Experimental gene therapy frees ‘bubble boy’ babies from a life of isolation
Treatment restores immune-system function in young children with severe disorder.
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