Using Stem Cells to Regenerate Cornea and Treat Genetic Skin Disease

Filed in archive Diagnostics, Methodologies and Instrumentation , Drugs, Vaccines and Therapeutics , Gene Therapy , Stem Cells on October 24, 2007

During an international meeting of stem cell scientists in Milan, Italian scientists report on the use of stem cells to replace damaged corneal tissues in the eye and to treat Epidermolysis Bullosa, a rare but debilitating genetic disease of the skin characterized by highly fragile and blistering skin.

To restore eyesight in eyes where the cornea has been damaged, the scientists removed limbar stem cells taken from the healthy eye to culture a new cornea and graft it on to the damaged eye. The team showed that of 240 patients who were operated on in this way, the cornea regenerated successfully in 70% of cases.

To treat Epidermolysis Bullosa, the researchers obtained consent to carry out a small-scale trial of a novel gene therapy using skin holoclones on one patient, a 37-year-old male, on small part of his body .

"Most people have between seven and ten per cent of holoclones. Our man had none. Eventually we found a few in the palms of his hand and cultured them from a biopsy."
The team then used gene therapy to insert 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 to all intents normal, without the blistering and flaking.

"This demonstrates that it is possible to use stem cells in gene therapy for genetic skin disorders," Professor De Luca said.

Source: ESF