The present article is an overview of gene therapy with an emphasis on diff
erent approaches and its implications in the clinic. Genetic interventions
have been applied to the diagnosis of and therapy for an array of human dis
eases. The initial concept of gene therapy was focused on the treatment of
genetic diseases. Subsequently, the field of gene therapy has been expanded
, with a major focus on cancer. Although the results of early gene therapy-
based clinical trials have been encouraging, there is a need for gene deliv
ery vectors that feature reduced immunogenicity and improved targeting abil
ity. The results of phases I/II clinical trials have suggested the importan
t role of gene therapy as a versatile and powerful treatment tool, especial
ly for human cancers. One reasonable expectation is that performing gene th
erapy at an earlier stage in the disease process or for minimal residual di
sease may be more advantageous. (C) 2001 Elsevier Science Inc. All rights r
eserved.