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Human neural stem cells induce functional myelination in mice with severe dysmyelination.

Shiverer-immunodeficient (Shi-id) mice demonstrate defective myelination in the central nervous system (CNS) and significant ataxia by 2 to 3 weeks of life. Expanded, banked human neural stem cells (HuCNS-SCs) were transplanted into three sites in the brains of neonatal or juvenile Shi-id mice, which were asymptomatic or showed advanced hypomyelination, respectively. In both groups of mice, HuCNS-SCs engrafted and underwent preferential differentiation into oligodendrocytes. These oligodendrocytes generated compact myelin with normalized nodal organization, ultrastructure, and axon conduction velocities. Myelination was equivalent in neonatal and juvenile mice by quantitative histopathology and high-field ex vivo magnetic resonance imaging, which, through fractional anisotropy, revealed CNS myelination 5 to 7 weeks after HuCNS-SC transplantation. Transplanted HuCNS-SCs generated functional myelin in the CNS, even in animals with severe symptomatic hypomyelination, suggesting that this strategy may be useful for treating dysmyelinating diseases.

Pubmed ID: 23052293


  • Uchida N
  • Chen K
  • Dohse M
  • Hansen KD
  • Dean J
  • Buser JR
  • Riddle A
  • Beardsley DJ
  • Wan Y
  • Gong X
  • Nguyen T
  • Cummings BJ
  • Anderson AJ
  • Tamaki SJ
  • Tsukamoto A
  • Weissman IL
  • Matsumoto SG
  • Sherman LS
  • Kroenke CD
  • Back SA


Science translational medicine

Publication Data

October 10, 2012

Associated Grants

  • Agency: NINDS NIH HHS, Id: 1F30NS066704
  • Agency: NINDS NIH HHS, Id: 1R01NS054044
  • Agency: NINDS NIH HHS, Id: F30 NS066704
  • Agency: NCRR NIH HHS, Id: P51 RR000113
  • Agency: NCRR NIH HHS, Id: P51 RR000163
  • Agency: NCRR NIH HHS, Id: P51RR000163
  • Agency: NINDS NIH HHS, Id: R01 NS054044
  • Agency: NINDS NIH HHS, Id: R37 NS045737
  • Agency: NINDS NIH HHS, Id: R37NS045737-06S1/06S2

Mesh Terms

  • Animals
  • Brain
  • Central Nervous System
  • Demyelinating Diseases
  • Humans
  • Immunohistochemistry
  • Magnetic Resonance Imaging
  • Mice
  • Myelin Sheath
  • Neural Stem Cells
  • Stem Cell Transplantation