Retroviral modification of mesenchymal stem cells for gene therapy of hemophilia

Methods Mol Biol. 2008:433:203-12. doi: 10.1007/978-1-59745-237-3_12.

Abstract

Mesenchymal stem cells (MSCs) are a promising target for the delivery of secreted proteins due to their ease of isolation, expansion, and genetic modification. The bleeding disorder hemophilia A results from the deficiency of a secreted blood clotting factor termed factor VIII (fVIII). Hemophilia A could be cured by gene-transfer-based procedures targeting virtually any cell type, including MSCs. Here, we describe methods for retroviral modification of MSCs incorporating a high-expression porcine (HEP)-fVIII transgene and a murine model of hemophilia A. MSCs were isolated from bone marrow of hemophilia A mice, expanded, and transduced ex vivo. Genetically modified MSCs secreted high levels of HEP-fVIII into the conditioned medium. HEP-fVIII was purified from the conditioned medium and demonstrated to have a specific activity, relative electrophoretic mobility, and proteolytic activation pattern similar to HEP-fVIII produced by other commercial cell lines. Collectively, these data support the concept that MSCs can be utilized as a cellular vehicle for successful gene-transfer-based therapy of hemophilia A and other disorders resulting from the deficiency of a secreted protein.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Separation
  • Electrophoresis, Polyacrylamide Gel
  • Factor VIII / biosynthesis
  • Factor VIII / genetics*
  • Factor VIII / therapeutic use
  • Genetic Therapy / methods*
  • Hemophilia A / genetics*
  • Hemophilia A / therapy*
  • Humans
  • Mesenchymal Stem Cells / metabolism*
  • Mice
  • Retroviridae / genetics*
  • Swine
  • Transduction, Genetic

Substances

  • Factor VIII