Non-technical Abstract 
Non-Technical Abstract 
HIV infection causes progressive destruction of the human immune system and 
eventually AIDS (Acquired Immune Deficiency Syndrome) in the overwhelming majority 
of patients. No curative treatments for HIV or AIDS currently exist. T cells are the main 
targets of HIV in the body. When a specific gene for a molecule called a ribozyme is 
introduced in the laboratory into human T cells they are protected (in the test tube) 
against HIV infection. The ribozyme does not have apparent harmful effects on the T 
cells. The ribozyme acts at two steps in the lifecycle of HIV: by cutting the genes of the 
virus as it enters cells and by cutting the genes of the offspring of virus in infected cells. 
In this study, we will evaluate the safety and usefulness of ribozyme gene therapy in 
four to six patients with HIV infection. We will do so by introducing the ribozyme gene 
into each patient’s T cells in the laboratory and then transferring the cells back into the 
body. We will then study whether the lifespan of these cells is prolonged by having the 
ribozyme gene and whether there are side effects of the therapy. The results will 
determine whether this ribozyme can safely protect T cells in patients with HIV 
infection and will aid design of future trials of gene therapy for HIV and AIDS. 
Recombinant DNA Research, Volume 18 
[379] 
