Targeted delivery of genes

Abstract

In the past few years, various gene delivery vehicles have been designed in order to make a vector effective for gene therapy application. Both viral and non-viral vectors have been used extensively as biological carriers for the transfer of foreign genes into living cells. But these vectors suffer from several draw backs which limit there use in future clinical application while using "gene therapy" for the treatment of several inherited disorders. The reconstituted Sendai viral envelopes, also known as virosomes are able to successfully deliver genes and other biologically active macromolecules to various cells in culture. Although such virosomes are known to fuse efficiently with the plasma membrane of target cells and served as excellent carriers for the fusion-mediated transfer on DNA, RNA, toxins, drugs, etc. into viable cultured cells, they result in non-specific delivery because of the presence of hemagglutinin protein (HN) and hence can not be used in vivo. On the other hand, reconstituted Sendai viral envelope devoid of the HN protein (F-virosomes) can fuse efficiently and preferentially with liver cells (both in vitro and in vivo) and deliver its contents to the cytosol of these cells. We conclude that this kind of targeted delivery can further be used for a wide variety of gene transfer strategies both In vitro and in vivo in the field of "gene therapy".