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Clinical Advances in Gene Therapy Updates on Clinical Trials of Gene Therapy in Haemophilia

Clinical Advances in Gene Therapy Updates on Clinical Trials of Gene Therapy in Haemophilia

 

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy

The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy

 

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

 

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.

Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity

Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity

 

Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.