Stem cell study shows potential for successful treatment of genetic skin disorders

Modena, Italy - Researchers at the University of Modena and Reggio Emilia have shown how stem cells not only can be used to treat damaged eyes but, in combination with gene therapy, can treat a rare and debilitating skin disease, Sciencedaily.com reports.

First, researchers showed that cells of the cornea are constantly being replaced by new cells from a surrounding area called the limbus. The cells differentiate into corneal epithelium and migrate to the cornea. A corneal transplant, says the study, will only be successful if the limbus remains intact and can continue to replenish the new cornea.

In cases where the limbus is destroyed, researchers say, a new cornea can be grown from limbar stem cells taken from the healthy eye. Of 240 patients undergoing this procedure, the cornea regenerated successfully in 70 percent of cases, the study says.

The same theory was applied to the rare, debilitating genetic skin syndrome epidermolysis bullosa, in which the skin is fragile and prone to blistering due to faulty proteins that anchor the surface layers of skin to the body. One form of the disease is characterized by a mutation in one of the anchoring proteins, laminin 5.

Researchers conducted a small-scale trial of gene therapy using holoclones - skin stem cells - on a small part of a 37-year-old male patient’s body.

Because the patient’s body was so badly affected, the only holoclones to be found were a few on the patient’s palms. Using gene therapy, the research team inserted the correct laminin gene into the growing cells and grafted the new tissue onto the patient’s body. The graft was successful, and after several months the skin remained relatively normal and exhibited no blistering or flaking.

The researchers concluded that this small trial showed it is possible to use stem cells in gene therapy for genetic skin disorders.