Transplantation of human central nervous system stem cells - neuroprotection in retinal degeneration

Trevor J. McGill, Benjamin Cottam, Bin Lu, Shaomei Wang, Sergej Girman, Chunyu Tian, Stephen L. Huhn, Ray D. Lund, Alexandra Capela

Research output: Contribution to journalArticlepeer-review

103 Scopus citations


Stem cells derived from the human brain and grown as neurospheres (HuCNS-SC) have been shown to be effective in treating central neurodegenerative conditions in a variety of animal models. Human safety data in neurodegenerative disorders are currently being accrued. In the present study, we explored the efficacy of HuCNS-SC in a rodent model of retinal degeneration, the Royal College of Surgeons (RCS) rat, and extended our previous cell transplantation studies to include an in-depth examination of donor cell behavior and phenotype post-transplantation. As a first step, we have shown that HuCNS-SC protect host photoreceptors and preserve visual function after transplantation into the subretinal space of postnatal day 21 RCS rats. Moreover, cone photoreceptor density remained relatively constant over several months, consistent with the sustained visual acuity and luminance sensitivity functional outcomes. The novel findings of this study include the characterization and quantification of donor cell radial migration from the injection site and within the subretinal space as well as the demonstration that donor cells maintain an immature phenotype throughout the 7months of the experiment and undergo very limited proliferation with no evidence of uncontrolled growth or tumor-like formation. Given the efficacy findings and lack of adverse events in the RCS rat in combination with the results from ongoing clinical investigations, HuCNS-SC appear to be a well-suited candidate for cell therapy in retinal degenerative conditions.

Original languageEnglish (US)
Pages (from-to)468-477
Number of pages10
JournalEuropean Journal of Neuroscience
Issue number3
StatePublished - Feb 2012


  • RCS rat
  • Retina
  • Stem cell
  • Vision

ASJC Scopus subject areas

  • General Neuroscience


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