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

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

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May 06, 2019
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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

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