Gene therapy can cure "bubble boy" disease in babies, researchers find



Researchers have successfully cured several Babies of X linked severe combined immunodeficiency (SCID-X1) —and condition that causes babies to be born without working immune system, commonly referred to as “bubble boy” disease — by using experimental gene therapy technique involving HIV.

The study team developed and lentiviral vector, based on HIV, to transfer and correct copy of the defective gene that causes the condition in the bone marrow stemming.


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