Regeneratie van de gehele menselijk epidermis dmv transgene stamcellen

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Junctional epidermolysis bullosa (JEB) is a severe and often lethal genetic disease caused by mutations in genes encoding the basement membrane component laminin-332. Surviving patients with JEB develop chronic wounds to the skin and mucosa, which impair their quality of life and lead to skin cancer. Here we show that autologous transgenic keratinocyte cultures regenerated an entire, fully functional epidermis on a seven-year-old child suffering from a devastating, life-threatening form of JEB. The proviral integration pattern was maintained in vivo and epidermal renewal did not cause any clonal selection. Clonal tracing showed that the human epidermis is sustained not by equipotent progenitors, but by a limited number of long-lived stem cells, detected as holoclones, that can extensively self-renew in vitro and in vivo and produce progenitors that replenish terminally differentiated keratinocytes. This study provides a blueprint that can be applied to other stem cell-mediated combined ex vivo cell and gene therapies.

https://www.nature.com/articles/nature24487

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‘Extraordinary’ tale: Stem cells heal a young boy’s lethal skin disease

The complications of the little boy’s genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. There’s no cure for the disease, and it is often fatal for kids. At the burn unit at Children’s Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing — not even his father’s offer to donate his skin — worked to heal his wounds.

“We were absolutely sure we could do nothing for this kid,” Dr. Tobias Rothoeft, a pediatrician with Children’s Hospital in Bochum, which is affiliated with Ruhr University. “[We thought] that he would die.”

As a last-ditch effort, the boy’s father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them — such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boy’s body by grafting on genetically modified cells taken from the boy’s healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide — but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

... meer in de bron: https://www.statnews.com/(...)idermolysis-bullosa/