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Hemophilia B is an ideal target for AAV-based gene therapy because it is caused by genetic mutations in a single gene small enough to fit inside a standard AAV vector. In clinical trials, AAV-based gene therapies in hemophilia B are currently being investigated to determine if patients can display durable endogenous factor IX (FIX) activity.
Naturally occurring AAVs have serotype-specific tropism for particular cells and tissues, including the liver where FIX is produced, making AAV vectors a focus of study in gene therapy for hemophilia B.
The goals of AAV gene therapy for hemophilia B are to provide long-term benefits, with sustained factor activity levels from a single administration of treatment, to reduce spontaneous bleeding and the need for chronic prophylaxis.
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