NIH researchers restore children's immune systems with refinements in gene therapy

New approach replaces life-long, painful injections

Researchers have demonstrated that a refined gene therapy approach safely restores the immune systems of some children with severe combined immunodeficiency (SCID). The rare condition blocks the normal development of a newborn’s immune system, leaving the child susceptible to every passing microbe.

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This page was last updated on Friday, January 21, 2022