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Updated: Aug 12, 2026

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
Published on: July 23, 2016
Gene transfer with lipospermines and polyethylenimines
1Laboratoire de Physiologie Générale et Comparée, U.R.A.90 CNRS, Museum National d'Histoire Naturelle, F-75231, Paris Cedex 5, France
Abstract:
It is an obvious and basic principle that to be efficient, gene therapy requires effective gene transfer followed by adequate gene expression. However, getting DNA, a pro-drug, into the cell and into the nucleus, remains a crucially limiting factor. Even recombinant viral methods still show poor performances in clinical situations and non-viral methods are considered classically to be of yet lower efficiency. Here, we consider the mode of action, the nature of the complexes formed with DNA and the transfection potentials of two categories of inert, cationic vectors, the lipospermines and polyethylenimine. Both are among the best vectors currently available for in vitro work. Moreover, polyethylenimine is proving to be a versatile and effective carrier for different in vivo situations, especially for delivering genes into the mammalian brain.

