Inflammatory factors linked to inhibition of factor VIII gene therapy in hemophilia A

As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting factor VIII from the transferred FVIII gene. Researchers compared the levels of multiple pro- and anti-inflammatory cytokines in a mouse model of hemophilia A that received FVIII gene therapy at different ages and either did or did not exhibit FVIII inhibitor formation, as presented in an article published in Human Gene Therapy.