Gene Therapy: A Medical Revolution

The Journey of Gene Therapy

 

The current state of gene therapy wouldn't be possible if it wasn't for the pioneers that started gene therapy off in the 20th century. After the revolutionary discovery of DNA and basic genetic information, these couple of historical events set the foundation for our current technology of gene therapy.

 

 

Gene therapy was first talked about in the 1970s when a paper was published in Science called “Gene Therapy for Human Genetic Diseases” by Theodore Friedmann and Richard Roblin. This paper incorporated how good DNA could be used to replace defective DNA in people with disorders.

• In 1983, scientists from Baylor College of Medicine in Texas said that one day, gene therapy could be a possible approach for treating those with Lesch-Nyhan disease, a rare neurological disorder.

• In 1985 Dr. Anderson and Dr. Blaese worked together to demonstrate how cells from patients with ADA deficiency could be fixed in tissue culture. They used a retrovirus as a vector to move the correct ADA gene into the cells of the patient.

• In 1986, the same team of scientists got together and studied how effectively the right genes were transferred into the bone marrow cells in animals, but the number of cells that were able to receive the correct gene wasn’t enough for a useful treatment.

• In 1988, the scientists decided to go back and use white blood cells (T cells) instead of bone marrow cells. Switching over greatly increased the number of right genes taken up by the cells in the animals, and the experiments were so successfully that scientists started thinking of ways that they could do this in humans.

• In 1990, Dr. Anderson, Blaese, and Dr. Kenneth Culver made use of a virus to deliver the right ADA gene to two girls that had ADA deficiency and each girl was given continuous treatments over two years. The two girls now live normal lives and are able to produce ADA now.In 1993 scientists used gene therapy to treat newborn babies that had ADA deficiency and after one treatment, the babies are showing improvement in the growth of ADA genes in their bodies.

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