Recipient: Department of Cell Biology and Virology, Heinrich Pette Institute (HPI) for Experimental Virology and Immunology, Hamburg
Funding: GO-Bio Phase I (01.02.2008 - 31.12.2011, 2.149.664 Euro)

Summary

Around 40 million people are currently estimated to be infected with HIV, and many are diagnosed with the immunodeficiency syndrome AIDS. Despite decades of research, no cure to help these people has been found to date. Existing drugs are directed only against the spread of the AIDS-causing HIV virus, which are able to keep the disease in check at best. Because the virus genome integrates itself firmly into the DNA of the host cell, leaving behind a genetic blueprint for the production of ever more viruses in the body of the affected individual, it is not possible to reverse an infection.

The work of Joachim Hauber at the Heinrich Pette Institute for Experimental Virology and Immunology in Hamburg is aimed, for the first time, at breaking this vicious circle. Here, Hauber is working alongside a team led by Frank Buchholz from the Max Planck Institute for Molecular Cell Biology in Dresden. Thereby, the researchers have found a means of completely removing the genome of HI viruses from infected cells through the use of the recombinase enzyme as a form of molecular scissors. This enzyme has been optimised in a biotechnological process to enable it to detect the viral genetic material and cut it out of the genome of affected cells. Within the framework of GO-Bio, Hauber and his team are now planning the development of a gene therapy procedure for use on humans on the basis of this cell culture-based method. This will initially encompass the optimisation of recombinase production as well as the testing of suitable methods of gene transfer via viral transport systems (vectors), which carry the enzyme into the genome of infected cells. Here, the scientists will make use of the established technologies of somatic gene therapy. The treatment will be carried out with an ex vivo approach in which infected cells are removed from the patient before being treated and subsequently returned. The implementation of this therapy is planned to take place within a yet-to-be-founded company, which will also pursue clinical application together with selected partners.