Thanks to gene therapy, a boy born without an immune system can now play in the yard.

Jameson Golliday, in his bubble stroller, reaches out to brother Shawn (Courtesy Jennifer Golliday)

I met two-year-old Jameson Golliday on a patch of lawn in Bloomington, Illinios. He had blue eyes, dark hair, and wore a striped shirt, shorts, and Crocs. He followed his mother, Jennifer, 31, as she pushed a wheelbarrow around the yard, picking up sticks. The neighbors were burning lawn debris and fly ash was swirling in the sky, big fluffy flakes, falling lightly all around us. Jamesons brother, Shawn, 5, was wearing a Star Wars t-shirt and tossed a plastic sword to his grandfather. There was the bat-bat of plastic, and things quickly turned Galactic.

Jamie and his motherretreated to a quiet spot in the yard. She lets him run free in the backyard, but he cant play with other children or touch high-contact surfaces such as playground equipment.

Its super sad, Jennifer told me. He sees kids off in the distance and points at them. I know he wants to interact.

Jamie was born with X-SCID, or "bubble boy disease,"which means he has no immune system. At birth he had no mature T-cells, the rugged soldiers of the immune system that sense and fight infections, due to mutations in the IL-2 gene on his X-chromosome, which rendered the gene non-functional.

David Vetter was the most famous bubble boy. In the 1970s and 1980s he lived in a bubble, using only items sterilized with 140-degree ethylene oxide gas. Doctors tried a bone marrow transplant from his sister to introduce working T-cells into his system. But the Epstein-Barr virus was sleeping in the marrow, and it triggered the growth of many tumors. Vetter died at age 12.

When Jamie was born he had a small cough, but unlike most colds, it didnt go away, not for a year. His mom noticed a bump on his hip one day, which turned out to be a cluster of B-cells unsuccessfully trying to fight an infection. This clustering looked like cancer, and caused doctors to diagnose Jamie with diffuse B-cell lymphoma, which is very uncommon in infants. Later tests revealed Jamies illness for what it wasX-SCID.

In April 2012, not long after Jamies diagnosis, Jennifer and Jamie, then 1, took a medical jet to Cincinnati Childrens Hospital, so that Jamie could participate in a trial for gene therapya treatment that wasnt available in David Vetters time. Jennifer improvised a bubble stroller out of a rain tarp and a baby stroller, to transport him back and forth between the hospital and the Ronald McDonald House where they stayed in Cincinnati. Inside, he wore a surgical mask.

Doctors drew some of Jamies bone marrow, and dosed it with a virus carrying the IL-2 gene. The virus serves as a tiny pilot, carrying the payload of the IL-2 gene into his cells, installing it in his genome. That gene builds a protein called a cytokine, which sends a signal telling T-cells to mature. Weeks after Jamie received his shiny new IL-2 gene, a blood test showed 13 mature T-cells per microliter, where before hed had none.

Link:
How to Cure a Bubble Boy

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