Gene Therapy Provides Long Term Benefits From Skin Diseases
Stem cell-based gene therapy holds promise for the therapy of devastating genetic skin diseases, but the long-term clinical results of this method have been not clear. In a research online in the ISSCR’s journal Stem Cell Reports, published by Cell Press, scientists analyzed a patient with a genetic skin disorder identified as epidermolysis bullosa (EB) almost 7 years after he had gone through a gene therapy process as part of a clinical study. The study exposed that a small number of skin stem cells replanted into the patient’s legs were adequate to recover normal skin function, without resulting in any adverse side effects.
“These results pave the way for the upcoming safe use of epidermal stem cells for combined cell and gene treatment of epidermolysis bullosa and other genetic skin problems,” states senior study author Michele De Luca.
EB is a painful problem that leads to the skin to be very fragile and to blister easily, and it can also lead to life-threatening infections. Due to the fact there is no cure for the disease, present therapy strategies concentrate on reducing symptoms. To assess stem cell-based gene treatment as a potential treatment, De Luca and his co-workers formerly released a phase I/II clinical trial at the University of Modena and enrolled an EB patient known as Claudio. The scientists took skin stem cells from Claudio’s palm, fixed the genetic defect in these cells, and then transplanted them into Claudio’s upper legs.
In the new research, De Luca and his group found that this therapy resulted in long-term recovery of normal skin function. Almost seven years later, Claudio’s upper legs seemed normal and did not show symptoms of blisters, and there was no proof of tumor growth. Extremely, a small number of transplanted stem cells was adequate for long-lasting skin regeneration.
Although Claudio’s skin had gone through about 80 cycles of renewal during this time interval, the replanted stem cells continue to retained molecular functions of palm skin cells and did not follow functions of leg skin cells. ” This identification shows that adult stem cells mainly replenish the cells in which they normally reside, with little plasticity to regenerate other tissues,” De Luca states that. “This calls into question the expected plasticity of adult stem cells and highlights the need to properly chose the right type of stem cell for therapeutic tissue regeneration.”