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Infants' immune disease treated with gene therapy

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.

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