Gene therapy restores immunity in infants with rare immunodeficiency disease

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.