DUARTE, Calif., June 16 (UPI) — U.S. medical researchers say they have demonstrated the first successful gene therapy in patients with AIDS-related lymphoma.
City of Hope researchers in California said their study showed the long-term persistence of anti-HIV genes in patients with the AIDS-related cancer.
In the investigational therapy, patients underwent autologous hematopoietic cell transplantation in which their own blood stem cells were harvested, genetically engineered with three ribonucleic acids that block the human immunodeficiency virus from infecting new cells and then returned to them.
The gene therapy was developed by City of Hope Professor John Rossi, with technology that uses ribozymes and short strands of RNA to selectively silence specific genes against HIV infection.
The scientists said the goal of the therapy is to reboot the immune system to once again identify HIV and mount a response to the infection by lowering the viral load.
“While highly active antiretroviral drugs have managed to turn HIV infection from an immediate death sentence to a long-term manageable chronic condition, we are still seeking a cure,” said Professor David DiGiusto, lead author of the study. “Our research and clinical trials are showing promise for this novel approach to treating HIV patients.”
The research, led by Dr. John Zaia, appears in the early online edition of the journal Science Translational Medicine.
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