According to BBC, 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 such as bubbles and tend to die as infants.
US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or “bubble boy” disease.
The gene therapy involved collecting the babies’ bone marrow and correcting the genetic defect in their DNA soon after their birth. Now, 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.”
Currently, the best treatment for SCID-XI is a bone marrow transplant with a tissue-matched sibling donor. But according to St Jude, more than 80% of these patients lack such donors and must rely on blood stem cells from other donors.
This process is less likely to cure the bubble boy disease and is more likely to cause serious side effects as a result of treatment.
