Gene therapy can cure “bubble boy” disease in babies, researchers findBMJ 2019; 365 doi: https://doi.org/10.1136/bmj.l1848 (Published 18 April 2019) Cite this as: BMJ 2019;365:l1848
- Elisabeth Mahase
- The BMJ
Researchers have successfully cured several babies of X linked severe combined immunodeficiency (SCID-X1)—a condition that causes babies to be born without a working immune system, commonly referred to as “bubble boy” disease—by using an experimental gene therapy technique involving HIV.
The study team developed a lentiviral vector, based on HIV, to transfer a correct copy of the defective gene that causes the condition into the bone marrow stem …