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Gene therapy in an era of emerging treatment options for hemophilia B
Author(s) -
Monahan P. E.
Publication year - 2015
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/jth.12957
Subject(s) - factor ix , medicine , clotting factor , genetic enhancement , haemophilia , context (archaeology) , population , coagulopathy , recombinant dna , immunology , pediatrics , gene , genetics , biology , paleontology , environmental health
Summary Factor IX deficiency (hemophilia B) is less common than factor VIII deficiency (hemophilia A), and innovations in therapy for hemophilia B have generally lagged behind those for hemophilia A. Recently, the first sustained correction of the hemophilia bleeding phenotype by clotting factor gene therapy has been described using recombinant adeno‐associated virus ( AAV ) to deliver factor IX . Despite this success, many individuals with hemophilia B, including children, men with active hepatitis, and individuals who have pre‐existing natural immunity to AAV , are not eligible for the current iteration of hemophilia B gene therapy. In addition, recent advances in recombinant factor IX protein engineering have led some hemophilia treaters to reconsider the urgency of genetic cure. Current clinical and preclinical approaches to advancing AAV ‐based and alternative approaches to factor IX gene therapy are considered in the context of current demographics and treatment of the hemophilia B population.