Until today, no treatment was available for people with genetic skin disorder known as epidermolysis bullosa (EB), but gene therapy may have an answer to their problem.
Researchers at the University of Modena and Reggio Emilia in Italy, after evaluating a patient suffering from EB who underwent gene therapy treatment, found that skin stem cells transplanted into the patient's legs restored normal skin function, without causing any side-effects.
"These findings pave the way for the future safe use of epidermal stem cells for combined cell and gene therapy of epidermolysis bullosa and other genetic skin diseases," Michele De Luca, senior study author, said.
The team extracted a small number of skin stem cells from the patient's palm, corrected the genetic defect in these cells and then transplanted these into the upper legs.
The patient's upper legs looked normal and did not show signs of blisters and there was no evidence of tumour development.
"This finding suggests that adult stem cells primarily regenerate the tissue in which they normally reside, with little plasticity to regenerate other tissues. This research highlights the need to carefully choose the right type of stem cell for therapeutic tissue regeneration," the study, published in the journal Stem Cell Reports, emphasised.
EB is a painful condition that causes the skin to be very fragile and to blister easily, and it can also cause life-threatening infections. Because there is no cure for the disease, current treatment strategies focus on relieving symptoms.