Gene therapy cures siblings of “bubble boy” syndrome

| | September 14, 2012
This article or excerpt is included in the GLP’s daily curated selection of ideologically diverse news, opinion and analysis of biotechnology innovation.

Abbygail Ainslie was born perfect in July 2011. She weighed nine pounds and the genetic tests that have become standard at most hospitals showed no problem whatsoever. But her mother, Jessica Ainslie, didn’t feel right.

“She was a beautiful baby girl,” Ainslie, who lives in Peoria, Arizona, told NBC News. “We got her tested and the pediatrician said, ‘Yeah, everything looks good’,” she added. But everything wasn’t good. Ainslie’s son Colton had been born with a rare genetic disease called severe combined immune deficiency syndrome (SCID), and she knew Abby had a one in three chance of having it, too.

Sure enough, specialized genetic testing done at Duke University confirmed Ainslie’s fears. Abby had the same form of SCID, often called “bubble boy disease” after the case of 13-year-old David Vetter, who died in the 1970s after living in a plastic isolation ward.

Colton and Abbygail are among three children successfully treated for their immune deficiency during an experiment detailed in Tuesday’s issue of the journal Blood. Ainslie says she never doubted her decision to have Abbygail, despite the risks.

View the original article here: Gene therapy cures siblings of “bubble boy” syndrome

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