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Pioneering stem cell gene therapy cures infants with bubble baby disease

Evangelina Vaccaro (far right), who in 2012 received treatment developed by UCLA’s Dr. Donald Kohn for bubble baby disease, with her family before her first day of school.   UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can...