(HealthDay)—Early results suggest that stem cell gene therapy is a safe and effective treatment for boys with early-stage cerebral adrenoleukodystrophy, according to a study published online Oct. 4 in the New England Journal of Medicine to coincide with the 2017 Child Neurology Society Annual Meeting, being held Oct. 4 to 7 in Kansas City. Florian Eichler, M.D.,...
Tag: <span>stem cell gene therapy</span>
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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...