We All Would Fly
Anton Delgado, five years old, recently arrived in Minneapolis from Texas. He wears a protective mask and bandages over his arms. He must. His skin is so delicate that even normal contact makes it blister and fall off.
Anton is one of the “butterfly children”—those who suffer from the rare and deadly epidermolysis bullosa, commonly known as EB.
Anton and his parents came here because the University of Minnesota Children’s Hospital is the only place in the world that offers a bone marrow transplant therapy for EB.
Pioneering Treatment, Multidisciplinary Team
“EB, in its most severe forms, destroys who you are,” says Dr. Jakub Tolar, who leads the U team that’s working with Anton. “There are two hundred children born today in the United States that, because of a genetic disorder, will not see their fifth birthday.”
EB is not yet curable, but the U’s transplant therapy is bringing children and families hope—stem cells from a healthy donor help reduce blistering and heal a patient’s skin.
U physicians carry out transplant therapy about five times a year. The multidisciplinary therapy team is extensive. It includes specialists in dermatology, infectious disease, anesthesiology, pediatric surgery, gastroenterology, nephrology, and nutrition and pain management.
A Treatment for All Genetic Disorders?
U researchers are now exploring a new way to treat EB—genome editing. The idea is to go into a gene and swap out “errors” for the correct information, enabling the gene to perform normally.
The promise is immense. “Genome editing has the potential to treat or cure thousands of genetic conditions that affect 10 percent of Americans,” says Tolar. “Any genetic disorder, in principle, is treatable. Perhaps curable.”