Tuesday, March 2, 2010

Gene Based Therapy for HIV

UCLA AIDS Institute researchers report they have been able to remove a specific cell receptor from human cells that is critical for binding HIV. Using a siRNA molecule called short hairpin RNA ( shRNA) to induce RNA interference in human blood stem cells, they were able to inhibit the expression of an unneeded cell receptor called CCR5. Individuals who lack this receptor are naturally resistant to HIV. Please find the article in this months- Blood, Journal of the American Society of Hematology.
Now for my thoughts. This represents a paradigm shift and a completely new strategy for treatment of HIV in currently infected individuals. Depending on how difficult this shRNA was to transplant, the treatment could potentially be of value in high risk populations as a new type of vaccine. Are there other receptors for viruses or parasites representing potential targets for this type of therapy- HBC, HCV for example. This is one of the most exciting potential therapies I have come across- eliminating the expression of a particular receptor to effect change in disease susceptibility. It's particularly interesting from the perspective of regenerative medicine, perhaps eliminating receptors that have been identified with disease in the course of replacing tissue with adult stem cells. This is very exciting and most promising. I'm certain we'll be hearing more about this type of receptor modification treatment in the next few years.