Stem cell gene therapy shows promise for treating `devastating` skin diseases
Washington: Researchers evaluated a patient with a genetic skin disorder known as epidermolysis bullosa (EB) nearly seven years after he had undergone a gene therapy procedure as part of a clinical trial.
They found that a small number of skin stem cells transplanted into the patient's legs were sufficient to restore normal skin function, without causing any adverse side effects.
To evaluate stem cell-based gene therapy as a potential treatment, Senior study author Michele De Luca of the University of Modena and Reggio Emilia , and his colleagues previously launched a phase I/II clinical trial at the University of Modena and recruited an EB patient named Claudio.
The researchers took skin stem cells from Claudio's palm, corrected the genetic defect in these cells, and then transplanted them into Claudio's upper legs.
In the new study, De Luca and his team found that this treatment resulted in long-term restoration of normal skin function. Nearly seven years later, Claudio's upper legs looked normal and did not show signs of blisters, and there was no evidence of tumour development. Remarkably, a small number of transplanted stem cells was sufficient for long-lasting skin regeneration.
Even though Claudio's skin had undergone about 80 cycles of renewal during this time period, the transplanted stem cells still retained molecular features of palm skin cells and did not adopt features of leg skin cells.
The study is published in journal Stem Cell Reports.