Collection of a mobilized peripheral blood apheresis product from a patient with mucopolysaccharidosis type VII and subsequent CD34+ cell isolation

A. Alex Hofling, Mark S. Sands, Douglas M. Lublin, Gerhard Bauer, Steven Devine

Research output: Contribution to journalArticle

4 Scopus citations


The effectiveness of bone marrow transplantation for lysosomal storage diseases like mucopolysaccharidosis type VII (MPSVII) suggests that a gene therapy strategy targeting autologous hematopoietic progenitor cells could be successful. Given the severe systemic manifestations of MPSVII including storage disease in the bone and bone marrow, it was unclear whether sufficient numbers of hematopoietic progenitor cells (CD34+) could be mobilized into the peripheral circulation and subsequently purified from these patients. As reported here, G-CSF mobilization and apheresis were successful, providing a product of 4 × 1010 nucleated cells containing 0.3% CD34 + progenitors. CD34 + cells were magnetically separated from the product to a final purity of 85% with a 64% yield. These results indicate that hematopoietic progenitors can safely be gathered from an MPSVII patient in numbers sufficient for the trial of clinical gene therapy applications.

Original languageEnglish (US)
Pages (from-to)151-153
Number of pages3
JournalJournal of Clinical Apheresis
Issue number3
StatePublished - 2004
Externally publishedYes



  • β-glucuronidase
  • Gene therapy
  • Lysosomal storage disease
  • Sly syndrome

ASJC Scopus subject areas

  • Hematology

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