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Normal lives
Researchers at the St. Jude Children's Research Hospital reportedly used HIV to cure infants born with the "bubble boy" disease – a remarkable example of diverting a deadly virus for a new treatment.
"Children are cured," said researcher Ewelina Mamcarz NBC News. "They came to see us as little children, sometimes as young as 2 months old, with serious infections. Now they are at home, lead a normal life and attend daycare. "
Danger Everywhere
The X-linked severe combined immunodeficiency (SCID-X1) is a rare genetic disorder caused by a mutation in the gamma subunit gene of the interleukin-2 receptor (IL2RG).
People born with SCID-X1 do not have a functioning immune system, making exposure to a potentially life-threatening germ. In fact, many children born with SCID-X1 die before their second birthday.
Genetical therapy
In a study published Thursday in the New England Journal of Medicine, St. Jude's team explains how she developed gene therapy to effectively cure 10 infants born with SCID-X1.
After taking bone marrow samples from each patient, they used a modified version of HIV to insert an unmutated IL2RG gene into the patient's stem cells. Then they administered to patients busulfan, a drug that helps the donor stem cells grow, and then was returned to the patient's body.
Most left the hospital in one month.
"Although longer follow-up is needed to evaluate the late effects of treatment," said researcher Mort Cowan, "these findings suggest that most patients treated with this gene therapy will develop a sustainable and complete immune response without side effects."
READ MORE: St. Jude gene therapy heals babies with "bubble boy" disease [St. Jude]
More about gene therapy: Scientists have used gene therapy to cure deafness in mice
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