Gene therapy has emerged as a new concept of therapeutic strategies to trea
t diseases which do not respond to the conventional therapies. The principl
e of gene therapy is to introduce genetic materials into patient cells to p
roduce therapeutic proteins in these cells. Gene therapy is now at the stag
e where a number of clinical trials have been carried out to patients with
gene-deficiency disease or cancer. Genetic materials for gene therapy are g
enerally composed of gene expression system and gene delivery system. For t
he clinical application of gene therapy in a way which conventional drugs a
re used, researches have been focused on the design of gene delivery system
which can offer high transfection efficiency with minimal toxicity. Curren
tly, viral delivery systems generally provide higher transfection efficienc
y compared with non-viral delivery systems while non-viral delivery systems
are less toxic, less immunogenic and manufacturable in large scale compare
d with viral systems. Recently, novel strategies towards the design of new
non-viral delivery system, combination of viral and non-viral delivery syst
ems and targeted delivery system have been extensively studied. The continu
ed effort in this area will lead us to develop gene medicine as "gene as a
drug'' in the near future.