Widespread regional myocardial transfection by plasmid encoding Del-1 following retrograde coronary venous delivery.

This study quantifies myocardial transfection following percutaneous retrograde coronary venous delivery (RCVD) of a plasmid encoding human Del-1. RCVD of Del-1, GFP plasmid, or marker dye was conducted in 14 pigs. After selective cannulation of a coronary vein, a delivery site was confirmed by contrast injection and myocardial blush.

Poly-L-glutamate, an anionic polymer, enhances transgene expression for plasmids delivered by intramuscular injection with in vivo electroporation.

Intramuscular (i.m.) injection of plasmids followed by electropermeabilization is an efficient process to deliver genes into skeletal myofibers that permits proteins to be produced and secreted at therapeutically relevant levels.

Gene therapy for the treatment of hemophilia B using PINC-formulated plasmid delivered to muscle with electroporation.

Gene therapy, as a safe and efficacious treatment or prevention of diseases, is one of the next fundamental medical innovations. Direct injection of plasmid into skeletal muscle is still a relatively inefficient and highly variable method of gene transfer. However, published reports have shown that application of an electric field to the muscle immediately after plasmid injection increases gene expression at least 2 orders of magnitude.

Muscle-specific enhancement of gene expression by incorporation of SV40 enhancer in the expression plasmid.

Skeletal muscle is established as an ideal tissue for gene delivery to treat systemic diseases. However, the relatively low levels of gene expression obtained from using naturally occurring promoters, including the strong cytomegalovirus (CMV) enhancer/promoter (E/P), have limited the use of muscle as a target tissue. The relatively weak simian virus 40 (SV40) enhancer is known to have dual functions promoting localization of DNA to the nucleus and activating transcription.