US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or “bubble boy” disease.
The babies, born with little to no immune protection, now have fully functional immune systems.
Untreated babies with this disorder have to live in completely sterile conditions and tend to die as infants.
The gene therapy involved collecting the babies’ bone marrow and correcting the genetic defect in their DNA soon after their birth.
The “correct” gene – used to fix the defect – was inserted into an altered version of one of HIV, the virus that causes AIDS.
Researchers said most of the babies were discharged from the hospital within one month.
Dr Ewelina Mamcarz of St Jude, an author of the study, said in a statement: “These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives.”
“This is a first for patients with SCID-X1,” she added, referring to the most common type of SCID.
The patients were treated at St Jude Children’s Research Hospital in Memphis and at UCSF Benioff Children’s Hospital in San Francisco.
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