Gene therapy was initially conceived to cure monogenetic disorders, however the spectrum of gene therapeutic approaches is widening very rapidly to include everything from simple genetic disorders to acquired diseases like cancer, human immune deficiency virus (HIV), and hepatitis B virus. Somatic gene therapy has been well accepted among the general public, whereas germline gene therapy is facing ethical dilemmas (106).
Before gene therapy can move from benches to beds, there are a number of technical obstacles to overcome Isolation and characterization of therapeutic genes necessitates extensive study about their regulation and stability after transfer into the genome. Efficient mechanisms for gene insertion and targeting to particular cells need more scientific endeavors. Finally, expression of the gene, and encounter of the gene product with immune system, need to be well understood before transfer.
Gene therapy is a rapidly growing field of research. Besides those described in this article, a number of other human disorders are being explored at the molecular level. The genes responsible for hearing loss syndrome have been identified (107). Gene therapy also seems to be the treatment of choice for many complex hematological and lysosomal storage disorders (108,109). Considerable evidence has accrued to deduce that behavioral changes like alcoholism are also under the influence of certain genes (110). It has been suggested that the characterization of such genes will help in the understanding of human behavioral mechanisms.
The secret of ideal and effective gene therapy lies in the development of delivery systems with high transduction efficiency, low cost, tissue specificity, sustained action of transgenes, and site-specific integration into the human genome without deleterious effects. This will materialize the dream of gene therapeutic approaches for controlling inherited and noninherited complex disorders.
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