Experimental gene therapy frees ‘bubble boy’ babies from a life of isolation

Treatment restores immune-system function in young children with severe disorder.

Go to the profile of Nature Research
May 06, 2019

An experimental gene therapy has restored functioning immune systems to seven young children with a severe disorder that would have sentenced them to a life of isolation to avoid potentially deadly infections. They are now with family at home, and an eighth child was slated to be released from hospital in mid-April.

The children have mutations in a gene that is crucial for immune-system development, causing a disorder called X-linked severe combined immunodeficiency (SCID-X1). The gene-therapy treatment replaces the mutated gene, called IL2RG, with a corrected copy. SCID-X1 and related disorders are sometimes called ‘bubble-boy’ diseases because of the plastic enclosures that were once used to protect affected children from possible infection. For them, even a common cold can be fatal.

By: Heidi Ledford/Nature News

No comments yet.