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Pioneering stem cell gene therapy cures infants with bubble baby disease
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.
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| Categories: | Tags: stem cells, immune system, genetics, genome modification, gene engineering, immunodeficiency, Adenosine deaminase-deficient | Comments: (1) | View Count: (839)