A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells don’t develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received experimental gene therapy. They experienced substantial improvements in immune system function and normal growth up to two years after treatment.
Gene therapy restores immunity in infants with rare immunodeficiency disease
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