Can hemophilia be treated with gene therapy?
Hemophilia is a genetic disease that prevents blood from clotting properly leading to prolonged internal and external bleeding. There are existing treatments for hemophilia that require lifelong administration, while gene therapy may offer a one-time treatment to address the cause of the disease.
What is gene therapy hemophilia?
Gene therapy for hemophilia involves using a modified virus (which does not cause disease) to introduce a copy of the gene that encodes for the clotting factor that’s missing in patients. Following treatment with the virus, patients should begin producing their own clotting factor normally.
How successful is gene therapy for hemophilia?
Gene therapy for hemophilia is here – and it’s working. The field is advancing at a steady clip: Results from recent trials of this revolutionary approach have restored patients’ anticoagulant factor activity levels to normal or near-normal levels and reduced patients’ annualized bleeding rates by nearly 90 percent.
Is hemophilia A candidate for gene therapy?
Haemophilia is an ideal candidate for gene therapy, as it is caused by mutations in a single gene. A number of vectors have been used in an attempt to obtain therapeutic levels of factor VIII and factor IX in animal models, with some success.
Which gene therapy is best for hemophilia?
As of October 2020, BioMarin’s valrox is the only gene therapy for hemophilia A that has reported interim phase III results.
Why is hemophilia good for gene therapy?
Hemophilia has always been an ideal target for gene therapy. The disease is caused by a single mutation; a gene has to be added, not knocked out, to fix it; and boosting even small levels of clotting protein can have a big impact.
How does gene therapy help hemophilia?
A gene therapy treatment restored blood clotting and transformed the disease from severe to mild. Hemophilia is a rare bleeding disorder in which blood doesn’t clot normally. Hemophilia B is caused by mutations in the gene for coagulation factor IX, a protein that helps blood to clot.
How does AAV work?
How does AAV work? Simply put, AAV is transformed from a naturally occurring virus into a delivery mechanism for gene therapy. The viral DNA is replaced with new DNA, and it becomes a precisely coded vector and is no longer considered a virus, as most of the viral components have been replaced.
What is hemophilia Factor 8?
Listen. May also be called: Factor VIII Deficiency. Hemophilia is a disease that prevents blood from clotting properly. A clot helps stop bleeding after a cut or injury. In factor VIII deficiency (hemophilia A), the body doesn’t make enough factor VIII (factor 8), one of the substances the body needs to form a clot.
What are the causes of hemophilia?
Hemophilia is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot. This change or mutation can prevent the clotting protein from working properly or to be missing altogether. These genes are located on the X chromosome.