Gene Therapy Cured ‘Bubble Boys’

An experimental gene therapy has cured eight infants with the immune system deficiency known as Bubble Boy Disease.

'It was the Moors.' / Image: Fox
"It was the Moors." / Image: Fox

Chances are the only “Bubble Boy” you’re familiar with was arguing that the Moors invaded Spain in 8th Century. Or was it the Moops? Regardless, a recent Bloomberg article contained good news for bubble boys everywhere: a cure has been found! Bubble Boy Disease refers to an immune-system deficiency so severe that children were at one time kept in total isolation for fear that any attack on the immune system could be lethal.

A team of researchers at St. Jude Children’s Research Hospital in Memphis has developed a personalized gene therapy treatment to correct the genetic issue. It proved successful in eight male infants who are now toddlers who are leading normal lives. St. Jude licensed the therapy to Mustang Bio Inc., a small biopharmaceutical company that plans to file for global regulatory approval by the end of 2021.