Importance of Curing Genetic Disease
There are more than 4000 genetic disorders, and that number is likely to increase as we discover more about our DNA. Many of these diseases are incurable.
So, what is gene therapy?
Gene therapy attempts to cure genetic disorders by replacing a defective gene in the human body with a properly functioning gene.
The first FDA-approved gene therapy procedure was performed by Dr. French Anderson in September 1994, on a child born with a rare genetic disorder, known as severe combined immunodeficiency. This caused her to have no effective immune system, resulting in frequent infections, and a poor quality of life.
The procedure consisted of removing the child’s white blood cells, inserting the good functional copies of the desired gene into some of the cells, and then putting them back into her body. The procedure is not a permanent fix, and needs to be performed every few months. But she's alive and well and has a strengthened immune system.