Advances in gene therapy for hemophilia

ABSTRACT

Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneousbleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenousfactor has improved patients’ quality of life, it has not been possible to establish a long-term treatment. Due tothe severity of the disease and the need for repetitive doses throughout the patient’s life, replacement therapyhas become a high-cost treatment option; therefore, the development of self-sustainable long-term therapies iscritical. Hemophilia is a good candidate for gene therapy because it is a monogenic disease that can becounteracted by expression of the missing factor. In this article, we review some of the most relevant advancesin gene therapy for this illness.