What is the role of gene therapy in the treatment of hemophilia A?

Updated: Jun 05, 2020
  • Author: Douglass A Drelich, MD; Chief Editor: Srikanth Nagalla, MBBS, MS, FACP  more...
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With the cloning of FVIII and advances in molecular technologies, the possibility of a cure for hemophilia with gene therapy was conceived. [64] Possible approaches to gene therapy for hemophilia A include the following [65] :

  • Ex vivo gene therapy, in which cells to be transplanted are genetically modified to secrete factor VIII and then are reimplanted into the recipient

  • In vivo gene therapy, in which a vector (typically a virus altered to include FVIII DNA) is directly injected into the patient

  • Nonautologous gene therapy, in which cells modified to secrete FVIII are packaged in immunoprotected devices and implanted into recipients

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