GENE-TRANSFER WITH LIPOSPERMINES AND POLYETHYLENIMINES

It is an obvious and basic principle that to be efficient, gene therap y requires effective gene transfer followed by adequate gene expressio n. However, getting DNA, a pro-drug, into the cell and into the nucleu s, remains a crucially limiting: factor. Even recombinant viral method s still show poor performances in clinical situations and non-viral me thods are considered classically to be of yet lower efficiency. Here, we consider the mode of action, the nature of the complexes formed wit h DNA and the transfection potentials of two categories of inert, cati onic vectors, the lipospermines and polyethylenimine. Both are among t he best vectors currently available for in vitro work. Moreover, polye thylenimine is proving to be a versatile and effective carrier for dif ferent in vivo situations, especially for delivering genes into the ma mmalian brain. (C) 1998 Elsevier Science B.V.