Goal: AAV gene therapy limits for Hemophilia A

In this week's episode, Blood editor Dr. James Griffin interviews authors Drs. Vincent Muczynski and Mark Geyer on their latest research published in Blood. Dr. Muczynski's research asks if there could there be a better gene than the factor VIII (FVIII) gene to transfer for curative treatment of hemophilia A? Dr. Geyer then explores CAR T cells armed with interleukin-18 (IL-18) secretion that target CD371, a transmembrane glycoprotein with high expression on AML and leukemia-initiating cells. Both studies explore finding novel targets for these powerful treatment modalities. 

Featured Articles:

• Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody
• CD371-targeted CAR T cells secreting interleukin-18 exhibit robust expansion and clear refractory acute myeloid leukemia