“Bubble Boys” Lose Bubbles Thanks to Gene Therapy

“Bubble Boys” Lose Bubbles Thanks to Gene Therapy

Historically, patients with severe combined immunodeficiency (SCID), an often fatal congenital disorder in which the cells involved in immune responses fail to work properly, have been kept in isolation in a sterile environment to protect them from infection. Currently, the only long-term treatment for so-called bubble boy disease is a bone marrow transplant, but an experimental gene therapy is showing promise for boys with X-linked SCID. Of the nine babies given the treatment, six developed immune systems capable of fighting off infection. More… Discuss

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