Gene Therapy

Abstract

Genes are the blueprints for proteins and serve as building blocks for tissues. Genes are regulators of chemical reactions inside the living cells. Mutation in a gene causes a change in the expressed structure of protein leading to protein dysfunction. Genetic errors disrupt gene expression and result in diseases. Advancement in biotechnology has helped in understanding the genetic basis of inherited diseases. Gene therapy is an experimental technique that uses genes as medicine for correcting defective genes that are responsible for disease development. Gene therapy has the potential to eliminate a wide range of inherited and acquired human diseases. A number of clinical trials in gene therapy are being carried out throughout the world. Gene therapy approach involves the treatment of disease by introducing new genetic instructions into the tissues of patients to correct the defective or abnormal gene. In gene therapy, mostly somatic (nonsex) cells are targeted for treatment. Due to controversies associated with the involvement of germ cells, this therapy is not attempted. As somatic gene therapy is directed at the individual, it has no impact on future generations. Changes directed at somatic cells are not inherited. The problem of “gene delivery” i.e., the need to get replacement genes into the desired tissues is the most difficult part and needs intensive research. The majority of current gene therapy clinical trials involve the mechanisms of viruses as “vectors”. These viral vectors are capable of integrating at random sites in the host’s cells. To date, gene therapy is being used to treat several genetic disorders. A large number of clinical trials are underway to test gene therapy as a treatment of choice for many life-threatening diseases.