The absorption enhancer sodium deoxycholate promotes high gene transfer in skeletal muscles.

Gene delivery to skeletal muscle is a promising strategy for the treatment of muscle disorders and for the systemic secretion of therapeutic proteins. In addition, muscle is an attractive target tissue because it is easily accessible. However, very few synthetic vectors proved capable of surpassing naked DNA mediated muscle gene transfer.

The reverse block copolymer Pluronic 25R2 promotes DNA transfection of skeletal muscle.

Muscle is an important and attractive target for gene therapy. Recent findings have shown that neutral amphiphilic triblock copolymers with a PEO-PPO-PEO arrangement significantly increase muscle transfection as compared to naked DNA. We were interested in evaluating whether reverse Pluronics (PPO-PEO-PPO) also possess transfection properties.

Evaluation of the muscle gene transfer activity of a series of amphiphilic triblock copolymers.

Background: Amphiphilic triblock copolymers such as the polyethylene oxide-polypropylene oxide-polyethylene oxide L64 (PEO(13)-PPO(30)-PEO(13)) significantly increase transgene expression after injection of DNA/polymer mixtures into skeletal muscles. To better understand the way such copolymers act, we studied the behaviour of different poloxamers, including L64, both in vitro and in vivo.

Methods: The in vitro and in vivo transfection activity of five copolymers that differ either by their molecular weight or by their hydrophilic/hydrophobic balance was evaluated.