Gen therapy against AIDS
The immune deficiency disease AIDS could be stopped in humans for the first time by gene therapy. A phase I clinical trial was successful in five patients, scientists from the US report.
The physicians working with Carl June from the University of Pennsylvania in Philadelphia had taken T cells from their HIV-infected subjects and genetically manipulated them: through infection with genetically modified lentiviruses – which also included the HI virus – produced the immune cells RNA molecules that should paralyze the genome of the AIDS pathogen. Each patient then received about ten billion altered T cells back - this corresponds to two to ten percent of the total supply of immune cells in the human body.
In fact, the viral load remained constant in all five patients over the next nine months. In one test person, the number of HI viruses even decreased.
The researchers conclude that gene therapy could help against AIDS. However, the number of patients is too small and the duration of the study too short to draw any further conclusions. Long-term effects can only be seen after 15 years. A phase II study is now to test this gene therapy on a larger number of AIDS patients. (aj)