Gene therapy for hemophilia: Progress to date and challenges moving forward

Over the past decades hemophilia has been transformed from a debilitating disease to a manageable condition. However, the current treatment options are expensive, complex, and inaccessible to a large portion of the global population. Moreover, the development of antibodies to replacement factors, termed inhibitors, is a common complication that not only renders conventional prophylaxis regimens ineffective but also increase the annual bleeding rate in affected patients. Fortunately, much progress has been made toward developing a curative gene therapy treatment for hemophilia and these efforts have led to a series of human trials with promising results.

Source: Transfusion and Apheresis Science - August 5, 2019 Category: Hematology Authors: Kandace L. Gollomp, Bhavya S. Doshi, Valder R. Arruda Source Type: research