Rare 'Bubble Boy Disease' Likely Cured with New Gene Therapy

Live Science | 4/18/2019 | Staff
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Eight infants with a severe immune disorder, sometimes known as "bubble boy disease," appear to be cured of the disease thanks to an experimental gene therapy, according to a new study.

The disorder, officially called X-linked severe combined immunodeficiency (SCID-X1), causes babies to be born with little to no immune protection, making them prone to developing life-threatening infections. It's caused by a specific gene mutation.

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All of the children are now producing the immune cells needed to fend off the barrage of germs that humans encounter in their everyday lives, according to the study, published Wednesday (April 17) in the The New England Journal of Medicine.

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"These patients are toddlers now, who are responding to vaccinations and have immune systems to make all [the] immune cells they need for protection from infections as they explore the world and live normal lives," lead study author Dr. Ewelina Mamcarz, a pediatric hematologist-oncologist at the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy in Memphis, Tennessee, said in a statement.

About 16 months after their treatment, the patients are developing normally and have not experienced serious side effects from the therapy. But they will still need to be monitored for a longer period to determine if the treatment is long-lasting and doesn't cause side effects later in life, the researchers said.

Boy

"Bubble boy"

SCID-X1 is caused by a mutation in a gene called IL2RG, which...
(Excerpt) Read more at: Live Science
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