An entire replacement skin has been grown for the first time using stem cells. The work has been described as one of the most impressive examples to date of the use of stem cells in humans.
The genetically modified skin was created to replace the skin of a seven year old Syrian boy who was suffering from a devastating genetic disorder called junctional epidermolysis bullosa.
The condition is caused by a mutation in the gene that produces the protein that anchors the epidermis to the deeper layers of skin beneath. The boy’s condition has causes skin to become fragile and blister, causing him to lose 80% of his surface skin (epidermis) , leaving him covered in infected wounds. The disorder meant that he did not have long left to live after all conventional therapies had failed.
As a last resort the boy’s family asked for help from Italian scientists who had pioneered a technique to regenerate skin. To date the technique had only been used to successfully graft laboratory-grown skin onto small areas of the body.
The team first took a sample of the patient’s remaining healthy skin, and genetically modified the skin cells to produce a healthy version of LAMB3 – the boy’s missing protein gene.
Because the skin contains its own specialised stem cells – which allow the epidermis to constantly renew – the scientists were able to grow a graft in culture large enough to cover almost the entire body of the boy. The specialised skin stem cells meant that once the transplant had integrated, the skin could renew and repair itself as healthy skin should.
The graft was attached in two separate operations in 2015, and within a month had integrated into the lower layers of skin.
“Once you have regenerated the epidermis, the stem cells keep making the renewal of the epidermis as in a normal [healthy person],” said Professor De Luca, lead author of the study.
Two years later and the procedure has been deemed a complete success. The boy has healthy skin without the need to take medication or ointments.
Looking to the future, if the treatment is proven to be safe in the long term, scientists believe the approach could be used to treat mild to severe skin disorders.
“The success of this combined cell and gene therapy will have huge implications for the field of regenerative medicine and the treatment of genetic diseases.”
Said Dr Higgins, lecturer of Bioengineering at Imperial College London, whose own work focuses on regenerative medicine.