Gene Therapy

Gene therapy Gene therapy is a powerful new technology that can change the future way of medical care. The potential of gene therapy offers great promise for healing and relieving hereditary disorders currently plaguing myriad people. In the past decade many studies have been done to understand all aspects of gene therapy, but there are still many things to learn before it becomes an effective treatment. Although clinical efficacy has not been demonstrated, many gene therapists predict that initial clinical success will occur in the near future.

Gene therapy uses DNA as a drug for treating diseases. Gene therapy was originally conceptualized in 1972 and the authors urged attention before starting gene research on humans. The first FDA approved gene therapy trial in the US was held in 1990. This is a 4 year old girl named Ashanti DeSilva who received ADA - SCID treatment. This is a disease that prevents her from fighting infection. Dr. W French Anderson is the principal investigator of clinical trials and works at the National Heart, Lung and Blood Laboratory. Since then, more than 1,700 clinical trials have been performed using a variety of gene therapy technologies.

The first clinical trial of gene therapy was done in 1990 by the National Institutes of Health in Washington, DC, and it was done dozens of times thereafter. Until this year, the first gene therapy began at the clinical trial stage and received the first gene therapy approved by the Federal Drug Administration (FDA). Novartis' Kimuria (tisagenlecleucel) approved on August 30, 2017 is a remedy for children and young patients with certain acute lymphoblastic leukemia (ALL). It is T cell immunotherapy, which means that each dose is a patient's own T cell, a customized treatment made using immune cells found in the blood.