Cellular therapy for childhood neurodegenerative disease. Part I: Rationale and preclinical studies

Daniel J. Guillaume, Stephen L. Huhn, Nathan R. Selden, Robert D. Steiner

Research output: Contribution to journalReview article

21 Scopus citations

Abstract

Successful cellular replacement in the diseased human central nervous system (CNS) faces numerous hurdles. In this first installment of a 2-part review, the authors report on the preclinical challenges involved in preparing for a major Phase I trial investigating the safety of human neural stem cell transplantation in a lysosomal storage disorder. Specifically, they discuss choice of the ideal disease for treatment, best donor cell type and source for implantation, the in vitro and in vivo methods used to estimate safety and efficacy, the challenges to noninvasive tracking of cells after transplantation, and the unique issues related to the immunology of CNS cellular transplantation.

Original languageEnglish (US)
Article numberE21
JournalNeurosurgical focus
Volume24
Issue number3-4
DOIs
StatePublished - Mar 1 2008

Keywords

  • Cell replacement therapy
  • Central nervous system cell transplantation
  • Human stem cell
  • Neural stem cel
  • Neurodegenerative diease
  • Restorative neurosurgery

ASJC Scopus subject areas

  • Surgery
  • Clinical Neurology

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