Scientists at the research center Medica Aplicada (CIMA ) of the University of Navarra have genetically modified a virus to be able to express proteins of therapeutic interest. "Two years ago we discovered mutations in the genome of the 'Semliki Forest virus' that allowed it to replicate in mammalian cells without producing harmful effects. These are vectors from which some viral genes were deleted so that they cannot spread. We have now used these vectors to generate stable cell lines that produce therapeutic proteins at very high levels," explains Dr. Cristian Smerdou, researcher work . The results were recently published in the journal New Biotechnology.

In the study, the scientists have generated cells capable of producing two human proteins with therapeutic properties: insulin-like growth factor-I (IGF-I) and cardiotrophin-1. In addition, preclinical programs of study conducted at CIMA and a clinical essay involving the University of Navarra Clinic have confirmed that this protein can be used to treat liver cirrhosis. On the other hand, preclinical programs of study developed at CIMA have shown that cardiotrophin I is a very potent molecule for inducing liver regeneration. It would have application in many pathologies in which severe liver damage occurs".

Increasing stability

The vector used at work is a virus that is not very pathogenic for humans and whose genetic material is an RNA molecule, whereas most organisms contain DNA genomes. According to Dr. Smerdou, "We are currently refining the expression system to increase its stability. The fact that it is based on a viral RNA means that it can theoretically accumulate a certain issue of mutations if the cells over-amplify. So we think a more stable system could be based on a similar vector integrated into the cell's DNA genome."

The development of these systems is being carried out at partnership with business 3PBiopharmaceuticals. The goal is to produce proteins with a Degree of clinical purity that will allow their use in humans at medium deadline.