Education

Learn About Gene Therapy for Hemophilia

ASGCT Staff - November 12, 2019

Hemophilia occurs when the genes that are vital to producing clotting factors are faulty. 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. 

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for hemophilia.

Watch ASGCT's Hemophilia and Gene Therapy Video

The resources on hemophilia are now available on ASGCT.org. Hemophilia is a genetic disease that occurs when the genes that are vital to producing clotting factors are faulty. Clotting factors are proteins in blood that help our bodies stop bleeds. You can learn more about hemophilia's effect on the body here.

The information on hemophilia is part of the launch of a second round of material in ASGCT’s Patient Education program. ASGCT has been releasing new resources weekly on the Patient Education portal. Keep an eye on our website for the last two new resources of 2019:

  • Pompe—November 19
  • CAR T Basics—December 3

Let us know what you think of this second batch of resources. Send feedback via the ASGCT Contact form or at info@asgct.org

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