Abstract

GENE THERAPY: A BOON TO MEDICAL SCIENCE

Shreya Parkar*, Rutuja Sawant, Prajakta Kegade and Akshay Gade

Abstract

The human genome contains ∼25,000 genes that encode a wide variety of proteins called the building blocks of the cell to drive every biological process necessary for life. Although a significant advancement has been made in developing modern medicine, including chemotherapy, radiation, and surgery, results into alter the body’s chemistry and create dependency over time, and offer only temporary relief by reducing disease symptoms. These issues are partly addressed by developing gene therapy. Human gene therapy is defined as the introduction of new genetic material directly into the cells of an individual to produce a therapeutic benefit. Mainly the treatment of disease is done by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for a disease. The human genome has been an objective in the medicinal field since the recognition of the gene as the basic unit of heredity. It has gained tremendous success by understanding the capacity for gene improvement employing the correction of altered (mutated) genes or site-specific modifications that have treatment as a target. It holds considerable potential for the treatment of both hereditary genetic disorders and infectious diseases. Gene therapy has become an alternative treatment for a wide range of infectious diseases. The broad field of gene therapy promises several innovative treatments that are likely to become important in preventing diseases such as fetal abnormalities, diabetes, and cancer. Till now, Twenty gene therapy products have already been approved and over two thousand human gene therapy, clinical trials have been reported worldwide. These advances raise great hope to treat rare and inherited diseases.

Keywords: gene therapy, genetic disorders, retinal, fetal.