Human immunodeficiency virus (HIV) over time causes acquired immunodeficiency syndrome (AIDS), a condition in which progressive failure of the immune system allows life-threatening, opportunistic infections and cancers to thrive. Cell toxicity is a major problem when treating HIV.
This invention is a unique vector for modification of CD34+ hematopoietic stem cells for anti HIV gene therapy. The vector contains a modified version of the thymidine kinase gene, TKSR39, which can rapidly kill cells in the presence of gancyclovir (a drug already approved for Herpes virus treatment).
Features, Benefits & Advantages:
The technology targets the infected cells and not the virus hence the development of resistance is unlikely.
- By utilizing tat RNA to transiently express GFP in modified stem cells it’s providing for the first time a method to select modified stem cells that will increase the chances of a successful transplantation.
- The vector is safe as it does not express the gene in the absence of HIV infection. Ganciclovir has been extensively used for herpes (CMV) treatment in HIV infected patients without significant toxicity or side effects.
Stage of Development:
Prototype is in testing