Gene Therapy Treatment Proves Effective Against Rare Blood Clotting Disorder
A single injection. That’s all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of researchers at the University of North Carolina (UNC) and The Children’s Hospital of Philadelphia (CHOP).
Factor VII deficiency is a rare blood disorder that can lead to profuse bleeding after injuries, including minor cuts. Currently, patients must be injected regularly with protein factors. But the new gene therapy approach, details of which were published in the journal Blood, would require only one injection and occasional follow up to ensure that the body is still producing proper amounts of factor VII. Read the rest of this entry