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

BMJ 2019; 365 doi: (Published 18 April 2019) Cite this as: BMJ 2019;365:l1848
  1. Elisabeth Mahase
  1. 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 …

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