Polymer gene delivery

In order to reach the nucleus, a gene therapy vector must escape the endosome - a lipid vesicle that internally buds off from the cell membrane - or else it will be transported to the lysosome and degraded. Researchers at the Massachusetts Institute of Technology believe they have created a synthetic polymer gene delivery system that allows for such endosomal escape and gives comparable transfection as existing cationic systems, with the potential for less cytotoxicity.

The system is not ready to plug and play with clinical gene therapy compounds, however, as in vitro data for gene therapy vehicles is not necessarily predictive of in vivo results...