Online edition:ISSN 2758-089X

Human Gene Therapy: A Medicine of the Future

The remarkable progress which has been made in the field of gene transfer technology during the last five years clearly indicates that gene therapy hold a considerable promise for the future. The modes of gene delivery to appropriate target cells have been narrowd down and out of both viral and nonviral methods of gene transfer, adenovectors have produced the best results. Most modern gene-therapeutic approaches are now based on the introduction of functional copies of defective genes into cells and a large number of target cells and tissues are being explored for various gene therapy applications. Successful gene therapy trials in a few selected genetic disorders have shown a long-term stable expression of transfered genes. Somatic gene manipulation although has shown clear advantages over germ-line therapy but it has not yet entered into clinical scale as the better understanding of somatic cell transplantation is still inadequate. A massive international efforts are being geared toward gene therapy research but there are still many technical difficulties e.g. isolation of genes and their regulatory regions, selection of best possible gene delivery systems etc, need to be overcome. All the indications are that the gene therapy will be perhaps a clinical reality in the early part of next century but the important question is safety of patients and the efficacy of gene transduction. Ethical considerations of gene therapy can not be overlooked. Opposition undoubtedly will occur in some cases because of potential to manipulate the genetic framework of future generations but eventually the luminous promise of gene therapy, probably will outweigh the potential pitfalls.

Niazi G, et al