman

Haifeng Chen

George Natsoulis

The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof from transduced muscle cells such that systemic delivery is achieved.

Methods of treating anemia using recombinant adeno-associated virus virions

The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.

Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions

Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors

This invention relates to novel adenoviruses useful in the production of high titers of recombinant adeno-associated virus (rAAV) comprising a foreign DNA insert and methods of making these adenoviruses. The adenovirus comprises the AAV rep gene in which the p5 promoter is replaced by a minimal promoter or by no promoter. The invention also provides methods of producing high levels of rAAV as a substantially homogenous preparation and composition of rAAV.

Production of recombinant AAV using adenovirus comprising AAV rep/cap genes

The present invention relates generally to immunization methods using recombinant viral vectors. In particular, the invention relates to methods and compositions for immunizing a subject with a nucleic acid molecule encoding an antigen of interest, wherein the nucleic acid molecule is delivered to the subject via a recombinant AAV virion.

Induction of immune response to antigens expressed by recombinant adeno-associated virus

Adeno associated virus vectors for the treatment of a cardiomyopathy

The use of recombinant adeno-associated virus (AAV) virions for the treatment of solid tumors is disclosed. The invention provides for the use of recombinant AAV virions to deliver an AAV vector containing a drug-susceptibility gene and a second gene capable of providing an ancillary effect to solid tumor cells. The second gene can be used to enhance the immunogenicity of the transduced tumor cell. Alternatively, the second gene can be used to provide a tumorstatic effect. The invention also provides for the use of recombinant AAV virions to deliver an interferon gene, or a tumor suppressor gene to provide a therapeutic effect in a transduced tumor cell.

Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors

Methods for delivering DNA to the bloodstream using recombinant adeno-associated virus vectors

The present invention provides methods and compositions for producing high titer preparations of recombinant AAV (&ldquo;rAAV&rdquo;) virions. The compositions of the present invention include AAV helper function systems and host cells. The present invention also includes methods of using AAV helper function vectors that effect the production of only small amounts of the long forms of Rep protein, and rAAV virions produced by such methods.

High-efficiency AAV helper functions

Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions to treat lysosomal storage disease

Methods of treating disease using recombinant adeno-associated virus virions administered to muscle

The present invention provides methods and compositions for producing high titer preparations of recombinant AAV ('rAAV') virions. The compositions of the present invention include AAV helper function systems and host cells. The present invention also includes methods of using AAV helper function vectors that effect the production of only small amounts of the long forms of Rep protein, and rAAV virions produced by such methods.

High-efficiency AA V helper functions

Growing community of inventors

Menlo Park, CA, United States of America

Gary J Kurtzman