Gene Therapy from labs to clinics

Scientists have been working for several years to bring gene therapy a clinical reality. In a boosting move, the Food & Drug Administration (FDA) has approved gene therapy for clinical use in a rare form of child blindness. It is the first such treatment to receive United States approval for an inherited disease. In previous years, the FDA accepted two other treatments through gene therapy process. The process targets blood cancers patients which involves extraction of immune cells from blood, correcting them genetically in the lab to check cancer and replacing them in the patient’s body. The novel gene-therapy has now turned from promises to cures.

Human genes hold coded information for production of specific protein in the body which is core to the health of an individual.  In most cases of disease or other problems, there is no one single cause. The disease is cause of performance of genes, the environment and choices made by the person. Many of the diseases as Immune issues, blood disorders, Cancer, metabolism disorders are most of the time are due to genetic disorders i.e mutations. Gene therapy is a revolutionary way to fix genetic disorders in a body.

The gene therapy process is based on insertion of genes into patient’s body cells and tissues to treat a disease, or hereditary diseases in which mutant gene is replaced or edited to bring it to proper functionality. The process may include replacing the mutated/defected gene that causes disease with a healthy copy of the functional gene; or inactivating a mutated gene, functioning improperly; or introducing a new gene into the body to help fight a disease.

There are number of successful trail case studies to support this revolutionary therapy. Developed in 1972 as a medical technique, the first gene therapy clinical trial was conducted by William French Anderson and Michael Blaese in 1990 at the NIH clinical Center. On 14 September 1990, first successful gene therapy performed on Ashi DeSilva to treat ADA-SCID. Between 1989 and 2015; over 2,300 clinical trials has been conducted and many of them has been successful.

Eventually, gene therapy will become a staple of 21st century medicine brining rescue from 4,000 medical conditions resulting from gene disorders. If some of these genetic disorders can be corrected through gene therapy, individuals suffering from these diseases may recover to enjoy longer & healthier lives. The positive aspect of gene therapy is long lasting.

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