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