Scientists Gene-Edited HIV to Cure “Bubble Boy” Disease
"The children are cured... they are home, living normal lives, attending daycare."
Researchers from St. Jude Children’s Research Hospital have reportedly used HIV to cure infants born with “bubble boy” disease — a remarkable example of hijacking a deadly virus for a new treatment.
“The children are cured,” researcher Ewelina Mamcarz told NBC News. “They came to us as little infants, some of them as young as 2 months, with severe infections. Now they are home, living normal lives, attending daycare.”
X-linked severe combined immunodeficiency (SCID-X1) is a rare genetic disorder caused by a mutation in the interleukin-2 receptor subunit gamma (IL2RG) gene.
People born with SCID-X1 lack a functioning immune system, which makes exposure to any germ potentially life-threatening. In fact, many children born with SCID-X1 die before their second birthday.
After collecting bone marrow samples from each patient, they used a modified version of HIV to place a non-mutated IL2RG gene into the patients’ stem cells. Then they dosed the patients with busulfan, a drug that helps make room for donor stem cells to grow, and returned the cells to the patients’ bodies.
Most were discharged from the hospital within a month.
“While longer follow-up is needed to assess any late effects of treatment,” researcher Mort Cowan said in a news release, “these results suggest most patients treated with this gene therapy will develop a complete durable immune response without side effects.”
READ MORE: St. Jude gene therapy cures babies with ‘bubble boy’ disease [St. Jude]
More on gene therapy: Scientists Used Gene Therapy to Cure Deafness in Mice
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