Canine fucosidosis: A model for retroviral gene transfer into haematopoietic stem cells

Margaret L. Ferrara, Teresa Occhiodoro, Maria Fuller, Wayne J. Hawthorne, Suzy Teutsch, Virginia E. Tucker, John J. Hopwood, Graeme J. Stewart, Don S. Anson

Research output: Contribution to journalArticlepeer-review

15 Citations (Scopus)


Severe progressive fatal neurological degeneration occurs in fucosidosis, a storage disease. Bone marrow transplantation into affected dogs has shown that haematopoietic stem cells can provide enzyme producing daughter cells to the central nervous system, altering disease course. This makes canine fucosidosis an ideal large animal model for gene therapy. Fucosidosis affected allogeneic or autologous canine marrow was transduced ex vivo by cocultivation, then transplanted into fucosidosis affected dogs conditioned with total lymphoid irradiation. The vectors were Moloney murine leukaemia virus based. Transduction efficiency was increased with multiple cytokines in short term marrow culture. Despite high levels of transduction, proviral sequence was detected 2 months post transplant in only one dog. Early or total graft failure occurred in all transplants. We believe lack of engraftment could be caused by differentiation or change of repopulating ability of marrow cells occurring with mumble cytokine mixes in culture media.

Original languageEnglish
Pages (from-to)361-366
Number of pages6
JournalNeuromuscular Disorders
Issue number5
Publication statusPublished - Jul 1997


  • Canine fucosidosis
  • Haematopoietic stem cells
  • Retroviral gene therapy
  • Storage disease

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

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