Source: City of Hope
Date: June 16, 2010
Summary:
City of Hope researchers demonstrated the first successful long-term persistence of anti-HIV genes in patients with AIDS-related lymphoma. In the investigational therapy, patients underwent autologous hematopoietic cell transplantation (HCT) in which their own blood stem cells were harvested and genetically engineered with three anti-HIV ribonucleic acids (RNAs) that block HIV from infecting new cells. The study appears online June 16 in the journal Science Translational Medicine.
The gene therapy was developed by City of Hope’s John Rossi, Ph.D., Lidow Family Research Chair and chair and professor, molecular and cellular biology, with technology that uses ribozymes and short strands of RNA, also known as small interfering RNA (siRNA), to selectively silence specific genes against HIV infection. The ribozyme molecule prevents the patient’s white blood cells from producing a protein called CCR5, which HIV needs to enter a cell. The new CCR5-deficient immune cells the patient produces are effectively resistant to HIV infection. Additionally, the siRNA inactivates the virus directly, and a third component, called a TAR decoy sequesters the HIV regulatory Tat protein from the virus. 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.
