Gene therapy for the hemophilias

Recent advances in the field of gene transfer are producing tantalizing results suggesting that the potential to correct disease at a molecular level may be at hand. Genetic correction of the hemophilias--bleeding disorders that stem from the deficiency of functional factor VIII or IX--represent models for the development of a basic understanding of how gene therapy will be achieved. The goals for hemophilia gene transfer are to produce therapeutic amounts of the coagulant protein while minimizing an immune response or antibody inhibitor. This requires the use of nontoxic vectors to deliver genes that express the protein in a functional form for the life of the patient. Based on a scientific understanding of the molecular and cellular defects leading to the bleeding phenotype, gene transfer studies at the laboratory and clinic have produced exciting results. The author here provides a critical assessment of the state of hemophilia gene transfer and its relevance to the field as a whole.