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95% of kids with “bubble boy” disease cured by one-time gene therapy

95% of kids with “bubble boy” disease cured by one-time gene therapy

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the “b...

A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.

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