The Start of Gene Therapy

Although the concept of gene therapy began in the 1960s and early 1970s, the first approved gene therapy trial took place in 1990.

Gene therapy is used to treat genetic diseases at a molecular level to correct defective genes. It can stop a protein from causing harm, restore a protein's function, or intensify the existing function of a protein. The therapy is based on the ability to carry the correct genes to those that are affected.

The first gene therapy trial treated a four-year-old girl in September 1990 at the NIH Clinical Center. She was diagnosed with adenosine deaminase deficiency, a genetic disease that made her defenseless when faced with infections. To treat the girl, doctors removed her white blood cells, grew them in a lab, inserted the missing gene for creating adenosine deaminase, and finally put the cells back into her bloodstream. Laboratory results showed that the therapy strengthened her immune system by 40%.


After this incredible milestone, the future was looking bright for gene therapy. However, in 1999, 18-year-old Jesse Gelsinger suffered a negative immune reaction to the genetic treatment and became the first to die from this type of therapy.

Due to this negative news, gene therapy programs were shut down and the FDA criticized the trial's design.

When gene therapy finally began making a return, it did so gradually and with caution. China was the first nation to approve Gendicine, a gene therapy for neck and head cancer. To this day, there remain challenges with the therapy in terms of its practicality. However, there have been a number of recent approvals in gene therapy approvals, and many more that are currently in the process of testing. The therapy's recent success can be attributed to improvements in the viral vector technologies that deliver genetic material to the patient's cells.


https://history.nih.gov/exhibits/genetics/sect4.htm
http://www.genetherapynet.com/JoomlaTest2/index.php?option=com_content&view=article&id=161:historic-overview-of-gene-therapy&catid=97:patient-information&Itemid=14
https://www.labiotech.eu/features/gene-therapy-history/