What will Gene Therapy Mean for Human Skin Disease?

A recent study looks at the benefits of stem cell-based gene therapy.

Treatments for this devasting genetic skin conditions have been difficult to tackle over the years, but scientists continue to test the importance of the therapy. 

Researchers evaluated a patient with the genetic skin disorder epidermolysis bullosa (EB), who had undergone a gene therapy procedure as part of a clinical trial nearly seven years ago. Study findings showed that a small number of skin stem cells transplanted into the patients legs were enough to restore the skin to normal function without causing any adverse 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," senior study author Michele De Luca of the University of Modena and Reggio Emilia said, via a press release.

Fortunately, in a new study, De Luca and colleagues found that stem-cell based gene therapy could help restore the normal function of the skin. Background information from the study notes that seven years after the findings, Claudio's upper legs looked normal without any signs of blisters or tumor development. Study authors note how remarkable it is that only a small number of cells were needed to restore the skin to its normal condition.

EB is a rare yet painful condition that causes the skin to become very fragile and blister easily. For those suffering from this health issue, it can result in life-threatening infections with limited treatment options, with no cures for the health issue at this time. However, researchers continue to study the importance and prevalence of stem cell-based gene therapy.

Researchers took skin cells from Claudio's palm and corrected the genetic deficit in the cells. They then placed the same cells in his upper legs. However, the study notes that even after Claudio's skin had undergone close to 80 cycles of renewal during this time period, the transplanted stem cells retained their molecular features of palm skin and did not adopt the features of the leg tissue.

"This finding suggests that adult stem cells primarily regenerate the tissue in which they normally reside, with little plasticity to regenerate other tissues," De Luca said, via the release. "This calls into question the supposed plasticity of adult stem cells and highlights the need to carefully chose the right type of stem cell for therapeutic tissue regeneration."

More information regarding the study can be found via the journal Stem Cell Report.