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2021-04-08| In-depthInterviews

The Transformative Potential of Gene Therapies for Hemophilia

by Rajaneesh K. Gopinath
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Hemophilia is an inherited blood disorder where victims suffer from uncontrolled bleeding post injuries. The major forms of the disease, Hemophilia A and B, are characterized by the genetic deficiency of factors VIII and IX, respectively. Of all the diverse therapies available, gene therapy holds incredible promise with its ability to cure the disease via a single-dose administration. Although safety and durability are the biggest hurdles for the field, “advances in gene therapy have been a terrific transformational change,” says Dr. John Pasi, an expert who has been closely involved in the design and development of clinical trials for new gene therapies.

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