DNA CONSTRUCTS FOR LONG-TERM EXPRESSION OF INTRAVASCULARLY INJECTED NAKED DNA

摘要

Hydrodynamic intravascular injection of recombinant DNA molecules that can be expressed in the liver provides sustained and/or high-level expression of genes in a simple, safe, inexpensive, and reproducible fashion. These DNA molecules include a first sequence with the promoter of a liver-expressed gene operably linked to a second sequence encoding a protein of interest. The DNA molecules can be expressed in vivo to produce functionally active proteins. Methods of the invention provide tools to study these proteins in vivo and therapeutic applications for the expressed DNA molecules.