Adrian Woolfson, MD, PhD, of Sangamo Therapeutics talks about the physical and psychological advantages of using gene therapy to treat hemophilia A.
Hemophilia A is a genetic blood disorder caused by a missing or defective factor VIII. Symptoms are dependent on the level factor VIII present but in more severe cases, excessive bleeding can lead to severe pain, especially at joints.
Currently, most people with severe hemophilia A take a recombinant factor VIII product to control bleeding. Unlike those treatments that require a lifetime of regular infusions, the gene therapy being developed by Sangamo Therapeutics would require just one injection that would ‘cure’ the person of the disease.
To learn more about this and other rare blood disorders, visit checkrare.com/diseases/blood-diseases/