There are positive indications that mesenchymal stem cells could play an important role in gene therapy, i.e. the transfer of healthy DNA into diseased cells, with the long term objective of successfully treating various serious diseases.
Scientists have recently reported the results of gene therapy in addressing the deficiency of alpha 1 antitrypsin, an enzyme necessary for maintaining normal liver function. In Europe, one child out of every 2000 is born with the deficiency of this enzyme, which leads to liver cirrhosis and pulmonary emphysema.
These children require liver transplantation, which is a serious and life-threatening surgical procedure. So it is possible today, knowing precisely the abnormality in the gene that regulates the production of the enzyme, to repair the stem cells, and then direct them into the liver.
Because the environment of the liver is familiar to the mesenchymal stem cells, they arrive, settle and remain in the liver. We have observed this process in animal models. Stem cells that were treated with gene therapy differentiate into liver cells and produced the natural enzyme for up to six weeks after administration.