Biol 100
Human Gene Therapy
Leeward Community College
Warren Katz
10/26/14
1
Biol 100
Human Gene Therapy
Historical Background
“Gene therapy attempts to treat genetic diseases at the molecular level by correcting
what is wrong with defective genes”(Human Genetics, 2010, p.1). Viruses are used by
medical researchers to deliver the correct gene to cells. Viruses deposit their own genetic
material into host cells to instruct those cells to make more viruses. In gene therapy, the
DNA for the desired gene is inserted into the genetic material of the virus. Fatty
molecules known as liposomes can be used as micropipettes, sometimes called “gene
guns” to insert genes into cells physically (p.1).
A four-year old and a nine year old girl were the first gene therapy recipients on
September 14, 1990 at the NIH Clinical Center. She has adenosine deaminase (ADA)
deficiency, a genetic disease which leaves her defenseless against infections. White blood
cells were taken from her, and the normal genes for making adenosine deaminase were
inserted into them. The corrected cells were reinjected into her by Dr. W. French
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