Gene Therapy

The non-surgical solution to desease

What is gene therapy?

Gene therapy is an experimental technique using genes to treat or prevent disease. This technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery in the future.

Where did gene therapy come from?



The gene therapy was first recognised in the 1960s in Bethesda, Maryland. In 1972, Theodore Friedmann and Richard Roblin published a scientific paper called "Gene therapy for human genetic disease?" which cited Stanfield Roger's proposal in 1970 that "good DNA" could be used to replace the defective DNA in people with genetic disorders.

How does the gene therapy work?

In the gene therapy, a patient's defective cells are first harvested from them. Then, scientists alter the virus, so they cannot reproduce. Next, the altered virus is mixed with the patient's cells. This creates genetically altered cells. The genetically altered cells are then reinjected into the patient's body. The altered cells can then produce the desired protein. A four year old girl was the first patient treated by gene therapy. The girl's white blood cells were taken from her, inserted with correct genes for making ADA, and then re-injected into her.


For visual, see pictures below.

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How was the gene therapy tested?

Gene therapy was first used on a four year old girl on September 14, 1990. The girl's white blood cells were taken from her, inserted with correct genes for making ADA, and then re-injected into her. This experiment was a success.

Techniques of the gene therapy

The SMaRT technique targets and repairs the mRNA transcripts copied from the mutated gene. Instead of attempting to replace the entire gene, this technique repairs only the section of the mRNA transcript that contains the mutation.

Theodore Friedmann

Dr. Theodore Friedmann, who received his M.D. from the University of Pennsylvania, serves as the Professor of Pediatrics and Director of the Gene Therapy Program at the University of California, in San Diego. Dr. Friedmann has over 40 years of research and experience in genetics and molecular therapies. He is an expert in Viral Vectors, and he has been a member of the Scientific Advisory Board of Tapestry Pharmaceuticals, since January 12, 2004. Since 2000, he has served as the chairman of the RAC at the National Institution of Health.


Dr. Friedmann believed that "good DNA" could be used to replace the defective DNA in a patient's cells. In 1968, he discovered that, by adding foreign DNA to cultured cells from patients with Lesch-Nyhan syndrome, they could correct genetic defects that caused rare and devastating neurological disorder.

Learn more about the gene therapy

For further detail on the gene therapy, click "Watch Video."