DOI: 10.1055/s-00000077

Seminars in Thrombosis and Hemostasis

References

Pipe S, Stine K, Rajasekhar A. , et al.
101HEMB01, a phase 1/2 open-label single ascending dose-finding trial of DTX101 (AAVrh10FIX) in patients with moderate/severe to severe hemophilia B demonstrated meaningful but transient expression of human factor IX.

Blood 2017;
130: 3333

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