Gene therapy is the medical technique whereby genes are added to some specific cells for altering their functionality. This process basically is the use of genes as medicine. This can be achieved by repairing an abnormal gene through a selective reverse mutation or regulating a particular gene’s expression to ensure that the right genes are expressed at the right time. In the Gene Therapy, a functional and normal copy of an abnormal gene is introduced to a defective cell where the abnormal gene resides.
The field of Gene Therapy, though still young, is highly promising and is at the forefront of medical research. Several advances have already been made in this field. Gene Therapy dreams of treating diseases like AIDS, Cancer, Cystic Fibrosis, Muscular Dystrophy, Hemophilia, Alzheimer’s, Diabetes, Sickle Cell Anemia and many more.
There are two types of Gene Therapy – Germ Line Gene Therapy and Somatic Gene Therapy. Germ Line Gene Therapy involves the modification of reproductive cells (sperm or egg). The modification involves the introduction of normal, functional genes to the sperm or egg for making the new normal genes inheritable to the offspring and passable to the later generations. This process has not yet been tried on humans due to both technical and ethical issues. However, Germ line Gene Therapy is theoretically highly effective in fighting genetic hereditary diseases. Somatic Gene Therapy involves the introduction of therapeutic genes to the somatic (body) cells of a patient. Somatic Gene Therapy does not result in changes that can be transmitted to offspring, and the changes and effects are felt only by the patient. Somatic Gene Therapy has the limitation of lasting only for a few months at most. This makes it require regular repetition of application. Apart from treating gene-related diseases, gene therapy can be used to prevent aging by suppressing the aging process through lengthening of gene telomeres, preventing muscle loss, and bone loss (decrease in the bone’s mineral density), and reducing neuromuscular coordination and balance. This aspect of gene therapy is still in trial and is highly experimental.