Therapeutic levels of human factor VIII in mice implanted with encapsulated cells : potential for gene therapy of hemophilia A

Garcia-Martin, C.;Chuah , Marinee;Van Damme, An;Vandendriessche, T.;Hortelano, G.;et.al.
(2002) Journal of Gene Medicine —

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Authors
  • Garcia-Martin, C.
    Author
  • Chuah , Marinee
    Collaborator
  • Van Damme, Anorcid-logoUCLouvain
    Collaborator
  • Vandendriessche, T.
    Collaborator
  • Hortelano, G.
    Collaborator
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Abstract
(en) BACKGROUND: A gene therapy delivery system based on microcapsules enclosing recombinant cells engineered to secrete a therapeutic protein has been evaluated. The microcapsules are implanted intraperitoneally. In order to prevent cell immune rejection, cells are enclosed in non-antigenic biocompatible alginate microcapsules prior to their implantation into mice. It has been shown that encapsulated myoblasts can deliver therapeutic levels of Factor IX (FIX) in mice. The delivery of human Factor VIII (hFVIII) in mice using microcapsules was evaluated in this study.
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Garcia-Martin, C. (2002). Therapeutic levels of human factor VIII in mice implanted with encapsulated cells : potential for gene therapy of hemophilia A. Journal of Gene Medicine. https://hdl.handle.net/2078.5/237088