Gene Therapy: A Veracity or Myth.

ABSTRACT

Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s gene. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Thus it may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favorably modify the clinical course of a condition. The scope of this new approach to the treatment of a condition is broad, with potential in the treatment of many genetic conditions. Though single gene disorders are best treated than multifactorial disorder; the challenge of developing successful gene therapy for any specific condition is considerable. The problem of ‘gene delivery’ into the desired tissues is very complex and challenging. Some of the ‘vectors’ for delivering the working copy of the gene to the target cells include using harmless viruses and non viral vectors. Till date, in gene therapy, only somatic cells and not the germ cells are targeted for treatment. The possible genetic manipulation of the germ cells remains the subject of intense ethical and philosophical discussion. Though some devastation was recorded in gene therapy trial; the potential benefits of new treatments must always be balanced against such risks. In particular, safety will appropriately remain an important consideration as the field of gene therapy evolves. The purpose of this review is to focus on merit and demerit of gene therapy and to provide information about its future prospective.