Gene Therapies and Regenerative Medicine to Treat Genetic Skin Conditions

In 2015 a seven year old boy escaped the horrors of war in Syria, but he still had his own battles to fight. At birth he had been diagnosed with epidermolysis bullosa, a genetic skin condition that had covered eighty percent of his body with raw open sores. Epidermolysis bullosa is caused by a defect on the boys LAMB3 gene which among other things produces a protein called laminin 332, a protein largely responsible for the adhesion of the epidermis to the dermis. Without this protein much of the epidermis on the boys body was constantly peeling away, leaving his dermis exposed, and causing him unbearable amounts of pain.
Luckily for the boy twelve years earlier Michele De Luca, a researcher at the University of Modena in Italy, had developed an experimental procedure to help a patient with a similar skin disorder. In short the boys doctors was able to send a healthy sample of the his skin to De Luca, who was then able to infect these skin cells with a virus that inserted a LAMB3 gene that functioned properly into the DNA of the cells. They then grew large sheets of the boys skin on scaffolds and were able to replace eighty percent of the boys skin with skin that contained a fully functioning LAMB3 gene and give him a chance at a normal life. One of the reasons they were successfully able to do this is because the epidermis is maintained by a small number of progenitor stem cells and since they replaced all of the ones in the boys skin with cells that had a functioning LAMB3 gene all future skin cells the boy produces will contain that gene as well.
I chose this article because the implications of this event are far reaching. Not only is it amazing but it is also an indicator of the potential of gene therapies and regenerative medicine. In my opinion if we as scientists are able to safely, reliably, and economically research and perform procedures that will help people overcome conditions on a genetic level, there is no end to the improvements that can be made on the human condition.

https://www.scientificamerican.com/article/massive-skin-replacement-saves-childs-life/