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Bluebird Bio’s gene therapy for blood disorders yields some impressive results ― but also raises questions

Bluebird Bio's trial of a gene therapy for two inherited blood disorders yielded some impressive results Friday ― but also raised key questions.

The field of gene therapy is making significant strides towards the day when a one-time treatment ― inserting a healthy gene into patients ― could cure a host of inherited, often fatal diseases. But it’s been a rocky road. And new clinical data presented this morning by Bluebird Bio make clear that success is far from guaranteed. Bluebird is testing a gene therapy for two inherited blood disorders ― and the data managed both to impress and to leave important questions unanswered.

On the impressive side: A patient with the inherited blood disease beta-thalassemia, which causes severe anemia, was able to stop regular blood transfusions one month after treatment and achieved a

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