Doctors find breakthrough in ‘bubble boy’ disease

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NEW YORK (NBC News) A gene therapy developed at St. Jude’s Children’s Hospital has cured 10 infants born with a rare genetic disease called SCID-X1, also known as “Bubble Boy Disease,” which is named for the way kids have to be protected from germs. 

Omarion is one of those 10 infants, who was born with the disease. He had no functioning immune system, making even the most common cold potentially deadly. 

“He was on protective isolation. So he couldn’t go outside and he couldn’t have contact with anyone,” said Omarion’s mom, Kristin Simpson. 

Now, Omarion is developing and growing normally after doctors took out his bone marrow, and replaced his defective gene with a healthy copy, and reinfused the cells back into his body. 

“We do that after two low doses of chemotherapy that makes room in their bone marrow for the newly corrected, gene corrected cells,” said Dr. Ewelina Mamcarz. 

After several months of treatment, Omarion and the other nine babies, developed a normal immune system. Previous forms of gene therapy for kids with SCID-X1 carried the risk of leukemia, so far this one does not. 

Omarion turns one next week, reaching a milestone both for him and the field of medicine. 

 

Copyright 2019 Nexstar Broadcasting, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

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