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Resources

Gene therapy and hemophilia B resources

ASK THE EXPERT VIDEOS

View answers to commonly asked questions about gene therapy for hemophilia B.

  • What are some current unmet needs of people with hemophilia B?

  • How do gene therapy approaches, such as gene transfer, silencing, and editing, differ from each other?

  • What is the difference between in vivo and ex vivo vector therapy?

  • Why are adeno-associated virus (AAV) vectors used in gene therapy?

  • How is targeted delivery of a gene achieved using the adeno-associated virus (AAV) vector platform?

  • Why is hemophilia B a good candidate for gene therapy?

  • How can hematologists prepare themselves to answer questions patients might have about gene therapy?

  • What factors need to be considered for hemophilia B gene therapy preparedness at your center?

  • What is the patient eligibility criteria for hemophilia B gene therapy clinical trials?

How AAV gene therapy works

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Educational Brochure

Download our brochure and learn more about the science of gene therapy for hemophilia B.

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