By editing Ada's own genes, her 9-year-old blood cells went from being oxygen deficient to oxygen rich, allowing her to stop receiving six-hour, monthly blood transfusions for life. In clinical trials, 90% of patients were able to discontinue transfusions, effectively curing their beta thalassemia. UCSF Benioff Children's Hospital Oakland, led by Mark Walters, MD, was one of three sites that led trials resulting in FDA approval. The therapy was developed by bluebird bio.
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Video by Pete Bell
Additional Photos by Shawna Benson Photography [ Ссылка ]
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