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A pioneering gene therapy aims to free patients of blood disease. Is a cure at hand?

"They think getting cured is totally normal," said Alissa Finlayson

The Finlayson Family, of Montana, from left is Ruby, 10, Clint, father, Ada, 9, Liam, 12, Alissa, mother, and Lily, 12, in the patio at Ronald McDonald House where Ada and Lily are being monitored after gene therapy for beta thalassemia by UCSF Benioff Children’s Hospital in Oakland, Calif., on Wednesday, Oct. 18, 2023. Since treatment in August, Ada no longer needs weekly blood transfusions for her rare genetic blood disorder, which causes severe anemia and potentially fatal organ damage. (Ray Chavez/Bay Area News Group)
The Finlayson Family, of Montana, from left is Ruby, 10, Clint, father, Ada, 9, Liam, 12, Alissa, mother, and Lily, 12, in the patio at Ronald McDonald House where Ada and Lily are being monitored after gene therapy for beta thalassemia by UCSF Benioff Children’s Hospital in Oakland, Calif., on Wednesday, Oct. 18, 2023. Since treatment in August, Ada no longer needs weekly blood transfusions for her rare genetic blood disorder, which causes severe anemia and potentially fatal organ damage. (Ray Chavez/Bay Area News Group)
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After years of blood transfusions, "they think getting cured is totally normal," said Alissa Finlayson.

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