New gene therapy shows promise against ‘bubble boy’ disease


A new variation of therapy, which uses a genetically-engineered virus to help the body create disease-fighting T-cells, shows early promise without evidence of leukaemia, according to research.

Experts re-look at severe infant immune systems deficiency disease with new therapy variations.

More than a decade ago, doctors showed dramatic progress in helping infants born with a severe deficiency in their immune systems, a condition known as “bubble boy” disease. But when one quarter of treated children developed leukaemia within two to five years, researchers went back to the drawing board.

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