Other

Thomas Jefferson University

Joseph E Rabinowitz

James M Wilson

Walter J Koch

Patrick Most

Sven T Pleger

Susan M Faust

A CpG-modified recombinant adeno-associated viral (AAV) vector is described. The vector carries a nucleic acid molecule comprising AAV inverted terminal repeat (ITR) sequences and an exogenous gene sequence under the control of regulatory sequences which control expression of the gene product, in which the nucleic acid sequences carried by the vector are modified to significantly reduce CpG di-nucleotides such that an immune response to the vector is reduced as compared to the unmodified AAV vector. Also provided are methods and regimens for delivering transgenes using these AAV viral vectors, in which the innate immune response to the vector and/or transgene is significantly modulated.

Constructs and methods for delivering molecules via viral vectors with blunted innate immune responses

The invention provides nucleic acid segments, compositions and methods for the treatment of heart failure, vascular dysfunction, endothelial dysfunction, diabetes, [Ca]i regulation and NO synthase dysfunction. Adeno-associated and adenovirus are used as gene delivery vectors for the nucleic acid segments to product long term over-expression of S100A1, a small calcium sensing protein associated with the disclosed ailments and dysfunctions.

Tissue specific gene therapy treatment

Growing community of inventors

Elkins Park, PA, United States of America

Joseph E Rabinowitz