A COMBINED ADENO-ASSOCIATED VIRUS AND ADENOVIRUS COCKTAIL GENE DELIVERY SYSTEM FOR HIGH EFFIENCY GENE EXPRESSION WITHOUT ELICITING IMMUNE RESPONSE IN IMMUNO-COMPETENT SUBJECTS

摘要

The present invention provides an efficient gene delivery system using Adeno-Associated Viral (AAV) vector in gene therapy. Furthermore, the invention provides a combined AAV and Adenovirus (Adv) cocktail gene delivery system which is even more efficient in in vivo gene delivery and expression without eliciting any significant immune responses in an immunocompetent subject. In particular, the invention provides a therapeutic agent and methods for preventing, treating, managing, or ameliorating various diseases and disorders including, but not limited to, bone diseases, by delivering Bone Morphogenetic Protein 2 (BMP-2) for new bone formation via gene therapy using said system. The invention provides a nucleic acid molecule comprising an AVV vector and a promoter operably linked to a sequence encoding BMP-2; and a nucleic acid molecule comprising an Adv vector and a promoter operably linked to a sequence encoding BMP-2, as well as vectors and host cells comprising said nucleic acid molecules, respectively.