How Do Gene Therapy Clotting Factors Work?

Reviewed on 8/2/2021

HOW DO GENE THERAPY CLOTTING FACTORS WORK?

Gene therapy clotting factors are drugs used for the treatment of hemophilia. The FDA approval for gene therapy is still pending.

Hemophilia is a medical condition characterized by a severe decrease in blood clotting that leads to extreme blood loss with a slight injury. It is usually caused by an inherited lack of a clotting factor, most often factor VIII. Clotting factors present in the human body are proteins in the blood that help control bleeding. Many clotting factors work together to form a clot that prevents excessive blood loss from the body.

Gene therapy codes for clotting factor VIII in the liver cells, which helps the body produce more clotting factors naturally, thereby reducing blood loss.

HOW ARE GENE THERAPY CLOTTING FACTORS USED?

Gene therapy clotting factors are beneficial for treating severe blood loss associated with hemophilia.

WHAT ARE SIDE EFFECTS OF GENE THERAPY CLOTTING FACTORS?

As gene therapy is still in clinical trials, no adverse effects have been reported.

WHAT ARE NAMES OF GENE THERAPY CLOTTING FACTORS?

Generic and brand names of gene therapy clotting factors include:

  • Roctavian
  • Valoctocogene Roxaparvovec (pending FDA approval)

QUESTION

What is hemophilia? See Answer
References
https://reference.medscape.com/drugs/clotting-factors-gene-therapy

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