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An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder, a promising sign that it could one day be defeated.
The study included 62 children with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) – a disorder caused by genetic mutations that leave children without working immune systems, making them extremely vulnerable to infections.
Existing therapies for ADA-SCID are expensive and risky, but without treatment
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