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In this study of 22 patients around the world treated with the gene therapy: Three of the nine patients with the most severe form of the disease stopped transfusions The need for blood transfusions was reduced by 73% overall in those patients 12 of the 13 patients with a less serious form of the disease no longer needed transfusions The researchers also said there were no safety concerns after the treatment in the short term

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It is used widely in pharma industry to ensure safety of the final product