Ten babies with X-linked severe combined immunodeficiency, or "bubble boy" disease, who received a gene therapy that replaces a defective gene variant soon after birth were successfully treated and had normal growth and development without complications or side effects. The findings in The New England Journal of Medicine also showed healthy immune systems and vaccine response among treated infants, but more studies are needed to identify any long-term issues.
Infants' immune disease treated with gene therapy
Sign up for Sigma Xi SmartBrief
News on scientific research and innovation
Get the intelligence you need: news and information that is changing your industry today, hand-curated by our professional editors from thousands of sources and delivered straight to your inbox.