Therapy for Severe Combined Immune Deficiency Syndrome (SCID)
very first successful gene therapy was used to treat SCID back in
1990. Often referred to as "bubble-boy syndrome," SCID
is caused by a single defective gene, and causes the human immune
system to fail. In other words, the body has no defenses against
diseases like chicken pox or even the common cold.
believed that replacing the defective gene could reverse the effects.
The first patient to undergo this experimental treatment was a 4-year-old
girl suffering from SCID. The team of researchers injected copies
of a working gene into specially engineered (created) viruses. The
viruses were injected into the patient, "delivering" billions
of the new healthy genes. The treatment was a success, but the patient
must still receives drug therapy to help her stay healthy.