Lipoplexes and Polyplexes: From Gene Delivery to Gene Expression - Nanotechnology for the Delivery of Therapeutic Nucleic Acids - page 1

Chapter 1
Lipoplexes and Polyplexes: From Gene
Delivery to Gene Expression
Gerardo Byk, * Mirit Cohen-Ohana, and Fiana Mirkin
Laboratory of Nano-Biotechnology, Department of Chemistry,
Bar Ilan University, Ramat Gan 52900, Israel
* [e-mail]
1.1 Introduction
The human genome is rapidly being decoded. A map of the human
genome composed of about 30000 genes was presented some
years ago [1, 2]. Together with these findings, an increasing number
of genes have emerged that mediate both genetic and acquired
diseases. These dramatic advances make gene therapy an important
avenue for the treatment of genetic human diseases as well as major
acquired killers like cancer and AIDS. Therefore, having access to
reliable gene transfer techniques is of critical importance.
Unfortunately, even after about 25 years of research, gene
transfer is still a rather inefficient process hampered by several
limitations intrinsically paired to the specific gene delivery
approaches. A breakthrough in gene therapy clinical trials will
probably emerge from combination and application of substantially
different gene delivery techniques based on viral and synthetic
vectors.